Tags » Rare Disease
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
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
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