FDA rejects Biologics License Application for valoctocogene roxaparvovec (Valrox) for severe haemophilia A

The agency recommended that the company complete the phase III trial and submit two-year follow-up safety and efficacy data for this potential gene therapy that is administered as a single infusion. There have been some concerns over the duration of response.

SPS commentary:

The phase III trial was fully enrolled in November 2019 and the last patient will complete two years of follow up study in November 2021.  A Marketing Authorisation Application was submitted to the European Medicines Agency in November 2019.


Biospace Inc.