Rocket Pharmaceuticals Halts FDA Approval Bid for Fanconi Anemia Gene Therapy

Rocket Pharmaceuticals has announced the withdrawal of its biologics license application (BLA) for RP-L102, a gene therapy 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 focus and pipeline priorities.

BLA Withdrawal and Strategic Realignment

The company initiated a rolling BLA for RP-L102, also known as mozafancogene autotemcel or Fanca-cel, in the first quarter of 2025. However, as part of a broader restructuring announced in July, Rocket has now voluntarily withdrawn the application. The biotech emphasized that this decision was based on "business and strategic considerations" rather than any safety or efficacy concerns.

Rocket's reprioritization strategy, unveiled earlier this year, aims to concentrate resources on programs with the clearest regulatory and commercial pathways. This restructuring included workforce reductions and a narrowing of the company's pipeline focus.

Implications for Fanconi Anemia Program

While the BLA withdrawal represents a setback for patients awaiting new treatments for Fanconi anemia, Rocket has not completely abandoned the program. The company stated it will "consider external partnership opportunities that could potentially enable the advancement of RP-L102 in the future." By withdrawing the BLA, Rocket preserves the option to re-engage with regulators at a later date, should a suitable strategic or partnership pathway emerge.

Broader Regulatory Challenges

The withdrawal of RP-L102's BLA is not an isolated incident for Rocket Pharmaceuticals. The company has faced several regulatory hurdles in recent years:

1. In 2024, the FDA denied Rocket's application for Kresladi, a treatment for severe leukocyte adhesion deficiency-I, citing manufacturing concerns. The company now plans to resubmit this BLA by the end of 2025.

2. In May 2025, a pivotal trial of RP-A50, an AAV9 gene therapy for Danon disease, was placed on clinical hold following a patient death. This hold was lifted in September 2025.

These challenges have contributed to Rocket's decision to shift its focus away from hematology assets, with the exception of Kresladi, and towards its cardiovascular pipeline. The company is now prioritizing its Danon disease program and treatments for two forms of cardiomyopathy.

As Rocket Pharmaceuticals navigates these strategic changes, the pharmaceutical industry will be watching closely to see how the company's refocused pipeline performs and whether external partnerships materialize to advance programs like RP-L102 for Fanconi anemia.