Decision on hemophilia A gene therapy Roctavian set for summer

FDA extended its review of BioMarin's clinical trial data

Marisa Wexler, MS avatar

by Marisa Wexler, MS |

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The U.S. Food and Drug Administration (FDA) is extending its review of BioMarin Pharmaceutical’s request to approve the gene therapy Roctavian (valoctocogene roxaparvovec) for adults with severe hemophilia A.

A decision had been expected by the end of this month, but that date has now been pushed to June 30. The FDA extended its review to have more time to go over new clinical trial data submitted by BioMarin.

“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 hemophilia A,” Hank Fuchs, MD, president of worldwide research and development at BioMarin, said in a company press release.

Hemophilia A is caused by mutations in the gene that provides instructions for making a clotting protein called factor VIII (FVIII). Roctavian is made to deliver a functional version of this gene to cells in the liver, where most clotting factor proteins are made, in order to restore FVIII production. It was granted conditional approval in Europe last year.

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BioMarin’s application is supported by data from the Phase 3 GENEr8-1 trial (NCT03370913). The study enrolled 134 men with severe hemophilia A, defined as FVIII activity levels lower than 1% of normative values. All the participants received a single dose of Roctavian, delivered via an infusion directly into the bloodstream.

Top-line data from the study suggested treatment reduced bleeding rates. At two years, more than 80% of patients were bleed-free without needing prophylactic therapies to prevent bleeding.

BioMarin recently announced three-year data from GENEr8-1, which continued to suggest Roctavian led to a long-term decrease in bleeding rates for most men with severe hemophilia in the trial. With more than 90% of patients still off preventive therapies, most haven’t had any clinically relevant bleeds three years after receiving gene therapy and median FVIII activity is at more than 8% of normal values.

Three-year data were recently submitted to the FDA, and the agency has extended its review of the application to have time to go over these new long-term findings.

“The three-year data enhance our application and further reinforce our belief that Roctavian has the potential to fundamentally transform care for people with hemophilia A,” Fuchs said.

The FDA has granted Roctavian several designations to incentivize its development and speed up its regulatory review, including regenerative medicine advanced therapy (RMAT), breakthrough therapy, and orphan drug status. Roctavian also has been designated an orphan drug in Europe.