European regulators have requested more data to review the marketing application for BioMarin’s haemophilia A gene therapy Valrox (valoctocogene roxaparvove).
The company disclosed in a US Securities and Exchange Commission filing that the European Medicines Agency has asked for full 52-week results from the 134 patients taking part in the ongoing Phase III study of the treatment.
BioMarin said it expects the last patient to reach 52 weeks of follow-up in November 2020, and that it is working with the regulator to enable a potential submission of the requested data by the end of the first quarter of 2021.
As a result of the EMA’s request, the review of the Valrox’ marketing application has reverted from an accelerated assessment to a standard review, the company confirmed.
