BioMarin receives FDA update for gene therapy for adults with severe haemophilia A

pharmafile | March 7, 2023 | News story | Medical Communications  

Global biotechnology company BioMarin Pharmaceutical has announced that it’s received an update from the FDA, detailing the extension of their review of the company’s Biologics License Application (BLA) for Roctavian™.


Roctavian™ (valoctocogene roxaparvovec) is a gene therapy for adults with severe haemophilia A, an X-linked genetic disorder caused by missing or defective Factor VIII ‒ a clotting protein.


In 2021, the FDA granted Regenerative Medicine Advanced Therapy (RMAT) designation to valoctocogene roxaparvovec, following the company’s Breakthrough Therapy designation in 2017. Valoctocogene roxaparvovec has also received Orphan Drug Designation (ODD) from the EMA and FDA. The EC granted conditional marketing authorisation for valoctocogene roxaparvovec under brand name Roctavian in mid-2022.


The FDA’s recent decision is based on the ongoing GENEr8-1 phase 3 study which is the largest and longest study to date in gene therapy for haemophilia, involving 134 patients. A three-year data analysis prompted a Major Amendment to its review due to the amount of additional data, with a new PDUFA Target Action Date of 30 June 2023 being established.


“We are continuing to work closely with FDA and appreciate the agency’s active engagement as we seek to deliver this important therapy to patients with severe haemophilia A. The three-year data enhances our application and further reinforce our belief that Roctavian has the potential to fundamentally transform care for people with haemophilia A,” Hank Fuchs MD, president of Worldwide Research and Development of BioMarin.


James Spargo

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