BioMarin’s Roctavian (valoctocogene roxaparvovec) Receives EC’s Conditional Marketing Authorization for Severe Hemophilia A
Shots:
- The EC has granted CMA to Roctavian for sev. hemophilia A in adult patients without a history of factor VIII inhibitors & without detectable Ab to AAV5
- The EC’s decision was based on the clinical development program incl. 2yr. results from the P-III (GENEr8-1) study showed a stable & durable bleed control with a reduction in mean ABR & mean annualized factor VIII infusion rate
- In the ongoing P-I/II dose escalation study, the results included 5 & 4yr. of follow-up from the 6e13 & 4e13vg/kg dose groups while 6e13vg/kg dose was well tolerated with no delayed-onset TRAEs. Roctavian continues to be made available to eligible patients with sev. haemophilia A & the company is expected to resubmit the BLA at the end of Sept. 2022
Ref: BioMarin | Image: BioMarin
Click here to read the full press release
Tags
Neha is a Senior Editor at PharmaShots. She is passionate and very enthusiastic about recent updates and developments in the life sciences and pharma industry. She covers Biopharma, MedTech, and Digital health segments along with different reports at PharmaShots. She can be contacted at connect@pharmashots.com.
