2022-06-27| Trials & Approvals

BioMarin Wins First Gene Therapy Recommendation For Hemophilia A in Europe

by Joy Lin
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The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has given a positive opinion recommending conditional marketing authorization for BioMarin’s gene therapy Roctavian, also known as valoctocogene roxaparvovec. This makes BioMarin’s gene therapy the first to be recommended for approval in Europe for Hemophilia A. 

The gene therapy is expected to get the marketing greenlight from the European Commission in the third quarter of this year, said BioMarin. 


Potential Gene Therapy for Hemophilia A


Hemophilia A is a genetic disorder caused by missing or defective congenital factor VIII (FVIII), a clotting protein. It affects an estimated 20,000 adults across Europe, the Middle East, and Africa.

Roctavian is a one-time infusion intended to treat severe hemophilia A in adults by restoring the normal expression and function of the missing clotting factor. The drug will be given to patients without a history of FVIII inhibitors and without detectable antibodies to adeno-associated virus stereotype 5 (AAV5), the viral vector that delivers the gene therapy into cells. 

BioMarin believes the drug will be made available to patients outside Europe (e.g. the Middle East and Africa) based on an EMA approval. 

Related article: Roe Reversal May Cause Downstream Issues in Repurposing Mifepristone


Multiple Studies for Safety and Efficacy of Roctavian


The positive CHMP opinion is drawn from the results of the Roctavian gene program for hemophilia A. This includes two-year outcomes from the Global GENEr8-1 Phase 3 study, supported by five and four years of follow up from the 6e13vg/kg and 4e13vg/kg dose cohorts in the Phase 1/2 dose escalation study.

In the pivotal GENEr8-1 study, the annualized bleeding rate was significantly reduced by 85% from baseline and was superior to FVIII enzyme replacement therapy, the standard of care.  

In general, the higher dose of Roctavian has been well-tolerated. Adverse events occurred most commonly early on and included temporary mild to moderate rises in liver enzymes with no long-lasting clinical consequences. The most common side effect is alanine aminotransferase (ALT) elevation, determined by a laboratory test of liver function. Other side effects include aspartate aminotransferase (AST) elevation, nausea, headache, and fatigue. 

A full approval from the EU will rely on Roctavian’s performance in other ongoing studies, including a longer term follow up for BioMarin’s pivotal trial GENEr8-1 and a study of corticosteroids. The latter is a Phase 3B single arm, open-label study evaluating Roctavian at a 6e13vg/kg dose with prophylactic corticosteroids in patients with hemophilia A.


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