Amid FDA questions, BioMarin confident of Roctavian approval timeline

BioMarin still expects a US approval decision for gene therapy Roctavian by March 31 despite FDA data demands and observations made during a pre-licensure inspection at a manufacturing facility in California. 

BioMarin is hoping to win US approval for its single-dose hemophilia A gene therapy this month, with a proposed US Food and Drug Administration (FDA) PDUFA (Prescription Drug User Fee Act) date for Roctavian (valoctocogene roxaparvovec) of March 31.

However, this date could be compromised following a pre-licensure inspection in December at the firm’s Novato, California gene therapy plant that resulted in numerous comments and observations.

Image: DepositPhotos/JHVEPhoto

When an FDA inspection ends in observations, whether critical or otherwise, a Form 483 is normally issued. A spokesperson from BioMarin would not confirm that this was the case following the Novato inspection but did tell us the pre-license inspection of the facility is complete, the firm has provided responses to all the comments and observations received, and BioMarin believes “all of the issues raised are addressable.”

No further details regarding the inspection were divulged.

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

On the firm’s Q4 2022 financial call this week, Hank Fuchs, president of Worldwide R&D, added “2023 regulatory milestones are tracking to plan,” following the submission of three-year Phase III Roctavian data as requested by the Agency.

“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.”

He added: “The FDA has also planned some clinical study site inspections that will take place this quarter prior to the PDUFA date. So, the review process is tracking to expectations.”

In Europe, Roctavian received approval in June 2022. With the launch underway, CEO JJ Bienaimé said the firm is “very encouraged by the interest” being shown for the therapy.

“While the process of finalizing reimbursement in Germany has taken longer than expected, given the novelty of the approach, at the patient level, we are very encouraged by the interest we are seeing for Roctavian,” he told stakeholders. “And patients have gone through a companion diagnostic testing in Germany as an important first step in the patient journey toward treatment.”

In its 2023 guidance, the firm is expecting Roctavian revenues of between $100 and $200 million.