It has proved to be a lackluster summer for the biopharmaceutical sector, with the BioWorld Biopharmaceutical index dropping about 2% in value during the past two months, in contrast to the general markets that have enjoyed a much stronger period.
Biomarin Pharmaceutical Inc.’s complete response letter (CRL) for Roctavian (valoctocogene roxaparvovec; Valrox) gene therapy for severe hemophilia A shocked the company, its investors and analysts mere days before its Aug. 21 PDUFA date. Now an approval and launch for what would have been the first approved hemophilia gene therapy is likely pushed back roughly two years.
