FDA approves Roctavian, the First Gene Therapy for Hemophilia A

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The FDA approved Roctavian (valocotogene roxaparvovec) today. It is the first gene therapy for hemophilia A, the most common form of the disease.

The therapy’s maker, BioMarin Pharmaceutical, is pricing the therapy at $2.9 million for a one-time treatment. The gaudy price tag is striking, but multimillion dollar prices for the growing number of gene therapies are common. The manufacturers and independent cost-effectiveness research groups say the seven-figure prices are justified, in part because patients will avoid the costs of lifelong treatments with other therapies. Hemophilia A is a hereditary disorder caused by a lack of blood clotting factor VIII, and patients with severe cases receive frequent infusions of clotting factors.

There are lingering questions, though, about how “durable” the supposedly one-time gene therapies are and whether patients will need eventually need to resume infusions of clotting factors or be treated in other ways. Payers, academic and cost-effectiveness experts have suggested that insurers tie payment for the gene therapies to measurable outcomes, which in the case of hemophilia might be how long the patient doesn’t need clotting factor infusions.

BioMarin is setting up an outcomes program for Roctavian that will reimburse payers up to 100% of cost if person does not respond to treatment. Part of the cost will be reimbursed on prorated basis if the patient stops responding after the first four years.

The FDA approved Hemgenix (etranacogene dezaparvovec), another gene therapy for hemophilia, in November 2022, and its manufacturer, CSL Behring in suburban Philadelphia, priced it at $3.5 million. The company announced on June 20 that the first patient had been treated with Hemgenix, a treatment for hemophilia B, which is caused by a lack of a different clotting factor, factor IX, than hemophilia A and is rarer. The FDA press release announcing the approval of Hemgenix said that about 15% of patients with hemophilia have hemophilia B; the Centers for Disease and Control says that there are about 30,000-33,000 males living with the disease. Hemophilia predominately affects men.

Pfizer issued a press release on Tuesday (June 27) that the FDA had accepted its application for approval of its hemophilia B gene therapy, called fidanacogene elaparvovec.

Related: Gene Therapies for Hemophilia: Promising But There’s Room for Improvement, Say Reviewers

The FDA approval of Roctavian was expected, although path thorough the FDA approval process has been long and tortuous and included a 2020 complete response letter. Most recently, the agency delayed its decision by three months to review more data submitted by BioMarin.

The agency’s approval is based on results of the BioMarin-sponsored Gene8-1study, an open-label study that included 134 participants who received a Roctavian solution. The results, which were reported in the New England Journal of Medicine in March, showed significant increases in clotting factor activity — how well the factor VIII is working to clot blood, according to a standard test at about a year after the infusion. The results also showed fewer instances of bleeding and less need for factor VIII among a sizable (112) subset of patients. The most common side effect was elevated alanine aminotransferase levels, which can signal liver or kidney damage. Patients were treated with glucocorticoids.