BioMarin Announces Delay in Application to FDA

Roctavian is Biomarin’s investigational hemophilia A gene therapy, which is currently in multiple ongoing clinical trials.

BioMarin recently announced that their planned timeline for the resubmission of a Biologics License Application to the U.S. Food and Drug Administration (FDA) for valoctocogene roxaparvovec, also known as Roctavian, has been pushed back to the end of September 2022 – the initial target had been June.

Roctavian is Biomarin’s investigational hemophilia A gene therapy, which is currently in multiple ongoing clinical trials to observe its safety and efficacy at various dose levels in adult patients with severe hemophilia A.

While this shift was prompted by an FDA request to Biomarin for additional information, no specifics were provided. The company did however note that the agency did not ask for new preclinical or clinical studies. This delay represents the second in as many years. In August of 2020 the FDA withheld approval for the therapy and requested further safety and efficacy data.

In a new press release, the company also provided updated phase 1/2 trial results showing significant reductions in mean annualized bleed rates and factor VIII replacement therapy usage. BioMarin plans to share this and other data at next month’s International Society on Thrombosis and Haemostasis 2022 Congress, which will be held July 9th-13th in London.

“We continue to work with regulatory authorities to potentially make available the first gene therapy in severe hemophilia A as an important treatment option, said Hank Fuchs, MD, President, Worldwide Research and Development at BioMarin. “The thorough feedback recently provided by the FDA increases our ability to support the FDA’s review of our application and hopefully makes Roctavian available as a choice for patients in the United States,”

NHF will continue to share relevant updates as they become available.

Source: BioPharma Dive and BioMarin press release, both dated May 31, 2022