BioMarin gets FDA greenlight for Roctavian

The US Food and Drug Administration (FDA) has approved BioMarin’s single-dose hemophilia A gene therapy, Roctavian (valoctocogene roxaparvovec).

Roctavian has been designed to replace the function of the mutated gene, which enables the individual to produce their own FVII protein and thus decrease bleeding episodes. In Europe, Roctavian received approval in June 2022.

“Today’s approval of Roctavian builds on BioMarin’s proven track record of advancing treatments that target the underlying cause of life-threatening genetic conditions, which has produced eight best or first-in-class commercial therapies,” said Jean-Jacques Bienaimé, CEO of BioMarin.

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“We are proud to now offer adults with severe hemophilia A, a one time, single-dose treatment options.”

The firm said the approval follows the “largest and longest” Phase III study. 134 patients received Roctavian and were monitored for at least 3 years. 112 of the patients experienced a mean annual bleeding rate (ABR) reduction of 52% after receiving the gene therapy. The company said it will continue to analyze the long-term effects of treatment as well as following all clinical trial participants for up to 15 years.

The gene therapy is manufactured at BioMarin’s Novato, California facility. According to the firm, the plant will be able to meet commercial demand throughout Roctavian’s product lifecycle.

The road to approval

BioMarin suffered US delays in the Roctavian approval process having received a Complete Response Letter (CRL) in 2020 after the FDA requested more data.

In March, the company announced it expected a US approval decision for Roctavian by March 31 despite FDA data demands and observations made during a pre-licensure inspection at its Novato facility.

At the time, Hank Fuchs, president of Worldwide R&D, said on the firm’s Q4 2022 financial call, “Should the FDA determine that the three-year data submission does represent a major amendment and thereby extending the PDUFA action date, we will share that update publicly. In the meantime, we continue to experience a high level of engagement with the agency as we are still under active review.”

Moreover, in its 2023 guidance, BioMarin said it is expecting Roctavian revenues of between $100 and $200 million.