Approved by the European Commission (EC) in August 2022, valoctocogene roxaparvovec is also poised to be the first gene therapy to launch in the United States for severe hemophilia A. Treatment benefit is expected to last for years, reduce the number of bleeding events, minimize the need for replacement factor VIII (FVIII) and negate the use of otherwise burdensome prophylaxis treatment.
• Marketing authorization: EC
• Marketing authorization for AAV5 DetectCDx™ Kit companion diagnostic: EC
• Biologics License Application (BLA) resubmitted: U.S. FDA
March 31, 2023:
• PDUFA date
Actual and expected launch:
• 2022: Europe
• 2023: United States
Patents estimated to expire beginning in 2033
How will valoctocogene roxaparvovec impact the market for hemophilia A?
What gaps in treatment does valoctocogene roxaparvovec fill?
Approximately 43% of people with hemophilia A experience painful, spontaneous bleeds into their muscles and joints that contribute to progressive, debilitating joint damage that can have a major impact on quality of life. Standard of care consists of 100 to 150 intravenous infusions (two to three times a week) of replacement FVIII a year and does not always prevent joint damage. Correction of the coagulation system can be life-changing, and valoctocogene roxaparvovec could potentially be the first treatment to accomplish this with a single infusion, eliminating the need for blood transfusions and FVIII replacement therapy.
What hurdles might it need to overcome to reach blockbuster status?
There are a number of factors that could influence whether valoctocogene roxaparvovec will be broadly adopted within the target population including the presence of safe, efficacious therapies; use restricted to patients older than 18 years; and loss of FVIII expression over time, as observed in clinical trials. In addition, the combination of this loss of efficacy, unpredictable and variable individual responses to the treatment and a lack of long-term safety data might make both patients and physicians hesitant to use, and payers hesitant to cover, a novel gene therapy. Coverage could depend on a set of predetermined conditions, and the patient copay might be high.
“We put the $2-3 million in the context of how much uncertainty exists around how much we’re currently paying in factor. If the return on investment takes two to three years (that is, BMN 270 really works) and we don’t need any factor VIII over at least a two- to three-year period, hopefully longer, and if I can recoup those costs over a two-year period, I think that would be seen as very positive.”
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