Report Suggests Valoctocogene Roxaparvovec Could Save Hemophilia A Patients Millions Over Lifetime


After 4 years, the 1-time cost of valoctocogene roxaparvovec appeared to be more cost-effective than the annual cost of emicizumab prophylaxis for hemophilia A, according to a new Draft Evidence Report.

The Institute for Clinical and Economic Review (ICER) released a Draft Evidence Report with updated information on the clinical effectiveness and value of valoctocogene roxaparvovec (Roctavian; BioMarin) for hemophilia A.

According to findings from the Draft Evidence Report, valoctocogene roxaparvovec could save a hemophilia A patient more than $4 million throughout their lifetime, while also improving their quality of life.

"BioMarin is pleased that ICER recognizes the potentially transformative impact of [valoctocogene roxaparvovec] as possibly the first gene therapy treatment for severe hemophilia A, and potential to not only deliver profound patient benefit, but also potential long-term health care savings," said Jeff Ajer, executive vice president, chief commercial officer at BioMarin, in a press release.

The Draft Evidence Report compared the 1-time cost of valoctocogene roxaparvovec to the annual cost of emicizumab prophylaxis. Total costs for valoctocogene roxaparvovec include treatment, treatment-related adverse events, treatment for bleeding, arthropathy, surgery, and costs not related to drugs.

Valoctocogene roxaparvovec had an estimated 1-time cost of $2.5 million, compared to a $640,000 yearly cost for emicizumab prophylaxis. ICER found that valoctocogene roxaparvovec saved costs and predict that it will increase quality-adjusted life years.

A single 6e13 vg/kg dose of valoctocogene roxaparvovec was observed to be well tolerated. The most common associated adverse effects (AEs) affected the liver.

Among participants, 80% had increased levels of alanine aminotransferase. Other AEs include elevated aspartate aminotransferase levels (67%), nausea (37%), headache (35%), and fatigue (30%).

Patients with hemophilia A, an X-linked genetic disorder, have blood that does not clot normally because their factor VIII protein does not function properly, which when functioning, will cause blood to clot. The result can be life-threatening blood loss from even minor injuries.

Factor VIII protein levels are less than 1% among patients with the most severe form of hemophilia Am which accounts for nearly 50% of all hemophilia A cases. One of the most common symptoms is spontaneous bleeding into the muscles and joints.

Traditional treatment for hemophilia A includes taking an antibody that mimics the missing protein 1 to 4 times per month. Another common treatment option would be to receive intravenous factor VIII infusions 2 to 3 times per week—though neither work perfectly. Many patients report breakthrough bleeds that can lead to joint damage and lower quality of life.

BioMarin announced plans to offer an outcomes-based warranty for valoctocogene roxaparvovec. This would allow patients to have 4 years of risk sharing.


BioMarin Pharmaceutical Inc., BioMarin's Gene Therapy for Adults with Severe Hemophilia A, ROCTAVIAN™ (valoctocogene roxaparvovec), Assessed to Provide Substantial Cost Savings Per Patient in a Preliminary Independent Report. BioMarin website. September 15, 2022. Accessed on September 19, 2022.,-ROCTAVIAN-TM-valoctocogene-roxaparvovec-,-Assessed-to-Provide-Substantial-Cost-Savings-Per-Patient-in-a-Preliminary-Independent-Report

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