FDA Approves BioMarin's Roctavian for Treatment of Severe Hemophilia A


Valoctocogene roxaparvovec-rvox (Roctavian; BioMarin) is a one-time gene therapy product administered as a single dose by intravenous infusion for hemophilia A.

The FDA approved valoctocogene roxaparvovec-rvox (Roctavian; BioMarin Pharmaceutical Inc) for the treatment of severe hemophilia A in adults without pre-existing antibodies to adeno-associated virus serotype 5 detected by an FDA-approved test.1

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Valoctocogene roxaparvovec-rvox is an adeno-associated virus vector-based gene therapy and is a one-time product administered as a single dose by intravenous infusion.1

“Hereditary hemophilia A is a potentially serious bleeding disorder. Severe cases of hemophilia A can cause life-threatening health issues due to increased risk of uncontrolled bleeding,” Peter Marks, MD, PhD, director of the FDA’s Center for Biologics Evaluation and Research, sad in a statement. “Today’s approval represents an important advance in providing treatment options for patients with this bleeding disorder, and treatment with gene therapy may reduce the need for ongoing routine therapy.”1

The gene expressed in valoctocogene roxaparvovec-rvox increases blood levels of Factor VIII and reduces the risk of uncontrolled bleeding.1

The approval was based on the global phase 3 GENEr8-1 study (NCT03370913), the largest phase 3 trial of any gene therapy.2

The safety and efficacy of valoctocogene roxaparvovec-rvox were evaluated in a multinational study in men aged 18 to 70 years of age who had hemophilia A and were previously treated with Factor VIII replacement therapy. The effectiveness was based on results from a study with 112 individuals for at least 3 years after treatment with valoctocogene roxaparvovec-rvox.1

Furthermore, the results from the 3-year analysis of the phase 3 study showed that the individuals had an 82.9% reduction in treated bleeds overall compared with the baseline and led to a 96.8% reduction in Factor VIII usage overall when compared with baseline.2

After infusion, investigators found that the mean annualized bleeding rate decreased from 5.4 bleeds per year at baseline to 2.6 bleeds per year. Additionally, the majority of individuals who received valoctocogene roxaparvovec-rvox received corticosteroids to suppress the immune system, making the gene therapy effective and safe.1

Investigators said that treatment responses with valoctocogene roxaparvovec-rvox may decrease over time.1

The most common adverse events with valoctocogene roxaparvovec-rvox included mild changes in liver function, headache, nausea, vomiting, fatigue, abdominal pain, and infusion-related reactions. The FDA recommends close monitoring for infusion-related reactions and liver enzyme elevation with administration.1

Additionally, in some cases, treatment did increase Factor VIII activity levels above the normal limits.

However, there were no instances of thromboembolic events or cancers associated with valoctocogene roxaparvovec-rvox in clinical trials.1

Previously, the application of the drug received orphan, breakthrough therapy, regenerative medicine advanced therapy, and priority review designations.1

The drug was approved by the European Medicines Agency in August 2022.2


  1. FDA approves first gene therapy for adults with severe hemophilia A. News release. US Food and Drug Administration. June 29, 2023. Accessed June 30, 2023. https://www.fda.gov/news-events/press-announcements/fda-approves-first-gene-therapy-adults-severe-hemophilia
  2. US Food and Drug Administration approves BioMarin’s Roctavian (valoctocogene roxaparvovec-rvox), the first and only gene therapy for adults with severe hemophilia A. News release. PR Newswire. June 29, 2023. Accessed June 30, 2023. https://www.prnewswire.com/news-releases/us-food-and-drug-administration-approves-biomarins-roctavian-valoctocogene-roxaparvovec-rvox-the-first-and-only-gene-therapy-for-adults-with-severe-hemophilia-a-301867403.html
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