Tucked into its latest earnings report, BioMarin Pharmaceutical said that the Food and Drug Administration plans to convene a group of outside advisers to assess the company’s gene therapy for hemophilia.
BioMarin said it has not yet been given a date for when this advisory committee meeting will take place. The company officially resubmitted the approval application for its treatment, called Roctavian, to the FDA in late September, and expects a verdict by the end of March.
“This is something that we've been preparing for over the last few months,” said Henry Fuchs, BioMarin’s president of worldwide research and development, on a call with investors Wednesday. “We believe the [meeting] will provide a good forum to review the demonstrated bleeding control and established safety profile of Roctavian.”
BioMarin specifically designed its therapy to treat the more common, “A” form of hemophilia, in which patients lack a key blood-clotting protein known as Factor VIII. Roctavian uses a certain kind of virus to deliver a functional copy of the gene that makes this protein, and in clinical testing has been shown to substantially raise Factor VIII levels and prevent episodes of excessive bleeding in patients with “severe” hemophilia A.
BioMarin first sought FDA approval of Roctavian in late 2019, basing its application on initial results from a large, late-stage trial as well as a smaller one that had been running for longer. Though both studies generated positive results, the FDA ultimately rejected the request and asked for at least two years of follow-up data from each patient enrolled in that larger study.
BioMarin has since collected that data, which, according to the company, further establishes its therapy as a safe and effective treatment.
Earlier this year, Roctavian became the first hemophilia A gene therapy to be approved in Europe, where it’s now sold at a list price of roughly 1.5 million euros. A nod from the FDA would give Roctavian the same title in the U.S., cementing its leading position against rival treatments being developed by Roche and Sangamo Therapeutics.
The FDA is also currently reviewing a hemophilia B gene therapy developed by UniQure, a Netherlands-based biotechnology company, with a decision anticipated in the next month or so.
According to analysts, the FDA’s decision to hold an advisory panel for Roctavian was somewhat expected. “This isn’t a huge surprise,” wrote Paul Matteis, an analyst at the investment firm Stifel, in a note to clients.
Matteis and others believe the FDA will greenlight the therapy this time around, which could provide a significant lift to BioMarin’s bottom line. SVB Securities, for example, has forecasted around $2.2 billion in peak annual Roctavian sales.
BioMarin appears confident in the odds of approval, too. On Wednesday’s investor call, Fuchs noted how, among the 11 cell and gene therapy applications cleared by the FDA, nearly half had advisory committee meetings.