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Another Gene Tx Breakthrough – Roctavian

Another sonic boom could be heard (well….. at least by dogs) recently when the FDA approved yet another gene therapy! This time the news was truly noteworthy, as the therapy in question has been the aspirational hope of a well known, rare disease population for neigh on three decades. The approved therapy is Roctavian (valoctocogene roxaparvovec-rvox) from BioMarin Pharmaceutical, a gene therapy for the treatment of adults with severe hemophilia-A (congenital factor VIII (FVIII) deficiency with FVIII activity < 1 IU/dL) without antibodies to adeno-associated virus serotype 5 (AAV5) detected by an FDA-approved test.

Roctavian  is a one-time, single-dose infusion and  is the first approved gene therapy for severe hemophilia A in the U.S. 

BioMarin said it estimates only about 2,500 US patients would be eligible for the therapy.  Patients with certain underlying health conditions and problems are excluded from the prescribing information.  The company went on to explain that Roctavian was approved only for patients with severe disease who test negative for an antibody that attacks the virus Roctavian uses to deliver its helpful genetic material into cells. Upwards of 20,000 males in the US have hemophilia-A meaning that about 17,000 people are not candidates for this particular one-and-done therapy. However, this approval only fuels the hopes of the remaining hemophilia-A candidates.

CLICK HERE to access prescribing information

BioMarin claims that Roctavian will come to market at $2.9 million….. which is arguably not expensive when annual costs for traditional blood factors can run as much as $800,000 annually for a typical patient. They also pointed out that the price is less than the $3.5 million for a similar gene therapy for hemophilia-B approved last fall.

BioMarin did not announce details for logistics and distribution.


BioMarin finally secures FDA approval of hemophilia gene therapy

After a prolonged journey, the medicine, known as Roctavian, is now cleared for certain patients with hemophilia A, the more common form of the rare bleeding disorder.

June 29, 2023 — The Food and Drug Administration approved on Thursday the first gene therapy for the most common form of hemophilia, clearing the way for what patients, doctors and the medicine’s developer hope could be a one-time treatment for the rare bleeding disorder.

BioMarin Pharmaceutical, the California-based company behind the therapy, plans to sell it under the brand name Roctavian. It’s specifically meant to treat hemophilia A, which is caused by genetic mutations that inhibit the production of a key blood-clotting protein known as Factor VIII.

The company has set a list price of $2.9 million in the U.S., higher than the therapy’s cost in Europe, where it’s priced at about………………article continues

CLICK HERE to access the full article


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