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.
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.
The FDA’s bright line between orphan designation and exclusivity was erased, again, Monday for some drugs when the U.S. Court of Appeals for the District of Columbia denied the agency an en banc rehearing of Eagle Pharmaceuticals Inc. v. Alex Azar.
Regulatory snapshots, including global drug submissions and approvals, clinical trial approvals and other regulatory decisions and designations: Astrazeneca, Emmaus, Immatics, Kimera, Krystal.
The FDA’s go-ahead for Roche Holding AG’s Enspryng (satralizumab-mwge) in anti-aquaporin-4 antibody-positive neuromyelitis optica spectrum disorder had watchers of the space weighing its market odds against two therapies approved earlier: Soliris (eculizumab) from Boston-based Alexion Pharmaceuticals Inc., and the more recently cleared Uplizna (inebilizumab-cdon) from Viela Bio Inc., of Gaithersburg, Md.
Regulatory snapshots, including global drug submissions and approvals, clinical trial approvals and other regulatory decisions and designations: Applied, BTG, Emergent, G1, Henlius, Immunomedics, Mustang, Myovant, PTC, Roche, Tracon, Trinity, United.
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