- The Food and Drug Administration accepted BioMarin Pharmaceutical’s latest approval application for a gene therapy designed to treat severe hemophilia A, and has set a target decision date of March 31.
- This is the company’s second attempt to win FDA clearance for its therapy, which is known as Roctavian. In August 2020, the agency surprised BioMarin executives and investors by rejecting Roctavian and telling its maker that longer-term data on treated patients was needed.
- BioMarin said Wednesday it has responded to all of the FDA’s earlier concerns. The company is also offering to follow patients who participated in its research for 15 years and conduct a post-approval registry study that would provide data on how the therapy works in a “real-world” setting.
While gene therapy holds the promise of a permanent fix for genetic conditions like hemophilia, regulators are treading carefully with the technology and the many questions it raises.
BioMarin cautioned that the target decision date of March 31 could be extended with another data request during the review. And while the FDA hasn’t indicated it will hold a meeting of outside experts to review the application, a decision to convene an advisory panel wouldn’t be surprising, analysts said.
Still, analysts were pleasantly surprised by the FDA’s move to accept the latest BioMarin submission so quickly. The company itself has also been anticipating a nine-month target date from the outset, rather than the six-month review set by the FDA.
The agency’s actions to date suggest that the question of approval is now “when,” not “if,” SVB Securities analyst Joseph Schwartz wrote in a note to investors. The recent approval of the therapy in the European Union also bodes well. “[R]egulatory agencies tend to think more alike than differently,” Schwartz wrote in an Oct. 12 note to clients.
Hemophilia A is a genetic disorder caused when a blood clotting protein known as Factor VIII is either missing or malfunctioning. The result is episodes of internal bleeding that can be both painful and life-threatening.
There is currently no therapy that can fix the underlying genetic cause of hemophilia. Patients who have the condition are often treated with drugs that replace the missing clotting protein, though they can still have breakthrough episodes.
As BioMarin’s application advances, analysts will be closely watching the progress of another gene therapy, this one for hemophilia B. The FDA is due to act on an approval application from CSL Behring and UniQure by late November.
In the meantime, BioMarin is restructuring. Last week, the company announced plans to lay off about 120 employees, or 4% of its global workforce. Though BioMarin has brought eight drugs to market, it has rarely turned a profit since starting operations in the late 1990s.