Rocket Pharma Reaches FDA Alignment in Danon Heart Disease Treatment Trial

 Rocket Pharmaceuticals said it had reached alignment with the Food and Drug Administration on its Phase 2 trial of its treatment for Danon disease.

The Cranbury, N.J.-based pharmaceutical company said its study would evaluate the use of RP-A501 to treat 12 patients with Danon disease, a fatal cardiomyopathy with no cure. The Phase 2 trial will be an open-label study with a biomarker-based co-primary endpoint assessed after 12 months, the company said.

The trial will support accelerated approval of Rocket's treatment by the FDA.

The company said it expected to file its Clinical Trial Application/Investigational Medicinal Product Dossier for RP-A501, which will allow for studies to take place in the European Union in the third quarter.

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