Rocket Pharmaceuticals has suffered a rocky couple of years with regulators. (iStock / Getty Images Plus)
By James Waldron October 3, 2025
Having already altered the journey of its rare disease gene therapy to a hoped-for FDA approval, Rocket Pharmaceuticals has now aborted the mission entirely.
The biotech initiated a rolling biologics license application (BLA) for the candidate—known variously as RP-L102, mozafancogene autotemcel or Fanca-cel—in the first quarter of the year. The application was specifically to treat fanconi anemia, a rare genetic disorder characterized by bone marrow failure.
The future of the application appeared in doubt back in July, when Rocket said it “anticipated delays” to the RP-L102 program as part of a wider restructuring of the company’s pipeline and workforce. At the time, the biotech withdrew its approval application to European regulators and warned that an FDA approval was “no longer scheduled for 2026.”
Friday morning, the company confirmed that it has “voluntarily withdrawn” the BLA, blaming the decision on July’s reprioritization, “under which Rocket is focusing its resources on programs with the clearest regulatory and commercial pathways.”
