Rocket Pharmaceuticals Inc., a late-stage biotechnology company, announced that the U.S. Food and Drug Administration (FDA) has lifted the clinical hold on its pivotal Phase 2 trial of RP-A501 for treating Danon disease. The hold was removed in under three months, highlighting the FDA's efficient review process. The trial will proceed with a recalibrated dose, aligning with successful outcomes from the Phase 1 study. The study aims to support accelerated approval by evaluating the efficacy of RP-A501 through various biomarkers and clinical endpoints. A global natural history study is also running concurrently with this pivotal trial.
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