One of the 11 potential blockbuster drugs included in the Clarivate 2020 Cortellis Drugs to Watch analysis is Valrox (valoctocogene roxaparvovec) from San Rafael Calif-based Biomarin Pharmaceutical Inc., which is poised to reshape the treatment landscape for hemophilia A. The therapy moved one step closer to entering the U.S. market with the company reporting that that the FDA had accepted for priority review the BLA for its investigational AAV5 gene therapy for adults with hemophilia A, setting a PDUFA action date of Aug. 21, 2020.
Interestingly, the agency told the company it is not currently planning to hold an advisory committee meeting to discuss the application.
“The adcomm comes as an upside surprise,” noted J.P. Morgan analyst Cory Kasimov in a report on the company’s news. “In our view, this bodes well for the company as it suggests that there are no outstanding questions at this time from FDA that they are looking to get expert feedback on.”
If approved, Valrox would be the first potentially curative (one and done) approach to hemophilia A, eliminating the need for blood transfusions and factor VIII (FVIII) replacement therapy after a single infusion. People suffering from the most severe form of hemophilia A often experience painful, spontaneous bleeds into their muscles or joints and that group make up approximately 43% of the hemophilia A population. The standard of care (SOC) for such individuals is a prophylactic regimen of replacement FVIII infusions administered intravenously up to two to three times per week, or 100 to 150 infusions per year. Despite those treatments, many people continue to experience bleeds, resulting in progressive and debilitating joint damage, which can have a major impact on their quality of life.
Biomarin’s application is based on a phase III interim analysis of study participants treated with Valrox, along with three-year phase I/II data that found the high-dose cohort showed 100% resolution of target joints, a 96% reduction in mean annualized FVIII usage, and all patients remained off FVIII prophylaxis therapy. Biomarin has constructed, commissioned and validated a gene therapy manufacturing facility located in Novato, Calif., that will produce the therapy.
In a presentation at the annual J.P. Morgan Healthcare Conference in January, Biomarin Chairman and CEO Jean-Jacques Bienaime revealed the company was ramping up productivity at the plant, more than doubling its capacity to 10,000 doses annually.
Biomarin also announced that the FDA had accepted the premarket approval application for an AAV5 total antibody assay intended to serve as a companion diagnostic test for the AAA5 gene therapy. The blood test will help identify patients most likely to respond to AAV5-based gene therapy. Biomarin said it implemented the test in multiple clinical studies evaluating Valrox treatment of hemophilia A patients without antibodies to AAV5.
The positive news boosted Biomarin’s share value (NASDAQ:BMRN) closing Feb. 21 at $95.16, up $5.50, or 6.13%.