BioMarin Pharmaceutical disclosed in a filing with the US Securities and Exchange Commission that European regulators have asked for more data as part of their ongoing review of the marketing application for valoctocogene roxaparvovec (valrox), the company's proposed gene therapy for severe haemophilia A. As a result of the request, the ongoing review by the European Medicines Agency (EMA) has reverted from an accelerated assessment to a standard one.
Specifically, the EMA wants to see full 52-week results from the 134 patients enrolled in a Phase III study of valrox who received the 6e13 vg/kg dose. According to BioMarin, the last patient is expected to reach 52 weeks of follow-up in November, after which the company aims to submit the requested data by the end of the first quarter of 2021. BioMarin said it would provide an update regarding timing of the review following further interactions with the EMA.
The delay comes shortly after the FDA issued a complete response letter regarding BioMarin's US application for valrox in severe haemophilia. The agency requested data showing substantial evidence of a durable effect based on two years of follow-up on all participants enrolled in the ongoing Phase III 270-301 study. BioMarin indicated that the last patient in study 270-301 will complete two years of follow up in November 2021, with Stifel analyst Paul Matteis suggesting that approval could follow a year after that.
CEO Jean-Jacques Bienaimé previously said that if approved, BioMarin was looking at pricing the treatment, also known as BMN 270, at between $2 million and $3 million.
For further analysis, read ViewPoints: Durability concerns derail BioMarin's haemophilia A gene therapy, and see KOL Views Results: Leading expert says FDA's rebuff of BioMarin’s valrox raises bar for gene therapy – perhaps beyond haemophilia A.
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