Inspection of Roctavian Facility Set; FDA Seeks More Trial Data

Agency is reviewing BioMarin's BLA to treat adults with severe hemophilia A

Marisa Wexler, MS avatar

by Marisa Wexler, MS |

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The U.S. Food and Drug Administration (FDA) has scheduled its inspection of the facility in California where BioMarin Pharmaceutical would produce the gene therapy Roctavian (valoctocogene roxaparvovec) if it’s approved.

The agency is reviewing a biologics license application (BLA) from BioMarin that seeks approval of Roctavian to treat adults with severe hemophilia A.

“We appreciate the level of engagement from FDA this early in their BLA review cycle and are pleased to share that the inspection of our gene therapy manufacturing facility has now been scheduled,” Hank Fuchs, MD, president of worldwide research and development at BioMarin, said in a press release.

Hemophilia A is caused by mutations in the gene that provides instructions for making the clotting protein factor VIII (FVIII). Roctavian uses a viral vector to deliver a healthy version of this gene to cells in the liver, allowing the body to produce a functional version of the clotting protein.

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BioMarin’s BLA is supported by data from the Phase 3 GENEr8-1 trial (NCT03370913), wherein 134 men with severe hemophilia A were treated with a single dose of Roctavian. After two years, 80% were bleed-free and most were able to stop taking standard replacement therapies.

The FDA also has asked BioMarin to submit a three-year data analysis from the ongoing trial. This was expected, according to BioMarin.

“With their request related to the upcoming three-year data analysis from our Phase 3 GENEr8-1 study, FDA stated that these data are expected to provide longer-term efficacy and safety information and would thus be useful to people with hemophilia A and healthcare providers in making better and more informed decisions when considering [Roctavian] as a treatment choice should it be approved,” Fuchs said.

The FDA’s decision on Roctavian is expected by the end of March, but the decision may be extended by three months if the additional data is deemed a major amendment, according to BioMarin.

The FDA has not indicated to expect a change, however.

“We are encouraged by the bleed control results observed in the first cohort of patients who reached three years of observation in the Phase 3 study as shared in January, and we look forward to reviewing the three-year results from all participants. We anticipate sharing the three-year results from all 134 participants in our Phase 3 study in early 2023,” Fuchs said.

The FDA also announced plans to hold an advisory meeting where a group of experts will convene to review the data. The meeting is not yet scheduled.

“Depending on the timing of this meeting, BioMarin looks forward to the opportunity to discuss the recently requested 3-year data analysis with the Advisory Committee,” the company said.

Roctavian has recently gained conditional approval in the EU for treating adults with severe hemophilia A who have neither inhibitors nor detectable antibodies against the viral vector the gene therapy uses to deliver the corrected gene to patients’ cells.