Nov. 8 Quick Takes: FDA request could extend review of BioMarin’s hemophilia therapy

FDA has asked BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) to submit three-year data from the Phase III GENEr8-1 trial as part of the agency’s review of gene therapy valoctocogene roxaparvovec to treat hemophilia A. The biotech said the submission could result in a major amendment that may extend FDA’s review beyond the treatment’s PDUFA date of March 31, 2023. The agency has also scheduled an inspection of BioMarin’s manufacturing facility. The company resubmitted its BLA for the therapy in October following a 2020 complete response letter; EMA has approved valoctocogene roxaparvovec as Roctavian.

Sanofi (Euronext:SAN; NASDAQ:SNY) continues to build out its AI capabilities, this time in a deal with Insilico Medicine Inc. to discover drugs against six targets at the pharma’s China R&D center. Insilico will receive $21.5 million in an upfront payment and target nomination fees and is eligible for R&D and sales milestones of up to $1.2 billion, plus tiered royalties. Sanofi struck a discovery deal of similar size and scope with AI company Atomwise Inc. in August, covering up to five targets for $20 million up front and up to $1 billion total, and the pharma partnered with Owkin Inc. last November in a multi-cancer discovery deal...