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Aug 17, 2021

Rocket Relaunches Gene Therapy Program as FDA Lifts Clinical Hold

Posted by in category: biotech/medical

New gene therapy in trials for Danon disease.

“The only available treatment option for Danon disease is a heart transplant. Currently, there are no specific therapies available for the treatment of Danon disease.”


The U.S. Food and Drug Administration (FDA) lifted the clinical hold it placed on Rocket Pharmaceuticals’ experimental gene therapy for Danon disease. Patient enrollment in the Phase I study will resume, the company announced this morning.

New Jersey-based Rocket Pharmaceuticals said it intends to resume the Phase I program as quickly as possible. Dosing of patients in the pediatric cohort that was receiving the lowest-level of the medication will resume in the third quarter.

The FDA placed the clinical hold on Rocket’s RP-A501 in May of this year. The regulatory agency halted the study to ensure the company modified the study protocol, as well as other supporting documents that included revised guidelines for patient selection and safety management. The trial was not halted due to concerns over safety of the gene therapy. Rocket noted that no drug-related safety events have been observed in cohorts that received low and high doses.

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