BioMarin resubmits its hemophilia gene therapy to the FDA

Dive Brief:

• Two years after a surprising rejection, BioMarin Pharmaceutical has resubmitted its hemophilia gene therapy for approval by the Food and Drug Administration, announcing the completed filing on Thursday.
• BioMarin expects to hear from the FDA on whether its application has been accepted by late October. The company is anticipating a nine-month review as more clinical trial data is expected to emerge from ongoing studies of the therapy in patients with severe hemophilia.
• BioMarin’s gene therapy, called Roctavian, recently won approval in Europe and, if its review by the FDA goes according to the company’s plan, would be the first gene therapy for hemophilia on the market in the U.S.

Dive Insight:

Roctavian’s road back to the FDA has been long, as BioMarin has had to gather additional data in support of its therapy. Delays and new safety concerns further hampered the company’s resubmission plans.

The treatment is for people with severe hemophilia A, a genetic disease that leaves people with little or no clotting protein needed to prevent excessive bleeding.

While there are many treatments that work by replacing this protein artificially, Roctavian would be the first in the U.S. to address the disease’s root cause by replacing the defective gene with a functional copy.

The FDA rejected BioMarin’s original approval application back in August 2020 and requested more data to prove the treatment’s benefit for patients over a longer period of time.

BioMarin successfully gathered that follow-up data, showing its gene therapy could restore protein levels to “mild” levels and prevent bleeds. The company had expected to file for approval in June but pushed back to September after the FDA asked for further information.

Roctavian gained European approval last month, a few weeks before it was disclosed that a patient treated in clinical trials had developed leukemia. This was the second cancer case reported among patients taking Roctavian. Cases of cancer in clinical trials of gene therapies have been watched closely by U.S. regulators. However, in BioMarin’s case, genetic testing suggested the case may be naturally occurring, rather than stemming from the therapy. Trial monitors did not call for the study to be halted.

If approved in the U.S., Roctavian is expected to carry a high price tag, likely in the millions of dollars. In Europe, BioMarin is charging around 1.5 million euros, net of discounts. Other gene therapies cleared for the U.S. market have been priced at $2.1 million, $2.8 million and $3.0 million, respectively.

Pfizer and partner Sangamo Therapeutics as well as Spark Therapeutics — now owned by Roche — are also working on hemophilia gene therapies.

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