The U.S. Food and Drug Administration has lifted the clinical hold on the company’s mid-stage study of RP-A501 for the treatment of Danon disease.
Rocket Pharmaceuticals, Inc. (RCKT) on Wednesday said that the U.S. Food and Drug Administration (FDA) has lifted the clinical hold on its mid-stage study of RP-A501 for the treatment of Danon disease.
The hold, imposed roughly three months ago, was lifted after Rocket satisfactorily addressed issues outlined in the clinical hold. The agency authorized the study to resume with an adjusted dose of the investigational gene therapy.
Danon disease is a rare, genetic disorder primarily affecting the heart, skeletal muscles, and sometimes the brain. It’s characterized by weakening of the heart muscle, muscle weakness, and intellectual disability.
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