Sangamo Therapeutics will regain control of giroctocogene fitelparvovec, a hemophilia A gene therapy candidate, after Pfizer terminated their collaboration and licensing agreement. Pfizer cited its decision not to pursue a Biologics License Application or commercialization, despite positive Phase 3 trial results. Sangamo plans to explore all options for the program, including finding a new partner.
This decision is impactful because it unexpectedly shifts the trajectory of a promising hemophilia A therapy. While Pfizer’s decision is surprising given the positive clinical data, it creates an opportunity for another company to capitalize on a late-stage asset with demonstrated efficacy. This could also lead to faster patient access if a partner with experience in rare disease commercialization steps in quickly.
The Phase 3 AFFINE trial met its primary and key secondary endpoints, demonstrating superiority over standard prophylaxis in reducing bleeding rates. Pfizer’s withdrawal comes shortly before anticipated regulatory submissions, highlighting a potential disconnect between clinical success and strategic priorities. Sangamo will need to secure funding and potentially restructure its operations to manage the transition of the program.
Moving forward, Sangamo’s success hinges on finding a suitable partner to navigate regulatory submissions and commercialization of giroctocogene fitelparvovec. This situation underscores the complexities of drug development and the importance of alignment between collaborators. The hemophilia A community awaits the next chapter for this promising therapy, hopeful for a swift path towards patient access.
Jon Napitupulu is Director of Media Relations at The Clinical Trial Vanguard. Jon, a computer data scientist, focuses on the latest clinical trial industry news and trends.
