Rocket Pharmaceuticals announced it has reached an agreement with the U.S. Food and Drug Administration (FDA) on the key design elements for its global pivotal Phase 2 trial of RP-A501, a gene therapy for the treatment of Danon disease. This alignment clarifies the regulatory path forward and supports a potential accelerated approval pathway.
Key Details
- Pivotal Population: The FDA confirmed a pivotal efficacy population of 12 patients to be treated with a recalibrated dose of 3.8 x 10¹³ genome copies per kilogram (GC/kg).
- Trial Status: The first three patients treated under the modified protocol will be included in the pivotal group, leaving nine additional patients to be enrolled and treated.
- Endpoints & Timeline: The trial will use established 12-month co-primary endpoints intended to support accelerated approval. The company expects to complete dosing of the remaining patients by mid-2027.
- Safety: The FDA's review of initial safety data supports the continuation of enrollment and dosing under the modified protocol.