Caused when von Willebrand factor, a protein in the blood that is necessary for clotting, is either missing or not working properly, VWD affects men and women equally. Yet, when undiagnosed and untreated in women, it puts them at a greater risk for life-threatening bleeding following childbirth and for undergoing unnecessary hysterectomies.  

“Undiagnosed bleeding disorders, like von Willebrand disease, can have real health consequences for women,” said Elizabeth Battaglino Cahill, RN, executive director of HealthyWomen. “The goal of these new materials, available on HealthyWomen.org, is to spark honest conversations among mothers, sisters and daughters about the signs and symptoms of VWD and to hopefully underscore the need for even more education about this condition.”

The five signs and symptoms of von Willebrand disease are:

• Easy bruising
• Frequent or prolonged nosebleeds
• Heavy, prolonged menstruation
• Prolonged bleeding following injury or surgery
• Prolonged bleeding during dental procedures

“Because VWD is a hereditary disorder, many women dismiss tell-tale signs like easy bruising or heavy periods as normal because their mother or other female relatives experienced similar symptoms,” said Meera Chitlur, M.D., Director, Hemophilia Treatment Center and Hemostasis Program, Children’s Hospital of Michigan and who also works with the Foundation for Women & Girls with Blood Disorders (FWGBD), a non-profit advocacy and education organization dedicated to advancing physician and healthcare provider knowledge of the unique needs and challenges faced by women and adolescent girls with blood disorders.  “Unexplained bleeding or bruising is not normal and is something you should always discuss with your healthcare provider. If you are diagnosed with a bleeding disorder, treatments are available.”

The new materials include common questions about VWD answered by an expert, as well as real-life stories of women living with the condition. One of these women, Kristin Prior, 49, recalls her own diagnosis in 1996.

“While I had experienced many of the five signs and symptoms throughout early adulthood it wasn’t until I was 32 that I finally received my diagnosis of von Willebrand disease,” said Prior. “My hope is that women who are facing some of the same challenges that I did will read my story on HealthyWomen.org and realize that diagnosis and treatment can make a tremendous difference in their lives.”  

The von Willebrand disease educational resources were developed through an educational grant from CSL Behring, a world leader in developing and manufacturing safe and effective solutions to treat and manage bleeding disorders.

About HealthyWomen

HealthyWomen (HW) is the nation’s leading nonprofit health information source for women. For more than 20 years, women have been coming to HW for answers to their most pressing and personal health care questions. HW provides health information through a wide array of online content and print publications that are original, objective and reviewed and approved by medical experts. Its website, http://www.HealthyWomen.org®, was recognized by ForbesWoman as one of the “Top 100 Websites for Women” in 2010 and 2011 and was named the top women’s health website by Dr. Mehmet Oz in O, The Oprah Magazine. To learn more, visit www.HealthyWomen.org.

About the Foundation for Women & Girls with Blood Disorders

The Foundation for Women & Girls with Blood Disorders (FWGBD) is a non-profit advocacy and education organization dedicated to advancing physician and healthcare provider knowledge of the unique needs and challenges faced by women and adolescent girls with blood disorders.  Founded in 2010, the Foundation’s mission is to ensure that all women and adolescent girls with blood disorders are correctly diagnosed and optimally treated and managed at every life stage.  For more information, please visit www.fwgbd.org.

About CSL Behring

CSL Behring is a leader in the plasma protein therapeutics industry. Committed to saving lives and improving the quality of life for people with rare and serious diseases, the company manufactures and markets a range of plasma-derived and recombinant therapies worldwide. CSL Behring therapies are indicated for the treatment of coagulation disorders including hemophilia and von Willebrand disease, primary immune deficiencies, hereditary angioedema and inherited respiratory disease. The company’s products are also used in cardiac surgery, organ transplantation, burn treatment and to prevent hemolytic diseases in newborns. CSL Behring operates one of the world’s largest plasma collection networks, CSL Plasma. CSL Behring is a subsidiary of CSL Limited (ASX: CSL), a biopharmaceutical company headquartered in Melbourne, Australia. For more information, visit http://www.cslbehring.com/.

Contact

Erin Graves, Director of Communications and New Media
HealthyWomen
(732) 978-4947
egraves@HealthyWomen.org

Lauren Abel
MCS Public Relations on behalf of HealthyWomen and CSL Behring
(800) 477-9626
laurena@mcspr.com

SOURCE HealthyWomen

For Original Article click here.

31 Mar 2012

KING OF PRUSSIA, PA, USA - March 30, 2012 - CSL Behring announced today the first in human dosing of recombinant fusion protein linking coagulation factor VIIa with albumin (rVIIa-FP). The Phase I study will investigate in healthy volunteers the safety and pharmacokinetics of rVIIa-FP in comparison to placebo. CSL Behring, in collaboration with its parent company, CSL Limited (ASX: CSL.AX – News), is developing rVIIa-FP, a novel therapy to treat hemophilia A and hemophilia B patients who have inhibitors as part of the PROLONG 7- FP clinical study program. For more information about this study, please see www.clinicaltrials.gov.

CSL Behring’s albumin fusion technology uses albumin as the ideal recombinant genetic fusion partner for coagulation factor proteins due to its inherently long half-life, high potential for tolerability, known mechanism of clearance and low potential for immunogenic reactions. CSL Behring’s rVIIa albumin fusion protein is expected to exhibit a good tolerability profile and improved pharmacokinetics that may enable prophylaxis.

CSL Behring’s rVIIa-FP was previously granted Orphan Drug Designation by the European Commission and the United States Food and Drug Administration.

About Hemophilia
Hemophilia is a congenital bleeding disorder characterized by prolonged or spontaneous bleeding, especially into the muscles, joints, or internal organs. In nearly all cases, it affects only males. The disease is caused by deficient or defective blood coagulation proteins known as factor VIII or IX. The most common form of the disease is hemophilia A, or classic hemophilia, in which the clotting factor VIII is either deficient or defective. Hemophilia B is characterized by deficient or defective factor IX. Hemophilia A affects approximately 1 in 5,000 to 10,000 people. Hemophilia B affects approximately 1 in 25,000 to 50,000 people. The recommended treatment for patients who are factor deficient is to treat by replacement factor therapy. A complication in some patients is the development of inhibitory antibodies (inhibitors) to FVIII or FIX which render replacement therapy ineffective. This can occur in up to 25 percent of hemophilia A patients and approximately 5 percent of hemophilia B patients. One treatment option for these patients is recombinant activated factor VII (called a “bypassing agent”) which can be used to achieve hemostasis without the need for factor VIII or IX.

About the recombinant fusion protein linking coagulation factor VIIa with recombinant albumin (rVIIa-FP)
Preclinical studies have confirmed that CSL Behring’s rVIIa-FP has favorable pharmacokinetic properties compared with the existing recombinant FVIIa product. Significant increases in half-life have been observed across all animal species. The use of a bypassing agent with an extended half-life could offer significant benefit to those affected by hemophilia A or B with inhibitors and may offer patients the opportunity to be treated less frequently than with the currently available product.

CSL Behring’s clinical program intends to demonstrate that an extended half-life rVIIa-FP will result in a requirement for fewer doses while providing adequate therapeutic response in patients with hemophilia A and B with inhibitors.

About CSL Behring
CSL Behring is a leader in the plasma protein therapeutics industry. Committed to saving lives and improving the quality of life for people with rare and serious diseases, the company manufactures and markets a range of plasma-derived and recombinant therapies worldwide. CSL Behring therapies are indicated for the treatment of coagulation disorders including hemophilia and von Willebrand disease, primary immune deficiencies, hereditary angioedema and inherited respiratory disease. The company’s products are also used in cardiac surgery, organ transplantation, burn treatment and to prevent hemolytic disease of the newborn. CSL Behring operates one of the world’s largest plasma collection networks, CSL Plasma. CSL Behring is a subsidiary of CSL Limited (ASX:CSL.AX – News), a biopharmaceutical company headquartered in Melbourne, Australia. For information: http://www.cslbehring.com.

SOURCE: CSL Behring

The marketing terms “new and improved” and “longer lasting” are not limited to the latest brand of chewing gum. They also apply to a long list of therapies now in clinical trials for people with bleeding disorders. Some people have waited years for a new recombinant product; others a lifetime for any factor product to treat their rare condition. For many, their patience is about to pay off.

There are more drugs in the pipeline now than in the past few decades. “The companies’ commitment to continue to work on behalf of patients with bleeding disorders is what’s driving it,” says Val D. Bias, CEO of the National Hemophilia Foundation (NHF). The dilemma facing many patients in the future won’t be a lack of medications, but a plethora of products that act in a variety of ways. (See table “Bleeding Disorders Drugs in Human Clinical Trials.”)

For drugs to be approved and licensed by the US Food and Drug Administration (FDA), they have to go through a series of clinical trials. First they are tested on animals, such as mice; then they are tested on humans. Each phase of a clinical trial helps determine the drug’s safety, efficacy, optimal dosage and side effects. (See sidebar, “Clinical Trial Phases.”) The National Institutes of Health clinical trials registry at clinicaltrials.gov lists more than 250 trials on hemophilia and more than 60 on von Willebrand disease (VWD). (See “Clinical Trials 101.”).)

Tried and True vs. Something New

Prophylactic medications to treat hemophilia have given patients a new degree of freedom. They can self-infuse whenever and wherever it’s convenient.

“In terms of hemophilia A and B, I feel that the products we have right now are really good,” says Marion Koerper, MD, NHF medical advisor. She is also director emerita of the hemophilia treatment center at the University of California, San Francisco, where she practices pediatric hematology and oncology. “The factors do work to stop bleeding or, in the case of prophylaxis, prevent bleeding.”

However, prophylaxis is not perfect. “It’s only efficacious if the patient takes it the prescribed way,” Koerper says. The best time to give factor is in the morning before school or work, often the most hectic time of day. For busy families who delay treatment until bedtime, there are consequences. “That is not optimal because the child’s highest levels are while he’s asleep, rather than when he’s running around with his pals on the playground,” says Koerper.

Further, taking a product two or three times a week means that clotting strength can plummet on the off days. “When we give prophylaxis right now for a hemophilia A patient, we’re resolved to the fact that before their next prophy dose, their level in plasma could be as low as about 1% to 2%,” says Steven W. Pipe, MD, medical director, Pediatric Hemophilia and Coagulation Disorders Program, University of Michigan, Ann Arbor. That puts patients at risk for bleeding, especially if there is trauma. “Clearly, that’s not correction of their hemostasis.”

Products With Staying Power

To remedy that risk, pharmaceutical companies are creating new products that last longer in the bloodstream. The amount of factor VIII (FVIII) or factor FIX (FIX) in the blood is measured by its half-life, the time it takes for the amount of factor to be reduced by half. There are many variables involved, including blood type, but FVIII’s half-life is about 8–12 hours; FIX’s is about 18–24 hours. One option is to increase the interval between prophylactic doses, ideally to once a week for FIX products and twice a week for FVIII products. 

Another option is to retain the current prophylactic regimen, but avoid the precipitous drop in clotting factor as the next dosing time approaches. “We may be able to maintain much higher plasma levels than we’ve been able to previously with the same intervals that we’re currently using,” says Pipe.

One way to prevent factor products from degrading too quickly is to attach them to the chemical compound polyethylene glycol (PEG). This process, called PEGylation, increases the size of the factor protein molecule so that it circulates in the blood longer and is not cleared by the kidneys prematurely.

“Another strategy is to fuse the recombinant factor protein molecule to a partner protein that already has a long half-life,” says Pipe. Two naturally occurring partner proteins being fused to the FVIII or FIX molecule are albumin, which moves small molecules through the bloodstream, and Fc, a protein fragment that facilitates binding and recycling of immunoglobulin G (IgG).

Data from early clinical trials on Biogen Idec’s recombinant FVIII and FIX Fc fusion products, rFVIIIFc and rFIXFc, look promising. The A-LONG study on patients with severe hemophilia A showed a 1.7-fold increase in half-life during phase 1/2a clinical trials. B-LONG studies on patients with severe hemophilia B showed a nearly threefold increase in half-life during phase 1/2 trials. (See “Long Strides,” HemAware Summer 2011, p. 14.)

Adjunctive therapies, or drugs that are added to the primary factor product, are also being tested in clinical trials. Some use molecules that bind to tissue factor pathway inhibitor (TFPI), preventing it from hindering the action of FXa and thrombin, necessary for clot initiation and formation. Baxter’s BAX513 uses fucoidan, a seaweed extract being tested on healthy volunteers without hemophilia.

“If you block the proteins that are slowing down coagulation, you can actually restore normal clotting in hemophilic plasma without replacing the missing clotting factor,” says Pipe. For some patients, the adjunctive therapy may become the primary therapy, reducing the number of infusions needed, he says. A bonus is that some TFPI antagonists could be taken orally, such as the capsule form that delivered fucoidan to trial subjects.

“Compliance with bleeding disorders’ treatment is always an issue,” says Bias. “A drug that works better, faster and that you have to take less often can only improve that.”

Innovations for Inhibitors

An estimated 25% of patients with severe hemophilia A develop antibodies, called inhibitors, to the infused factor. Currently, patients undergo immune tolerance therapy to desensitize their immune systems or take a bypassing agent, such as FVIIa. The main drawbacks of the recombinant FVIIa product are that its half-life is only two hours and it is very expensive.

Inspiration Biopharmaceuticals is developing a recombinant porcine (pig) FVIII product for patients with inhibitors. “You can give a dose and get the measurable level of FVIII. That’s a distinct advantage when there’s a life-threatening­ bleed, like a head bleed (intracranial hemorrhage), or a limb-threatening bleed in someone with a compartment syndrome (increased pressure in a muscle in an enclosed space),” Koerper says. But because 80% of patients developed antibodies to plasma-derived pig factor within five days or after five doses, it is possible that a similar scenario might occur with the recombinant product. Results of the clinical trials will provide more data, but its use will probably be restricted.

The longer-lasting products may have an added benefit for inhibitor patients. “Some forms of PEGylation strategy and possibly even some of the fusion proteins may result in reduced risk for inhibitors,” says Pipe. Another product now being tested, Octapharma’s recombinant human-cl rhFVIII, may reduce the rate of inhibitor development because it uses proteins from human cells, not the typical hamster cells.

Recombinant VWD Product at Last

Recombinant products to treat FVIII and FIX were approved in 1992 and 1998, respectively; not so for von Willebrand factor (VWF). “It has bothered me for almost 20 years that I couldn’t offer a recombinant VWF product to my VWD patients,” says Koerper. That need will be fulfilled once Baxter’s recombinant VWF product goes through FDA approval and licensure. It will be targeted to patients with type 3 VWD, the most severe form, and those unresponsive to DDAVP, a synthetic hormone used to prevent or stop bleeds.

Gene Therapy Revisited

Researchers can now create precision drugs that treat diseases caused by specific genetic mutations. One such drug in phase 2 trials is Ataluren (PTC 124^®), manufactured by PTC Therapeutics Inc. It will be used for the approximately 10%–15% of patients with hemophilia A and B with a nonsense mutation, which halts factor production early. Ataluren introduces a molecule that allows the cell to read through the stop signal, making more clotting factor. It comes in a powder that is mixed in water. “Something that you can swallow is going to be a huge advantage because there are no needles involved,” Koerper says. (See sidebar “The Allure of Ataluren” in “What’s Your Genotype?” HemAware Spring 2010, p. 29.)

Rare Bleeding Disorders on the Radar

Patients with rare factor deficiencies know that being one in a million is hardly a cause for celebration. “People forget that there are other clotting factor deficiencies that, in some cases, have no treatment,” says Bias.

But hope is on the horizon. Companies that fractionate, or separate, plasma are interested in getting as many products out of it as they can, says Pipe. “Developing new markets for new plasma derivatives, such as the new FXIII product Corifact™ (approved by the FDA in March 2011), and RiaSTAP^®, a fibrinogen concentrate to treat FI deficiency (indicated for patients with congenital fibrinogen deficiency including afibrinogenemia and hypofibrinogenemia only), increases the sustainability and viability of the plasma fractionation industry.” Both products are manufactured by CSL Behring. Currently, Novo Nordisk has applied for a license for its recombinant FXIII product. British Plasma Laboratories has a plasma-derived FX product in phase 3 clinical trials.

“NHF is most supportive of new products for rare disorders or categories where products don’t currently exist, like the recombinant VWD product,” Bias says. “It’s important that people have access to a product that’s made for them.”

Time Frame for Trials

For drugs now in clinical trials, that access may take a few years. “From initiation of clinical trials to approval, it’s about a five-year window,” says Pipe. Drugs nearing the finish line—those in phase 3 or moving to FDA licensing—still have between 18 and 30 months, he says.

New Era of Optimism

Patients awaiting better, more effective or first-time products to treat their bleeding disorders have many reasons to be optimistic. “For the first time we’re now going to be offering agents that clearly behave differently. We’re not going to be faced with just a single-breed entity to choose from,” Pipe says.

The idea of having more distinct options may be foreign to some, but should be very welcome. New products with different mechanisms mean that treatments may soon be given in a more targeted, personalized manner. “When you have multiple choices it’s going to take some time for the clinicians and families to figure out what’s best for individual patients,” Pipe says.

When recombinant FVIII and FIX drugs came out two decades ago, Koerper thought they were the “ultimate products.” But with all of these recent innovations, she’s changed her thinking. “Now I realize there is so much more that can be done.”

—————————————————————————

CSL Behring has announced that the first patient has been screened in its recombinant coagulation single-chain factor VIII (rFVIII) trial, part of the AFFINITY clinical trial program. The CSL Behring rFVIII, called ‘rVIII-SingleChain’, is a novel molecule being studied for the treatment of hemophilia A. It is being developed by CSL Behring, in collaboration with its parent company, CSL Limited (ASX:CSL).

“CSL is advancing at a very encouraging rate with our recombinant coagulation factor development program,” said Dr. Andrew Cuthbertson, Chief Scientist CSL Limited. “Our ongoing commitment to providing safe and effective therapies to help improve the lives of those affected by hemophilia A and other rare bleeding disorders is being fulfilled with each milestone we reach. Our recombinant coagulation single-chain factor VIII molecule is showing strong promise. We look forward with excitement to seeing results of the phase I/III study in coming months.”

About the AFFINITY Phase I/III Study
The Phase I/III study is an open-label, multicenter trial that examines the crossover safety, efficacy and pharmacokinetics of recombinant coagulation single-chain factor VIII compared with recombinant human antihemophilic factor VIII (octocog alpha).

In Part 1 of the study, subjects will receive a single infusion of 50 IU/kg of octocog alpha followed by a single infusion of 50 IU/kg. In Parts 2 and 3 of the study, subjects will receive infusions of rVIII-SingleChain to prevent and treat bleeding (if required), at a dose and frequency determined by their study doctor (based on the subject’s underlying bleeding phenotype). More information about the study design can be found at www.clinicaltrials.gov.

Recombinant single-chain factor VIII consists of two linked protein chains – a heavy one and a light one. Under certain conditions, these chains can dissociate, resulting in the formation of separated, or “dissociated,” rFVIII chains. The CSL Behring rVIII-SingleChain uses a strong, covalent bond that connects the light and heavy chains, thereby creating a single chain rFVIII.

In-house studies have shown that the molecular integrity of rVIII-SingleChain is significantly increased using the single-chain design, resulting in a homogenous product that is more stable than currently available FVIII products. In addition, in-vitro studies have shown that rVIII-SingleChain demonstrates a very strong affinity for von Willebrand factor, resulting in a faster and more efficient binding to VWF. The FVIII/VWF complex plays an important role in the physiological activity and clearance of FVIII and has been shown to have an influence on the presentation of FVIII to the immune system.

The research leading to the initiation of the studies that CSL Behring is now conducting is the result of collaboration across the CSL Behring research sites in Marburg, Germany, in King of Prussia, USA, and at laboratories operated by CSL Limited in Melbourne, Australia.

About Hemophilia
Hemophilia is a congenital bleeding disorder characterized by prolonged or spontaneous bleeding, especially into the muscles, joints, or internal organs. In nearly all cases, it affects only males. The disease is caused by deficient or defective blood coagulation proteins known as factor VIII or IX. The most common form of the disease is hemophilia A, or classic hemophilia, in which the clotting factor VIII is either deficient or defective. Hemophilia B is characterized by deficient or defective factor IX. Hemophilia A affects approximately 1 in 5,000 to 10,000 people. Hemophilia B affects approximately 1 in 25,000 to 50,000 people. The recommended treatment for patients who are factor deficient is to treat by replacement factor therapy. A complication in some patients is the development of inhibitory antibodies (inhibitors) to FVIII or FIX which renders replacement therapy ineffective. This can occur in up to 25 percent of hemophilia A patients and around 5 percent of hemophilia B patients. One treatment option for these patients is recombinant activated factor VII (called a bypassing agent) that can be used to achieve hemostasis without the need for factor VIII or IX.

About CSL Behring
CSL Behring is a leader in the plasma protein therapeutics industry. Committed to saving lives and improving the quality of life for people with rare and serious diseases, the company manufactures and markets a range of plasma-derived and recombinant therapies worldwide. CSL Behring therapies are indicated for the treatment of coagulation disorders including hemophilia and von Willebrand disease, primary immune deficiencies, hereditary angioedema and inherited respiratory disease. The company’s products are also used in cardiac surgery, organ transplantation, burn treatment and to prevent hemolytic diseases in newborns. CSL Behring operates one of the world’s largest plasma collection networks, CSL Plasma. CSL Behring is a subsidiary of CSL Limited (ASX:CSL), a biopharmaceutical company headquartered in Melbourne, Australia. For more information, visit www.cslbehring.com.

###

Media Contact:
Sheila Burke
Worldwide Commercial Communications & Public Relations
CSL Behring
610-878-4209

Press release – Click here.

The CSL Behring rVIIa-FP clinical program will soon be initiated and intends to demonstrate that an extended half-life rVIIa-FP will result in a requirement for fewer doses while providing adequate therapeutic response in patients who have hemophilia A and B with inhibitors.

“CSL Behring’s albumin fusion technology uses albumin as the ideal recombinant genetic fusion partner for coagulation factor proteins because of its high tolerability, inherently long half-life, low potential for immunogenic reactions and known mechanism of clearance,” said Russell Basser, M.D., Senior Vice President, Global Clinical R&D at CSL Behring. “CSL Behring’s rVIIa albumin fusion protein is expected to exhibit a good tolerability profile and improved pharmacokinetics that may enable prophylaxis. We welcome Orphan Drug Designation for our rVIIa-FP and will work closely with the FDA to make this important therapy available for people in the U.S. with hemophilia A and hemophilia B with inhibitors.”

The FDA’s Orphan Drug Designation program provides orphan status to unique drugs and biologics, defined as those intended for the safe and effective treatment or prevention of rare diseases that affect fewer than 200,000 people in the U.S. Orphan designation qualifies the sponsor of the product for important tax credits, elimination of FDA license application fees and certain marketing incentives.

CSL Behring’s rVIIa-FP was granted Orphan Drug Designations (ODD) by the European Commission in May, 2011.

About Hemophilia
Hemophilia is a congenital bleeding disorder characterized by prolonged or spontaneous bleeding, especially into the muscles, joints, or internal organs. In nearly all cases, it affects only males. The disease is caused by deficient or defective blood coagulation proteins known as factor VIII or IX. The most common form of the disease is hemophilia A, or classic hemophilia, in which the clotting factor VIII is either deficient or defective.  Hemophilia B is characterized by deficient or defective factor IX. Hemophilia A affects approximately 1 in 5,000 to 10,000 people. Hemophilia B affects approximately 1 in 25,000 to 50,000 people. The recommended treatment for patients who are factor deficient is to treat by replacement factor therapy. A complication in some patients is the development of inhibitory antibodies (inhibitors) to FVIII or FIX which renders replacement therapy ineffective.  This can occur in up to 25 percent of hemophilia A patients and around 5 percent of hemophilia B patients.  One treatment option for these patients is recombinant activated factor VII (called a bypassing agent) which can be used to achieve hemostasis without the need for factor VIII or IX. 

About the recombinant fusion protein linking coagulation factor VIIa with albumin (rVIIa-FP)
Preclinical studies have confirmed that CSL Behring’s rVIIa-FP has favorable pharmacokinetic properties compared with the existing recombinant FVIIa product. Significant increases in half-life have been observed across all animal species. The use of a bypassing agent with an extended half-life could offer significant benefit to those affected by hemophilia A or B with inhibitors and may offer patients the opportunity to be treated less frequently than with currently available product.

About CSL Behring
CSL Behring is a leader in the plasma protein therapeutics industry. Committed to saving lives and improving the quality of life for people with rare and serious diseases, the company manufactures and markets a range of plasma-derived and recombinant therapies worldwide. CSL Behring therapies are indicated for the treatment of coagulation disorders including hemophilia and von Willebrand disease, primary immune deficiencies, hereditary angioedema and inherited respiratory disease. The company’s products are also used in cardiac surgery, organ transplantation, burn treatment and to prevent hemolytic diseases in newborns. CSL Behring operates one of the world’s largest plasma collection networks, CSL Plasma. CSL Behring is a subsidiary of CSL Limited (ASX:CSL), a biopharmaceutical company headquartered in Melbourne, Australia. For more information, visit www.cslbehring.com.

For Press release click here.

SOURCE CSL Behring

Results from the Phase I of the PROLONG-9FP trial program demonstrate clinical safety and improved pharmacokinetics of rIX-FP compared to current treatment options

St. Gallen, Switzerland — 02 February 2012

CSL Behring today announced the results of a Phase I study evaluating recombinant fusion protein linking coagulation Factor IX with albumin (rIX-FP) in patients with severe hemophilia B. Results of the study, which were presented during an oral session at the Gesellscaft fur Thrombose- und Hameostasegorschung (GTH) congress in Switzerland, showed that rIX-FP was well tolerated in all patients and lasted longer in the body, due to its prolonged half-life, compared with current Factor IX treatment options.

CSL Behring, in collaboration with its parent company, CSL Limited (ASX:CSL), is developing rIX-FP for the prophylaxis and treatment of bleeding episodes in patients with congenital Factor IX (FIX) deficiency as part of the PROLONG-9FP clinical study program.

“Hemophilia B is a rare and serious bleeding disorder that prevents normal blood clotting and requires frequent infusion of Factor IX concentrates to restore clotting ability,” said Elena Santagostino, M.D., Ph.D., Professor in the Medical School of Clinical and Experimental Hematology at the University of Milan/IRCCS Maggiore Hospital. “The results of this study suggest that rIX-FP is a promising investigational agent for improvement of prophylactic and on-demand treatment for patients with hemophilia B.”

In this analysis, no serious adverse events (including no hypersensitivity reactions), presence of inhibitors to Factor IX, or antibodies to rIX-FP were reported. Terminal half-life (a measure of how long the drug lasts in the body) was more than five-times longer in comparison to values associated with current recombinant FIX therapy. Incremental recovery and area under the curve (a measure of total exposure to the drug) were also significantly improved in comparison to values associated with current recombinant FIX therapy.

“The development of this new recombinant investigational agent further adds to CSL Behring’s long heritage of identifying innovative treatments to improve outcomes for those living with rare bleeding disorders,” said Dr. Stefan Schulte, Vice President of Research and Development, CSL Behring. “We look forward to further exploring the potential of rIX-FP in patients with hemophilia B.”

CSL Behring and CSL Limited have engineered the rIX-FP albumin fusion protein to extend the half-life of Factor IX while minimizing tolerability issues. In the process, recombinant albumin—a carrier protein with an inherently long half-life—is used as a fusion partner. A specifically designed linker connects the recombinant factor IX and recombinant albumin as a means of optimizing the efficacy of rIX-FP.

About the Phase I Study
The Phase I study, part of the PROLONG-9FP clinical program, was a multi-center, international open-label trial evaluating rIX-FP in patients with documented severe hemophilia B (FIX = 2%). The primary objective was to determine the safety of rIX-FP up to 28 days following intravenous injection of 25, 50, or 75 IU/kg. The secondary objective was to evaluate the pharmacokinetics of rIX-FP. More information about the study design can be found at www.clinicaltrials.gov.

About Hemophilia
Hemophilia is an inherited bleeding disorder characterized by prolonged or spontaneous bleeding, especially into the muscles and joints. In nearly all cases, it affects only males. The disease is caused by deficient or defective blood coagulation proteins known as factor VIII or IX. The most common form of the disease is hemophilia A, or classic hemophilia, in which the clotting factor VIII is either deficient or defective. Hemophilia B is characterized by deficient or defective factor IX. Hemophilia A affects approximately 1 in 5,000 to 10,000 people. Hemophilia B affects approximately 1 in 25,000 to 50,000 people. The recommended treatment for patients who are factor deficient is to treat by replacement factor therapy.

About CSL Behring
CSL Behring is a global leader in the plasma protein biotherapeutics industry. Passionate about improving the quality of patients’ lives, CSL Behring manufactures and markets a range of safe and effective plasma-derived and recombinant products and related services. The company’s therapies are used in the treatment of immune deficiency disorders, hereditary angioedema, haemophilia, von Willebrand disease, other bleeding disorders and inherited emphysema. Other products are used for the prevention of hemolytic diseases in the newborn, in cardiac surgery, organ transplantation and in the treatment of burns. The company also operates one of the world’s largest plasma collection networks, CSL Plasma. CSL Behring is a subsidiary of CSL Limited, a biopharmaceutical company with headquarters in Melbourne, Australia. For more information, visit www.cslbehring.com.

Media Contact:
Sheila A. Burke, Director, Communications & Public Relations
Worldwide Commercial Operations
CSL Behring
610-878-4209 (o)
484-919-2618 (c)
Sheila.Burke@cslbehring.com 

Etanjalie Ayala
Weber Shandwick
212-445-8225
eayala@webershandwick.com

SOURCE CSL Behring

Vienna, Austria — 12 January 2012

CSL Behring has announced that the first site has been initiated in its global phase II/III, multi-center study to evaluate the safety, efficacy and pharmacokinetics of recombinant fusion protein linking coagulation factor IX with recombinant albumin (rIX-FP). The site is located in Vienna, Austria. The prospective, open-label study will enroll adolescents and adults (12 – 65 years) who have hemophilia B. CSL Behring, in collaboration with its parent company, CSL Limited (ASX:CSL), is developing rIX-FP for the prophylaxis and treatment of bleeding episodes in patients with congenital Factor IX (FIX) deficiency as part of the PROLONG-9FP clinical study program.

“The unmet medical need is great for a factor IX product with an extended half-life for use in treating people with hemophilia B, a life-long, debilitating clotting disorder,” said Russell Basser, MD, Senior Vice President, Global Clinical R&D at CSL Behring. “Such a therapy can mean fewer injections for patients, and may enable or enhance prophylactic treatment. This would be an improved convenience that may result in a better quality of life for patients.”

“We have entered a very exciting and promising era for patients with hemophilia,” said Elena Santagostino, MD, Principal Investigator for the study. “A recombinant factor IX product with a longer half-life, such as the product that CSL Behring is developing, will have the potential to prevent bleeds in people who have hemophilia B. Today, these individuals generally must undergo frequent infusions of factor product to effectively manage their condition. Such a routine tends to present challenges; maintaining adherence to it can be difficult and one’s quality of life can therefore be impacted. The product in development today aims to reduce the number of infusions a patient with hemophilia must undergo. As treating physicians and clinical researchers, we are proud to be a part of this effort and look forward with great anticipation to the results of our research.”

To date, the PROLONG-9FP program has established study sites in Austria, Bulgaria, France, Germany, Italy, Spain and in Israel. It is anticipated that in coming months additional trial sites will be established in the United States, Japan, and Russia.

CSL Behring and CSL have engineered rIX-FP to extend the half life of Factor IX while minimizing any tolerability issues. In the process, recombinant albumin—a protein with an inherently long half-life—is used as a fusion partner. A specifically designed linker connects the recombinant factor IX and recombinant albumin as a means of optimizing the efficacy of rIX-FP.

The Phase II/III study consists of a screening period, a pharmacokinetic (PK) evaluation period, followed by an approximate 12-month safety and efficacy evaluation period with rIX-FP. A surgical prophylaxis sub-trial is included. More information can be found at http://clinicaltrials.gov/ct2/results?term=rIX-FP+.

About Hemophilia
Hemophilia is an inherited bleeding disorder characterized by prolonged or spontaneous bleeding, especially into the muscles, joints, or internal organs. In nearly all cases, it affects only males. The disease is caused by deficient or defective blood coagulation proteins known as factor VIII or IX. The most common form of the disease is hemophilia A, or classic hemophilia, in which the clotting factor VIII is either deficient or defective. Hemophilia B is characterized by deficient or defective factor IX. Hemophilia A affects approximately 1 in 5,000 to 10,000 people. Hemophilia B affects approximately 1 in 25,000 to 50,000 people. The recommended treatment for patients who are factor deficient is to treat by replacement factor therapy.

About CSL Behring
CSL Behring is a leader in the plasma protein therapeutics industry. Committed to saving lives and improving the quality of life for people with rare and serious diseases, the company manufactures and markets a range of plasma-derived and recombinant therapies worldwide. CSL Behring therapies are indicated for the treatment of coagulation disorders including hemophilia and von Willebrand disease, primary immune deficiencies, hereditary angioedema and inherited respiratory disease. The company’s products are also used in cardiac surgery, organ transplantation, burn treatment and to prevent hemolytic diseases in newborns. CSL Behring operates one of the world’s largest plasma collection networks, CSL Plasma. CSL Behring is a subsidiary of
CSL Limited (ASX: CSL), a biopharmaceutical company headquartered in Melbourne, Australia. For more information, visit www.cslbehring.com.

Contact:
Sheila A. Burke, Director, Communications & Public Relations
Worldwide Commercial Operations
CSL Behring
610-878-4209 (o)
484-919-2618 (c)
Sheila.Burke@cslbehring.com

CSL Behring announced today that it has been granted Orphan Drug Designations (ODD) by the European Commission for the development of its recombinant fusion protein linking coagulation factor VIIa with albumin (rVIIa-FP), a novel therapy to treat hemophilia A and hemophilia B patients with inhibitors. The designations would entitle CSL Behring to exclusively market recombinant factor VIIa fused with albumin in Europe for a period of 10 years if the product at the stage of license application fulfils the orphan drug requirements. Based on the submission of data from the company’s Pediatric Investigation Plan, once available, the 10-year market exclusivity may be extended to 12 years.

Under these designations European Medicines Agency (EMA) will also provide CSL Behring with development assistance and with reductions in certain regulatory fees.

“CSL Behring welcomes Orphan Drug Designation for rVIIa-FP as support of our ongoing commitment to developing, manufacturing and marketing products for the treatment of rare and serious diseases, such as hemophilia with inhibitors,” said Val Romberg, Senior Vice President, Global Research & Development. “We will continue to work closely with the EMA to make this important therapy available to patients as soon as possible.”

An orphan drug designation application has not yet been submitted in the United States.

By providing incentives to the pharmaceutical industry, the EU legislative framework for orphan medicines encourages the development of products intended to diagnose, prevent and treat life-threatening or chronically-debilitating conditions that impact up to 5 in 10,000 people in the European Union. The initiative helps improve access to quality medical care for patients who have rare diseases for which there are few, if any, approved treatments.

About Hemophilia
Hemophilia is an inherited bleeding disorder characterized by prolonged or spontaneous bleeding, especially into the muscles, joints, or internal organs. The disease is caused by deficient or defective blood coagulation proteins known as factor VIII or IX. The most common form of the disease is hemophilia A, or classic hemophilia, in which the clotting factor VIII is either deficient or defective. Hemophilia B is characterized by deficient or defective factor IX. Hemophilia A affects approximately 1 in 5,000 to 10,000 people. Hemophilia B affects approximately 1 in 25,000 to 50,000 people. The recommended treatment for patients who are factor deficient is to treat by replacement factor therapy.

Some patients develop inhibitors, factor VIII or IX neutralizing antibodies which render further replacement therapy ineffective. It has been reported that up to 33% of all severe hemophilia A and up to 6% of all severe hemophilia B patients develop inhibitors.

Patients who have become refractory to replacement factor therapy can be treated with recombinant human factor VIIa. Factor VIIa is an enzyme that can both initiate blood clotting and, at high dose, “bypass” the factor VIII and IX dependent steps involved in effective coagulation. With the current commercially available recombinant product, frequent injections are needed to adequately control or prevent bleeding due to its inherent short half-life.

About the recombinant fusion protein linking coagulation factor VIIa with albumin (rVIIa-FP)
Using a proprietary genetic fusion technology (patents pending), CSL Behring is in the pre-clinical phase of developing a novel fusion protein formed by linking recombinant Factor VIIa with albumin. Because albumin is the most abundant natural protein in plasma and has a very long half-life (i.e., more than 20 days), the CSL Behring fusion protein is expected to exhibit a good tolerability profile and improved pharmacokinetics that may allow for less frequent dosing.

Preclinical studies have confirmed that CSL Behring’s rVIIa-FP has favorable pharmacokinetic properties compared to the existing recombinant product. A half-life extension of greater than 8-fold has been observed. The use of a bypassing agent with an extended half-life could offer significant benefit to those affected by hemophilia A or B with inhibitors and may offer patients the opportunity to be treated less frequently than with the currently available product.

CSL Behring’s clinical program intends to demonstrate that an extended half-life rVIIa-FP will result in a requirement for fewer doses while providing adequate therapeutic response in patients with hemophilia A and B with inhibitors.

About CSL Behring
CSL Behring is a leader in the plasma protein therapeutics industry. Committed to saving lives and improving the quality of life for people with rare and serious diseases, the company manufactures and markets a range of plasma-derived and recombinant therapies worldwide. CSL Behring therapies are indicated for the treatment of coagulation disorders including hemophilia and von Willebrand disease, primary immune deficiencies, hereditary angioedema and inherited respiratory disease. The company’s products are also used in cardiac surgery, organ transplantation, burn treatment and to prevent hemolytic diseases in newborns. CSL Behring operates one of the world’s largest plasma collection networks, CSL Plasma. CSL Behring is a subsidiary of CSL Limited (ASX: CSL), a biopharmaceutical company headquartered in Melbourne, Australia. For more information, visit www.cslbehring.com.

FDA NEWS RELEASE
For Immediate Release: Feb. 17, 2011
The U.S. Food and Drug Administration today approved Corifact, the first product intended to prevent bleeding in people with the rare genetic defect congenital Factor XIII deficiency.

Patients with congenital Factor XIII deficiency don’t make enough Factor XIII, a substance that circulates in the blood and is important for normal clotting. Without treatment, people with the condition are at risk for life-threatening bleeding.

Congenital Factor XIII deficiency is rare and affects 1 out of every 3 million to 5 million people in the United States. The deficiency may lead to soft tissue bruising, mucosal bleeding and fatal intracranial bleeding. Newborns with Factor XIII deficiency may have umbilical cord bleeding.

“This product helps fill an important need,” said Karen Midthun, M.D., director of the FDA’s Center for Biologics Evaluation and Research.

Corifact received orphan-drug designation by the FDA because it is intended for use in a rare disease or condition. It was approved for marketing under the FDA’s accelerated approval regulations that require an on-going study to demonstrate that patients actually receive the clinical benefit predicted by the data obtained so far.

The FDA approved Corifact based on results of a clinical study of 14 people, including children, with congenital Factor XIII deficiency. The most common side effects observed were hypersensitivity reactions (allergy, rash, pruritus, and erythema), chills, fever, arthralgia, headache, elevated thrombin-antithrombin levels, and an increase in liver (hepatic) enzymes.

Corifact is made from the pooled plasma of healthy donors. People receiving Corifact may develop antibodies against Factor XIII that may make the product ineffective. It potentially can cause adverse events from abnormal clotting if doses higher than the labeled dose are given to patients.

Corifact is manufactured by CSL Behring of Marburg, Germany.

For more information: visit CSL Behring’s website.

For more information, including full prescribing information, visit http://www.corifact.com/.

The National Hemophilia Foundation (NHF) launched its new initiative, Victory for Women, at their 62nd Annual Meeting in New Orleans, November 11 through 13. Since most women affected by the most common hereditary bleeding disorder, von Willebrand disease (VWD), don’t even know they have it, the campaign is designed to generate awareness outside of the bleeding community and empower women to seek accurate diagnosis and healthcare.

At the center of the campaign is the music video, “Victory for Women,” featuring emerging contemporary musical artist, Jessica Rae, who also composed the music. The song uses a catchy tune and an emotional appeal to build awareness of and goodwill toward NHF. Tom Jordan, an award winning creative director at Hoffman York with a portfolio of original music, wrote the upbeat and optimistic lyrics to unite and evoke emotion among women with blood disorders. Kevin Kalausakas was brought on to add the visual element as the music video producer. The goal of the video is for people to “spread the word” about blood disorders through the various new communications and social media channels (Facebook, Twitter, etc.) and spark concern in women to protect their bodies and to form a community in which women with these blood disorders can turn to for support.

Also featured in the video is a maroon, silk scarf that was created for the Victory for Women initiative to serve as a symbol of unity and support for women with blood disorders. To view the video visit, http://on.fb.me/boW63k.

“We believe in the power of music to stir emotion and help people open their hearts to this incredible cause,” said Jordan. “We used the scarf in the video to show women they’re not alone.”

At the annual meeting, NHF also recognized three fashion, film and television luminaries for their efforts in helping put hemophilia and other bleeding disorders in the spotlight. Patrick McMullan, the famed New York City nightlife photographer and Vanity Fair contributing editor, was honored for donating his photography skills to create a “Faces of Women with Bleeding Disorders” calendar for the Victory for Women campaign featuring women with bleeding disorders wearing the maroon, silk scarf. McMullan’s efforts are fueled by his passion to keep his sister’s memory alive. Doreen McMullan McCarthy, who died in 1979 from complications of von Willebrand disease, is featured in “Iron Butterfly,” the cover story for the Fall 2010 issue of HemAware, NHF’s award-winning magazine and can be viewed at http://www.hemaware.org.

Creator and producer of USA Network TV show “Royal Pains” Andrew Lenchewski and his father, Dr. Enrique Lenchewski were recognized for creating the popular character Tucker Bryant, a teenager with hemophilia and Emmy Award-winning film producer, Marilyn Ness, was recognized for her documentary “Bad Blood: A Cautionary Tale.”

As the largest bleeding disorders conference in the country, NHF’s Annual Meeting was attended by more than 2,000 guests, including consumers, healthcare providers, researchers, industry representatives, and NHF chapter/association staff from across the country, and other global nonprofit organizations. Making the conference a great opportunity to kick off the Victory for Women initiative and start raising awareness about blood disorders affecting women.

NHF hopes to raise awareness of the Victory for Women initiative outside the bleeding disorders community through social media channels with Americans sharing this message with friends and family. To be a part of Victory for Women follow the cause on Facebook and Twitter. To view the “Victory for Women” music video check out the NHF YouTube channel or visit http://on.fb.me/boW63k.

For more information about the Victory for Women initiative or to make a donation, visit http://www.victoryforwomen.org. Donations of $15 receive the “Faces of Women with Bleeding Disorders” calendar; donations of $20 receive the red, silk scarf featured in the video and in the calendar.

About the National Hemophilia Foundation
The National Hemophilia Foundation is dedicated to finding better treatments and cures for bleeding and clotting disorders and to preventing the complications of these disorders through education, advocacy and research. Established in 1948, the National Hemophilia Foundation has 46 chapters throughout the country. Its programs and initiatives are made possible through the generosity of individuals, corporations and foundations as well as through a cooperative agreement with the Centers for Disease Control and Prevention (CDC). For more information about the National Hemophilia Foundation and bleeding disorders visit http://www.hemophilia.org.

About von Willebrand disease
Von Willebrand disease is a genetic bleeding disorder that prevents blood from clotting properly. This disorder can be far more serious in women, and is often undetected. While von Willebrand disease is the most common bleeding disorder, girls and women may suffer from other more rare bleeding disorders including platelet abnormalities. Women who are carriers for hemophilia may also experience symptoms consistent with mild hemophilia. For more information, visit http://www.victoryforwomen.org.

VWD is caused by a lack of working von Willebrand factor (VWF), a protein in the blood that is necessary for clotting. The Centers for Disease Control and Prevention (CDC) estimates that VWD affects one to two percent or three to six million people in the U.S. alone. While the disorder affects men and women equally, women are at a greater risk for miscarriage, life-threatening bleeding following childbirth, and for undergoing unnecessary hysterectomies.

“Women struggle with the consequences of this disorder for an average of 16 years before they receive a proper diagnosis because sometimes healthcare professionals interpret VWD symptoms as gynecologic, when in fact the underlying cause is hematologic,” said Josie Weiss, PhD, FNP-BC, Associate Professor, Christine E. Lynn College of Nursing at Florida Atlantic University and Fellow of the American Academy of Nurse Practitioners. “The teenage years are both the best time and provide the best opportunity to recognize von Willebrand disease because the onset of menstruation often reveals a commonly overlooked symptom — heavy and prolonged bleeding.”

The AANP urges young women to visit their healthcare provider if they are experiencing any of these five signs and symptoms of VWD:

• Easy bruising
• Frequent or prolonged nosebleeds
• Heavy, prolonged menstruation
• Prolonged bleeding following injury, childbirth or surgery
• Prolonged bleeding during dental procedures

AANP’s VWD Young Women’s Education Campaign includes educational materials developed for both healthcare professionals and patients. These materials include VWD fact sheets, a screening questionnaire and links to hemophilia treatment centers (HTCs) that specialize in treating bleeding disorders. The virtual toolkit can be found in the AANP Toolkits section of the organization’s website.

This campaign is supported through an educational grant from CSL Behring, a leader in the plasma protein therapeutics industry. CSL Behring is committed to the bleeding disorders community through outreach, education, research and support of a wide variety of activities and programs including Von U, its VWD community of care.

More information about VWD and links to HTCs can be found at www.AANP.org or www.allaboutbleeding-US.com

About von Willebrand Disease (VWD)

Von Willebrand disease (VWD) is caused by a deficiency or abnormality of von Willebrand factor (VWF), a protein in the blood that is necessary for normal blood clotting. Men and women are equally likely to be affected by VWD. VWD is classified by types, ranging from Type 1 (the most common and mild) to Type 3 (the most severe).

Women with VWD are more likely to experience heavy, prolonged menstruation. Other common symptoms of VWD include frequent nosebleeds and easy bruising. Bleeding can be mild or serious and can occur as a result of injury, or without any obvious cause. More serious symptoms include bleeding into joints and internal organs. The VWD patient may require special care during dental procedures, surgery and childbirth.

Treatments for VWD may include desmopressin acetate to release stored von Willebrand factor, von Willebrand factor replacement therapies to raise von Willebrand factor in the bloodstream and oral contraceptives to reduce menstrual bleeding.

About the American Academy of Nurse Practitioners

Created in 1985 to provide nurse practitioners with a unified way to network and to advocate for nurse practitioner issues across all specialties at the local, state, and national levels, the American Academy of Nurse Practitioners (AANP) has continually served as a major resource for NPs, their patients and other healthcare consumers, to promote excellence in practice, education and research; to provide legislative leadership to advance health policy; to establish healthcare standards and to advocate for access to high-quality healthcare. AANP is the oldest, largest and only full-service national professional membership organization for NPs of all specialties, representing the interests of the 135,000 NPs practicing in the United States today. For more information about AANP, visit www.aanp.org.

About CSL Behring

CSL Behring is a leader in the plasma protein therapeutics industry. Committed to saving lives and improving the quality of life for people with rare and serious diseases, the company manufacturers and markets a range of plasma-derived and recombinant therapies worldwide. CSL Behring therapies are indicated for the treatment of coagulation disorders including hemophilia and von Willebrand disease, primary immune deficiencies and inherited respiratory disease. The company’s products are also used in cardiac surgery, organ transplantation, burn treatment and to prevent hemolytic diseases in newborns. CSL Behring operates one of the world’s largest plasma collection networks, CSL Plasma. CSL Behring is a subsidiary of CSL Limited (ASX: CSL), a biopharmaceutical company headquartered in Melbourne, Australia. For more information, visit www.cslbehring.com.

CONTACT: Elaine Andrecovich MCS Healthcare Public Relations on behalf of AANP (800) 477-9626

SOURCE American Academy of Nurse Practitioners

Source: PR Newswire

Children with bleeding disorders and their families are invited to participate in four regional golf and baseball competitions to determine Junior National Champion

However, the reality of PID is very different.  First off, it’s not one disease. The World Health Organization recognizes over 150 different forms of PID. Some, such as selective IgA deficiency, are not even that rare, affecting as many as 1:500 of the population.  Furthermore, today, if a baby like David receives a bone marrow transplant within the first 3.5 months of life, the survival rate can be as high as 94 percent.  For many of the other PID diseases, patients can live healthy and productive lives with the help of replacement immune globulin therapy.

Produced from the plasma of thousands of healthy donors, immunoglobulin, is a concentrated mixture of antibodies that provides the patient with an “artificial immune system.”  When first developed in the 1950’s, the treatment was administered by painful intramuscular injections (IMIG).

In the 1970’s, the first intravenous immunoglobulin (IVIG) preparations were developed. These quickly became the standard of therapy, as they were more effective, reducing both the need for antibiotics and hospitalizations for infections.  However, the required monthly infusions could take several hours and were associated with a high incidence of side effects.

In 2006 the FDA approved the first subcutaneous immunoglobulin (SCIg) therapy for PID. Because SCIg can be self-administered, patients can schedule infusions to suit their own lifestyle. The dosage schedule  also allows patients to maintain more consistent immunoglobulin levels and for some, SCIg is also associated with fewer side-effects.

Last week, life got even better for these patients.  CSL Behring announced that the U.S. Food and Drug Administration (FDA) had granted marketing approval for Hizentra™, Immune Globulin Subcutaneous (Human), 20% Liquid. Hizentra is the first 20% concentration SCIg. This concentration enable patients to administer up to 20g of IgG in a single infusion, making it the first once weekly SCIg. It’s also the first SCIg to be stabilized with L-proline, allowing the product to be stored at room temperature.

“With its high concentration, Hizentra is a welcome new SCIg treatment option for patients managing primary immunodeficiencies,” said John Sleasman, M.D., Professor and Chief of the Division of Allergy, Immunology and Rheumatology at the University of South Florida College of Medicine, Department of Pediatrics. “Hizentra’s ready-to-use attribute will allow patients to infuse the product where and when it suits them, and physicians now have another product to select to best meet the individual needs of their patients.”

SRxA and Word on Health congratulate CSL Behring on developing a product that will truly improve the lives of patients with PID.

This new indication was granted by the FDA after determining Helixate FS is safe and effective for routine prophylaxis, which can reduce the frequency of bleeding episodes and the risk of joint damage in children with hemophilia A.

“The FDA’s approval of this additional indication for Helixate FS will significantly benefit the bleeding disorders community,” said Garrett E. Bergman, M.D. Senior Director of Medical Affairs, U.S. Commercial Operations at CSL Behring. “Prophylactic treatment will reduce the frequency of bleeding episodes in children with hemophilia A which, over time, will help prevent additional joint damage caused by bleeds.”

“Bleeding into joints, including the ankles, elbows and knees, is a concern for children with hemophilia A and their treatment providers,” said Jerry S. Powell, M.D., Director of the Hemophilia Treatment Center at the University of California, Davis. “The FDA’s approval of Helixate FS for routine prophylaxis is an innovation that will help our patient population effectively manage their condition. Modern treatment of hemophilia means preventing bleeding through routine prophylaxis, and we eagerly anticipate our patients reaching young adulthood with minimal joint damage and the ability to pursue normal lives.”

With more than 2.5 billion units infused to date, Helixate FS has been shown to be safe and effective in clinical studies as well as in post marketing use in the hemophilia community. For more information about Helixate FS, please visit www.HelixateFS.com or call CSL Behring Consumer Affairs at 1-888-508-6978.

About Helixate FS

Helixate FS is a recombinant factor VIII product indicated to control and prevent bleeding episodes in adults and children with hemophilia A. Helixate FS is also indicated for routine prophylaxis to reduce the frequency of bleeding episodes and the risk of joint damage in children with hemophilia A with no preexisting joint damage.

The most serious adverse reactions with Helixate FS are systemic hypersensitivity reactions and the development of high-titer inhibitors necessitating alternative treatments to antihemophilic factor. The most common adverse reactions observed in clinical trials were inhibitor formation in previously untreated or minimally treated patients, skin-associated hypersensitivity reactions, infusion site reactions, and central venous access device (CVAD) line-associated infections.

Helixate FS is contraindicated in patients who have manifested life-threatening immediate hypersensitivity reactions, including anaphylaxis, to the product or its components, including mouse or hamster proteins.

Helixate FS offers convenient administration with a 2.5-mL volume diluent for most assay sizes; no available factor VIII product has a smaller diluent size. No albumin is used in the formulation or purification of Helixate FS, and its manufacture includes a solvent/detergent viral inactivation step.

In 2006, the FDA gave approval for Helixate FS to be stored at room temperature (up to 25°C, 77°F) for three months. The storage guidelines for the hemophilia treatment provide users with greater flexibility and simplify storage options.

Helixate® FS is manufactured by Bayer HealthCare LLC for CSL Behring LLC.