FDA delays BioMarin’s gene therapy for haemophilia A

pharmafile | August 20, 2020 | News story | Research and Development, Sales and Marketing FDA 

The FDA has delayed Roctavin, BioMarin’s gene therapy for haemophilia A, by a year to wait for more extensive results, it has been revealed. 

The FDA said it needs longer term clinical trial data, and complete results from BioMarin’s two-year follow-up of its Phase 3 GENEr8-1 trial. This study included 134 patients and investigated the safety and effectiveness of a single infusion of Roctavian in adults with severe haemophilia A. 

The company had released an interim analysis last year, which included 26-week data on 16 treated patients. The treatment lowered the number of annual bleeds patients experienced by 85% since the start of the study, and the annual median usage of factor VIII (FVIII) by 84%. 

The trial is set to completely wrap up in November 2021, where the final patients will finish two years of post-treatment evaluation. Due to the FDA waiting for these final results, the regulatory body will not make a decision on its approval until either late next year or in 2022. 

The FDA also stated that the difference between the Phase 1 and 2 studies and the Phase 3 trial means they cannot rely on the earlier results to support Roctavin’s durability. 

Jean-Jacques Bienaime, BioMarin’s Chairman and CEO, said: “We are surprised and disappointed that the FDA introduced new expectations for the first time in the Complete Response Letter. We are confident in Roctavian gene therapy and its potential to redefine the treatment paradigm for people with haemophilia A. We remain committed to the haemophilia community and to leading the way to the first ever gene therapy in haemophilia A.”

Roctavin is a one-time gene therapy aimed at durably restoring the production of FVIII, the blood clotting protein missing in people with haemophilia A. 

Conor Kavanagh

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