- Gene therapy company Rocket Pharmaceuticals ( NASDAQ: RCKT ) announced Thursday that, subject to FDA feedback, the company will start the initial component of a pivotal global trial for its Danone disease candidate RP-A501 in H1 2023.
- A genetically inherited disorder, Danon Disease results in the accumulation of autophagosomes and glycogen, mainly in cardiac and other tissues, ultimately leading to heart failure and death.
- The announcement follows an end-of-Phase 1 meeting between the regulator and the company on the adeno-associated virus (AAV)-based gene therapy in Danon Disease.
- Based on FDA feedback, Rocket ( RCKT ) intends to use a natural history comparator and a biomarker-based composite endpoint in a single-arm open-label study design for RP-A501 in Danone disease.
- “We look forward to continued dialogue with the FDA on the design for our proposed pivotal trial, including discussion of appropriate external controls for the study and appropriate endpoints to support accelerated approval,” Chief Executive Gaurav Shah remarked.
- In September, Rocket Pharma ( RCKT ) said that RP-A501 sustained clinical benefit for up to 36 months in a Phase 1 trial for Danon Disease.
For further details see:
Rocket Pharma to start pivotal trial for Danone disease candidate in H1 2023