Roctavian (hemophilia drug)

2022: Product Release

On 24 August 2022, BioMarin Pharmaceutical announced that the European Commission had granted marketing authorization for Roctavian gene therapy for the treatment of severe hemophilia A in adult patients with no history of factor VIII inhibitors and no detectable antibodies to adeno-associated serotype 5 virus. The cost of the drug is 1.5 million euros.

The European Commission (EC) also approved the recommendation of the European Medicines Agency (EMA) to keep Roctavian as an orphan drug, which will provide it with a 10-year period of market exclusivity. The EMA recommendation notes that even with existing treatments, the medical drug can provide significant benefit to patients with severe hemophilia A.

The most expensive drug for hemophilia has been released. It costs 1.5 million euros

Single-use infusion is the first approved gene therapy for the treatment of hemophilia A and works by delivering a functional gene that is designed to allow the body to independently produce factor VIII without the need for constant hemophilia prevention. Roctavian facilitates patient treatment time compared to existing treatments. People with hemophilia A have a mutation in the gene responsible for producing factor VIII, the protein needed to coagulate blood.

It is estimated that more than 20 thousand adults suffer from severe hemophilia A in more than 70 countries of Europe, the Middle East and Africa. Out of 8 thousand adults with severe form in 24 countries that are within the range of BioMarin Pharmaceutical, which are subject to EMA permission.

The Commission based its decision on a significant amount of data from the Roctavian Clinical Development Program, the most extensively studied gene therapy for hemophilia A, including two years of results from a global phase 3 GENEr8-1 study. The study demonstrated stable and long-term bleeding control, including reductions in mean annual bleeding rate (ABR) and mean annual VIII factor infusion rate. In addition, the data include five and four years of follow-up of cohorts with doses of 6e13 vg/kg and 4e13 vg/kg, respectively, in the current phase 1/2 dose escalation study.

BioMarin Pharmaceutical pledged to continue working with the general public and EMA to monitor the long-term effects of treatment. Product information will be available in September 2022 on the EMA website under the Medicines tab.^[1]

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