BioMarin withdraws MAA for hemophilia gene therapy following safety, durability concerns

Among CHMP’s monthly roundup of opinions was an MAA withdrawal for BioMarin’s hemophilia gene therapy linked to EMA concerns about safety and durability — issues that FDA raised in August.

EMA said its review could not establish the safety profile of Roctavian valoctocogene roxaparvovec from BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) “due to the limited data available and the short duration of follow-up in treated patients,” nor the duration of benefits. The agency also cited “concerns about the variability of the responses amongst patients, which could not be explained based on the data available.”...