Why Is Rocket Pharma Stock Up 25% Pre-Market Today?

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.
Rising stock market chart on a trading board background.
Rising stock market chart on a trading board background. | Image source: Yuichiro Chino on Getty Images
Profile Image
Anan Ashraf·Stocktwits
Updated Aug 20, 2025 | 8:03 AM GMT-04
Share this article

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 following the death of a patient in the trial, was lifted after Rocket satisfactorily addressed issues outlined in the clinical hold, the company said. The agency authorized the study to resume with an adjusted dose of the investigational gene therapy.

Rocket Pharma stock shot up 25% in Wednesday’s pre-market session following the development. On Stocktwits, retail sentiment around RCKT rose from ‘bearish’ to ‘bullish’ territory over the past 24 hours, while message volume rose from 'low' to 'normal' levels. 

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.

Following the death of a patient, Rocket paused further dosing in the mid-stage study, and on May 23, the FDA placed a clinical hold on the trial to allow for further evaluation. The trial can now resume at an adjusted dose of RP-A501 in three patients, with a minimum four-week interval between dosing, followed by additional patients to complete the trial. The low dose has been determined as most likely to confer the safety and efficacy identified in the low-dose early-stage study cohorts, the company said.

Rocket also said that it will collaborate with investigators to implement an immunomodulatory regimen more closely reflecting the one administered in the early-stage study pediatric cohort. Further updates about the mid-stage study can be expected following review of data from the next three patients, the company said.

RCKT stock is down by 77% this year and by about 85% over the past 12 months.

Read also: Novo Nordisk Freezes Hiring To Cut Costs, Says Report: Retail Sees Stock Surpassing $100-Mark In A Year

Subscribe to Trends with Friends
All Newsletters
For serious investors with a serious sense of humor.

For updates and corrections, email newsroom[at]stocktwits[dot]com.

Read about our editorial guidelines and ethics policy