Rocket Pharmaceuticals Withdraws Gene Therapy Application, Shifts Focus

Rocket Pharmaceuticals has voluntarily withdrawn its biologics license application (BLA) for RP-L102, a gene therapy candidate aimed at treating fanconi anemia, a rare genetic disorder characterized by bone marrow failure. This decision marks a significant shift in the company's strategic direction and pipeline priorities.

BLA Withdrawal and Strategic Realignment

The withdrawal of the RP-L102 BLA, which was initiated as a rolling application in the first quarter of 2025, comes as part of a broader restructuring effort announced by Rocket in July. The company emphasized that the decision was based on "business and strategic considerations" rather than safety or efficacy concerns.

Rocket's restructuring plan involves focusing resources on programs with clearer regulatory and commercial pathways. This shift has led to a move away from certain hematology assets, with the exception of Kresladi, and a renewed focus on cardiovascular programs, including treatments for Danon disease and two forms of cardiomyopathy.

Regulatory Challenges and Future Outlook

The withdrawal of the RP-L102 application is not the only regulatory setback Rocket has faced recently. In 2024, the FDA denied the company's application for Kresladi, intended to treat severe leukocyte adhesion deficiency-I, due to manufacturing queries. Rocket now anticipates resubmitting the Kresladi BLA by the end of 2025.

Additionally, a pivotal trial of RP-A50, an AAV9 gene therapy for Danon disease, was placed on clinical hold in May 2025 following a patient death. The hold was lifted last month, allowing the trial to proceed.

Despite these challenges, Rocket remains open to potential external partnerships that could enable the advancement of RP-L102 in the future. The company stated that withdrawing the BLA preserves its ability to re-engage with regulators at a later date, should a suitable strategic or partnership opportunity arise to sustainably progress the program.