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Friday morning, the company confirmed that it has “voluntarily withdrawn” the BLA, blaming the decision on July’s reprioritization, “under which Rocket is focusing its resources on programs with the clearest regulatory and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.”
The decision was the result of “business and strategic considerations” rather than any safety or efficacy concerns, the company stressed in an Oct. 3 filing to the Securities and Exchange Commission.
“The company will consider external partnership opportunities that could potentially enable the advancement of RP-L102 in the future,” the biotech added. “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.”
Rocket has suffered a rocky couple of years with regulators. Last year, the FDA denied Rocket’s application to approve Kresladi to treat severe leukocyte adhesion deficiency-I due to manufacturing queries. The company now expects to resubmit that BLA by the end of 2025.
In May of this year, a pivotal trial of the AAV9 gene therapy RP-A50 in Danon disease was placed on clinical hold around the time of a patient death in the trial and only lifted last month.
The aim of Rocket’s restructuring was to move away from its hematology assets—although Kresladi remains a priority—and focus on the cardiovascular side of the pipeline, namely the Danon program and treatments for two forms of cardiomyopathy.