BioMarin resubmits its hemophilia gene therapy to the FDA – BioPharma Dive

Dive Brief:

Roctavians 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 companys 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 diseases root cause by replacing the defective gene with a functional copy.

The FDA rejected BioMarins original approval application back in August 2020 and requested more data to prove the treatments 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 mildlevels 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 BioMarins 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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BioMarin resubmits its hemophilia gene therapy to the FDA - BioPharma Dive