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BioMarin Receives Approval for First Gene Therapy for Adults With Severe Hemophilia A


Approximately 2500 individuals in the United States could be eligible to receive valoctocogene roxaparvovec.

FDA has approved BioMarin’s gene therapy, valoctocogene roxaparvovec, to treat certain adults with severe hemophilia A, as determined by FDA-approved testing. The therapy, to be sold as Roctavian, was approved last year by the European Medicines Agency.

valoctocogene roxaparvovec

valoctocogene roxaparvovec

About 6500 adults in the United States have severe hemophilia A, and BioMarin said in a statement it expects approximately 2500 of them to be eligible for the gene therapy with this approval. Information from the company states that the treatment is approved for adults with hemophilia A, “congenital factor VIII (FVIII) deficiency with factor VIII activity < 1 IU/dL), without antibodies to adeno-associated virus serotype 5 (AAV5) detected by an FDA-approved test.”

Valoctocogene roxaparvovec is a single-dose treatment given one time for patients with a disease characterized by uncontrolled bleeding. Approval was based on results for safety, efficacy, and durability in the global phase 3 GENEr8-1 study, which evaluated 134 patients who received the gene therapy. Of this group, 112 patients had baseline annualized bleeding rate (ABR) data prospectively collected for 6 months on FVIII prophylaxis prior to receiving the study therapy; this group had a mean 52% ABR reduction after treatment. The remaining 22 patients had baseline ABR collected retrospectively. All patients were followed for at least 3 years.

The study also found a substantial reduction in the rates of spontaneous bleeds and joint bleeds following treatment.

"Adults with severe hemophilia A face a lifelong burden, with frequent infusions and a high risk of health complications, including uncontrolled bleeding and irreversible joint damage," said Steven Pipe, professor of pediatrics and pathology at the University of Michigan and an investigator in the phase 3 study. Approval of the gene therapy, he said, “has the potential to transform the way we treat adults based on years of bleed control following a single, one-time infusion."

BioMarin officials announced in a conference call that the one-time infusion would be $2.9 million; with discounts it will net closer to the $1.9 million deemed "acceptable" in a review by ICER. In Europe, it costs €1.5 million, or about $1.6 million.

In a email regarding the list price, Biomarin officials said, "For the healthcare system, [the treatment] adds value in terms of both the clinical benefit it offers patients and the economic benefit it offers against the very high cost of standard-of-care therapy, which is about $800,000 a year for the typical patient."

Of interest to payers, the company is offering an outcomes-based warranty, which will be offered to all US insurers. The warranty will reimburse government and commercial payers up to 100% of wholesale acquisition cost in the event that a person does not respond to therapy.

“If an individual treated with Roctavian loses response at any time in the first 4 years after dosing, BioMarin will reimburse payers on a prorated basis for the cost of treatment,” the company promised in its statement announcing the approval.

Hemophilia A occurs in patients with a mutation in the gene that produces FVIII, the protein that causes blood clotting. This lifelong, genetic condition puts patients at risk of life-threatening bleeds, which can happen without warning. The disease is also painful, requiring patients to receive lifelong preventive therapy, including infusions or injections at regular intervals to keep clotting factor in the bloodstream.

With valoctocogene roxaparvovec, that could all change. As described in BioMarin’s statement, the gene therapy “is designed to replace the function of the mutated gene, allowing people with severe hemophilia A to produce their own FVIII and thereby limit bleeding episodes.”



"We are proud to now offer adults with severe hemophilia A, a one-time, single-dose treatment option,” said Jean-Jacques Bienaimé, chairman and CEO of BioMarin, in the statement. “We are especially grateful to the bleeding disorders community for its support of this program, and to all the patients and healthcare providers who participated in our clinical trials."

The therapy will be manufactured at the company's facility in Novato, California.

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