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BioMarin Announces RMAT Designation Granted to Valoctocogene Roxaparvovec for Hemophilia A americanpharmaceuticalreview
March 11, 2021
BioMarin Pharmaceutical announced the U.S. Food and Drug Administration (FDA) granted Regenerative Medicine Advanced Therapy (RMAT) designation to valoctocogene roxaparvovec, an investigational gene therapy for the treatment of adults with severe ...
USFDA grants FTD to efanesoctocog alfa for hemophilia A treatment pharmaceutical-business-review
February 25, 2021
The U.S. Food and Drug Administration (FDA) has granted efanesoctocog alfa – earlier termed BIVV001 (rFVIIIFc-VWF-XTEN) – Fast Track Designation (FTD) for the treatment of patients suffering from hemophilia A.
Pfizer, Sangamo announce updated results of phase 1/2 Alta study of giroctocogene fitelparvovec pharmaceutical-business-review
December 09, 2020
Pfizer and Sangamo Therapeutics, a genomic medicines company, announced updated follow-up data from the Phase 1/2 Alta study of giroctocogene fitelparvovec (SB-525 or PF-07055480), an investigational gene therapy for patients with severe hemophilia A.
BioMarin Receives CRL for Valoctocogene Roxaparvovec Gene Therapy for Severe Hemophilia A americanpharmaceuticalreview
August 21, 2020
BioMarin Pharmaceutical announced the U.S. Food and Drug Administration (FDA) issued a Complete Response Letter (CRL) to the Company's Biologics License Application (BLA) for valoctocogene roxaparvovec gene therapy for severe hemophilia A.
Induced pluripotent stem cells show success in treating hemophilia A in mice expresspharma
March 13, 2020
In a new study released in STEM CELLS Translational Medicine (SCTM), researchers at the University of California, Davis and the Chinese Academy of Science demonstrate how induced pluripotent ...
Valoctocogene Roxaparvovec BLA Accepted for Priority Review by FDA americanpharmaceuticalreview
March 09, 2020
BioMarin Pharmaceutical announced the U.S. Food and Drug Administration (FDA) has accepted for Priority Review the Biologics License Application (BLA) to the FDA for its investigational AAV5 gene therapy ...
Spark Therapeutics Announces Updated Data on SPK-8011 from Ongoing Phase 1/2 Dose-escalation Clinical Trial in Hemophilia A at 60th American Society of Hematology (ASH) Annual Meeting and Exposition firstwordpharma
December 03, 2018
Among the five participants treated in the 2x1012 vg/kg cohort who did not experience an immune response-associated decline in FVIII expression, beginning four weeks after vector infusion, there has been a 100-percent reduction in bleeds and a greater tha
Precision BioLogic Presents New Approach to Standardize and Improve Inhibitor Testing for People wit biospace
March 14, 2018
Precision BioLogic cooperates with Roche and Genentech under the Roche Group
Shire Granted EU Marketing Authorization for ADYNOVI [Antihemophilic Factor (Recombinant), Pegylated biospace
January 16, 2018
Shire plc, a world leader in disease biotechnology, today announced that the European Commission has granted marketing authorization to ADYNOVI [Antihemophilic Factor (Recombinant), PEGylated]
Shire, Rani to develop oral factor VIII therapy pharmatimes
December 07, 2017
Shire and Rani Therapeutics have signed a deal to work on developing an orally-delivered therapy for patients with hemophilia A.

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