Feb. 20 Product Development Quick Takes: Priority Review puts BioMarin on track to score first hemophilia gene therapy approval in U.S.; plus Gilead-CDC, Genfit
BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) could become the first company to have a hemophilia gene therapy approved in the U.S. FDA accepted and granted Priority Review to a BLA for valoctocogene roxaparvovec to treat severe hemophilia A, giving the candidate an Aug. 21 PDUFA date. The gene therapy -- which comprises an AAV5 vector delivering Factor VIII -- is also under accelerated assessment in the EU. At least four other hemophilia A gene therapies are in clinical testing.
Gilead has now been denied inter partes review of all four of the CDC's PrEP patents covering HIV drugs Truvada emtricitabine/tenofovir and Descovy emtricitabine/tenofovir alafenamide. USPTO's Patent Trial and Appeal Board (PTAB) said in its decisions this month that Gilead Sciences Inc. (NASDAQ:GILD) has not established a reasonable likelihood of prevailing on its assertion that the CDC's patents are invalid. Separately, CDC filed suit in November seeking royalties and damages from Gilead, asserting that Truvada and Descovy infringe on the agency's PrEP patents (see "Gilead Claims CDC's PrEP Patents are Invalid")...