Rocket Pharma Withdraws Gene Therapy BLA

Rocket Pharmaceuticals has voluntarily withdrawn its BLA for RP-L102 (mozafancogene autotemcel), an investigational gene therapy for treating Fanconi Anemia. The move follows the company’s previously announced corporate prioritization to focus its resources on programs with the clearest regulatory and commercial pathways.

The company notes its decision “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." 

Rocket says it will consider external partnership opportunities that could advance 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,” it says.