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# Rocket Pauses Phase 1 Danon Disease Trial
- URL: https://www.fdaweb.com/rocket-pauses-phase-1-danon-disease-trial/
- Published: 2021-05-11T12:00:00.000Z
- Updated: 2026-09-14T16:58:24.000Z
- Author: David McFarland
- Tags: Drugs, #legacy-id-D5149292

Rocket Pharmaceuticals says it is pausing its RP-A501 Danon Disease program for additional risk mitigation requested by FDA. The company [says](https://ir.rocketpharma.com/news-releases/news-release-details/rocket-pharmaceuticals-reports-first-quarter-2021-financial?ref=fdaweb.com) the agency asked it to pause patient dosing and modify the protocol and other supporting documents with revised guidelines for patient selection and management. Rocket says it expects to resume patient treatment in the third quarter of 2021.

The company also says new longer-term lower-dose data demonstrate durable expression and ongoing improvements in biomarkers, and evidence of a positive risk/benefit profile out to 18 months.