BioMarin Pharmaceutical Inc. BMRN announced that the European Commission has granted Orphan Drug designation to BMN 270 for the treatment of patients with hemophilia A.
In the EU, Orphan Drug designation is granted to products targeting a life-threatening or chronically debilitating rare disease that affects up to 5 in 10,000 individuals.
We remind investors that earlier this month, the candidate received an Orphan Drug designation in the U.S. for the same indication.
BioMarin is currently evaluating the safety and efficacy of the candidate in a phase I/II study in patients with severe hemophilia A. Patients will be monitored for safety and durability of effect over a period of five years. The company expects to provide a program update on the study on its R&D day, scheduled to be held next month.
According to the World Federation of Hemophilia, it has been estimated that approximately one in 10,000 individuals is born with hemophilia A.
We note that recently, Bayer AG’s BAYRY Kovaltry was approved in both the U.S. and the EU for the treatment of hemophilia A in children as well as adults.
Another company, Alnylam Pharmaceuticals, Inc. ALNY is developing its hemophilia candidate, fitusiran in a phase I study. The company expects to begin two phase III studies on the candidate for severe hemophilia A and B, with and without inhibitors, in mid and late 2016, respectively.
BioMarin currently has a Zacks Rank #3 (Hold). AMAG Pharmaceuticals, Inc. AMAG, with a Zacks Rank #1 (Strong Buy), is a better-ranked stock in the health care sector.
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