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.
Regulatory snapshots, including global drug submissions and approvals, clinical trial approvals and other regulatory decisions and designations: Biomarin, Cannformatics, Galapagos, Mylan, Precision, Sorrento.
The FDA granted accelerated approval to Viltepso (viltolarsen) to treat Duchenne muscular dystrophy patients (DMD) who have a confirmed mutation of the DMD gene amenable to exon 53 skipping, pitting it against Sarepta Therapeutics Inc.’s Vyondys (golodirsen), which was given accelerated approval last year in a controversial decision by the FDA following an agency complete response letter (CRL) highlighting safety concerns.
The latest global regulatory news, changes and updates affecting biopharma, including: Medicines and Healthcare Products Regulatory Agency, Moderna, Office of the U.S. Trade Representative, Pan American Health Organization, SEC, Therapeutic Goods Administration, World Health Organization.
Regulatory snapshots, including global submissions and approvals, clinical trial approvals and other regulatory decisions and designations: Clearmask, Diazyme, Genosity, Illuminoss, Sensiva.
Following a patient’s death in Poseida Therapeutics Inc.’s phase I trial of P-PSMA-101 in metastatic castrate-resistant prostate cancer (mCRPC), the FDA has put the study on clinical hold.
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