Roctavian, Hem A Gene Therapy, Given Conditional Approval in EU

Commission's OK is based on clinical trials of patients with severe conditions

Marisa Wexler, MS avatar

by Marisa Wexler, MS |

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The European Commission has granted conditional marketing authorization to BioMarin’s one-time gene therapy Roctavian (valoctocogene roxaparvovec) as a treatment for adults with severe hemophilia A who have neither inhibitors nor detectable antibodies against adeno-associated virus serotype 5 (AAV5).

The decision, which comes a couple of months after a positive recommendation from a committee of the European Medicines Agency (EMA), makes Roctavian the first gene therapy for hemophilia A authorized in the EU. The therapy is now expected to be available for an estimated 3,200 patients across 24 European countries.

“Roctavian approval in Europe is a historic milestone in medicine and is built upon almost four decades of scientific discovery, innovation, and perseverance,” Jean-Jacques Bienaimé, BioMarin chairman and CEO, said in a press release.

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“This approval in the EU represents a medical breakthrough in the treatment of patients with severe hemophilia A that expands the conversation between a patient and physician on treatment choices to now include a one-time infusion that protects from bleeds for several years,” said Johannes Oldenburg, director of the Institute of Experimental Haematology and Transfusion Medicine and the Haemophilia Centre at the University Clinic in Bonn, Germany.

Conditional marketing authorization is given to therapies that are expected to fill an unmet medical need, where the potential benefits of making the therapy available immediately outweigh the uncertainty due to a need for additional clinical data. BioMarin announced it plans to provide further data as it becomes available. Conversion to a full marketing authorization would be contingent on the provision of additional data from ongoing studies.

The European Commission has also endorsed the EMA’s decision to give Roctavian orphan drug designation, granting 10 years of guaranteed market exclusivity to the therapy.

“We thank the European Commission for recognizing Roctavian’s value as the first gene therapy for hemophilia A, a feat that we believe will transform how healthcare professionals and the patient community think about caring for bleeding disorders,” Bienaimé said.

Hemophilia A is caused by mutations in the gene that provides instructions for making a clotting protein called factor VIII (FVIII). Roctavian is designed to deliver a functional copy of this gene to the body’s cells using a harmless AAV5 viral vector, and thereby restoring FVIII production.

Clinical trial support

Roctavian’s conditional approval was supported largely by data from the global Phase 3 GENEr8-1 trial (NCT03370913), in which 134 men with severe hemophilia A were treated with a single infusion of Roctavian (6×10e13 vector genomes per kilogram). Findings from the ongoing trial showed that more than 80% of patients were bleed-free after two years, and most were off standard replacement therapies.

The approval was also supported by data from the Phase 1/2 study (NCT02576795), which enrolled 15 men with severe hemophilia A. Data from this ongoing trial showed the gene therapy was able to reduce bleed rates and the use of FVIII replacement therapies for up to six years. Other trials of Roctavian are also ongoing.

“It is exciting to imagine the possibilities of this approved gene therapy, which has demonstrated a substantial and sustained reduction in bleeding for patients, who potentially could be freed from the burden of regular infusions,” Oldenburg said.

BioMarin had originally applied for Roctavian’s approval in the U.S. and EU in 2020, based on six-month interim data from GENEr8-1 and three-year data from the Phase 1/2 trial. Regulators rejected the initial applications, citing a need for more data.

BioMarin plans to resubmit an application next month to the U.S. Food and Drug Administration (FDA) requesting Roctavian’s approval in the U.S. The company anticipates it will take nine months for the FDA to review the application.

“We are grateful to the patients, investigators and community, who dedicated their time and effort to this achievement and whose aspirations provided the driving force behind making this one-time therapy a reality,” Bienaimé said.