Rocket Pharmaceuticals withdraws BLA for Fanskya

In a regulatory filing, Rocket Pharmaceuticals (RCKT) announced that it has voluntarily withdrawn its Biologics License Application to the U.S. Food and Drug Administration for mozafancogene autotemcel, also known as “Fanskya,” “Fanca-cel,” or “RP-L102”, an investigational gene therapy for the treatment of Fanconi Anemia. This decision follows the company’s previously announced corporate prioritization, under which Rocket is focusing its resources on programs with the clearest regulatory and commercial pathways. As a result, Rocket ceased new internal investment in RP-L102 in July 2025, subsequently withdrew its Marketing Authorization Application with the European Medicines Agency in July 2025, and has now withdrawn its U.S. BLA. The company’s decision to withdraw the BLA is based on business and strategic considerations and does not reflect concerns regarding the safety or efficacy profile of RP-L102. Data generated to date continue to support that RP-L102 has been generally well tolerated, with no significant safety signals observed, and a risk-benefit profile that appears favorable. The company will consider external partnership opportunities that could potentially enable the advancement of RP-L102 in the future. Withdrawal of the BLA preserves Rocket’s ability to re-engage with regulators at a later date should there be an appropriate strategic or partnership pathway to sustainably progress the program.