Accelerated approval of BioMarin’s dwarfism drug clarifies endpoint

BioMarin added nearly $1.6 billion in market cap Friday after FDA granted accelerated approval to its therapy for achondroplasia, resolving some uncertainty around the agency’s opinion of endpoints measuring growth velocity and final adult height.

As recently as last month, at the Jefferies Gene Therapy/Editing Summit, Henry Fuchs, president of worldwide R&D at BioMarin Pharmaceutical Inc. (NASDAQ:BMRN), acknowledged it was unclear which direction FDA would take, suggesting either accelerated or full approval was possible based on the Phase III endpoint of annualized growth velocity (AGV)...