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Sanfilippo Syndrome: BioMarin Pharmaceutical's FDA Orphan Drug Designation

BioMarin Pharmaceutical receives FDA Orphan Drug Designation (ODD) on November 25th for its investigational Enzyme Replacement Treatment (ERT), BMN-250, for the treatment of Mucopolysaccharidosis III Type B (MPS IIIB, Sanfilippo Syndrome Type B). 164 more words

Orphan Drug Development

Rare Disease Gene Therapy Pricing: uniQure's Glybera Sets A Record High

No gene therapy has ever been approved for sale in the United States. In Europe, the EMA (European Medicines Agency) gives regulatory approval in October 2012 to Dutch biotech… 322 more words

Orphan Drug Development

Two years and one month later

Two years and one month ago, I was in a hospital room. My mom and step-dad were sleeping and I was in pain and overly frustrated. 455 more words


More Change

When I was diagnosed with brain tumors and knew I was going to have to have brain surgery, my biggest fear was that I would wake up from the surgery… 367 more words

Rare Disease

Europe’s November 2014 Products Recommended For Orphan Drug Designation

The European Medicines Agency (EMA) Committee for Orphan Medicinal Products (COMP) held a meeting November 11– 13, 2014.

At this meeting, there are 27 positive opinions recommending the following medicines for designation as orphan medicinal products. 464 more words

Orphan Drug Development

Introducing 1st Rare Disease Advocacy World (RDAW) USA 2015 Meeting

Patient advocacy groups are one of the most important powerful stakeholders that contribute to the success of orphan drug development and changes in policies for global regulatory agencies. 354 more words

Orphan Drug Development

Ultra-Rare Diseases: BioBlast Pharma Orphan Drug Designations

BioBlast Pharma, a Tel Aviv, Israel-based clinical-stage biotech founded in 2012, is developing a platform of products for rare and ultra-rare genetic diseases.

I – Cabaletta For Oculopharyngeal Muscular Dystrophy (OPMD) 329 more words

Orphan Drug Development