Clinical Trials
Gene Therapy
Regulatory & Standards

FDA Lifts Clinical Hold on Rocket’s Danon Disease Gene Therapy Trial

Georgi Makin
19 August 2021
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Rocket Pharmaceuticals has announced the FDA has lifted a clinical hold for gene therapy – RP-A501- for rare, Danon Disease. 

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After addressing FDA requests to modify the trial’s protocol and revising patient selection and management, Rocket Pharmaceuticals has confirmed that patient enrolment for the Phase I clinical trial can resume.

Rocket reports to be initiating the resumption of the trial as soon as possible, expecting dosing in the low-dose paediatric patient cohort as soon as Q3.

Gaurav Shah, CEO of Rocket Pharma, commented:

“We are grateful for the collaboration between the FDA and our team in reaching agreement on protocol updates allowing us to resume patient enrolment in our Danon Disease trial. We look forward to progressing this critical work on behalf of all Danon patients. We are moving as quickly as possible to resume dosing and commence treatment this quarter. Additionally, given the activity observed among young adults in our low-dose cohort, in agreement with the FDA, we are now proceeding with the paediatric cohort. This is another important step forward as we believe the paediatric Danon population has the potential to realize the maximum benefit from our Danon Disease gene therapy program. We continue to anticipate reporting updated longer-term data from the low-dose (6.7e13 vg/kg) and higher-dose (1.1e14 vg/kg) young adult cohorts in the fourth quarter.”


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Danon disease is a rare X-linked inherited disorder with a prevalence of 15,000–30,000 patients in the US and Europe. Mutations in the gene encoding autophagy mediator, lysosome-associated membrane protein 2 (LAMP-2), cause accumulation of autophagosomes and glycogen in tissues, such as cardiac muscle. Ultimately, this accumulation can lead to heart failure and death in male patients, in adolescence or young adulthood.

Currently, the only available treatment for Danon Disease is heart transplantation, but this is not considered a curative option and carries with it its own risks.

Rocket Pharmaceuticals is therefore investigating the use of a gene therapy product, RP-A501 – currently the only investigational gene therapy under development. Currently being evaluated in an ongoing Phase I clinical trial, RP-A501 consists of a recombinant adeno-associated serotype 9 (AAV9) capsid containing a functional version of the human LAMP2B transgene (AAV9.LAMP2B).

So far, Rocket reports, preliminary activity data and a favourable tolerability profile have already been observed. However, the trial was placed on clinical hold in May 2021 by the FDA, with guidance to modify the study protocol and other supporting documents with revised guidelines for patient selection and safety management.

Rocket reports that ‘no new drug-related safety events were observed in the low-dose or higher-dose young adult cohorts as part of the clinical hold or during the hold’.

Source: Rocket Pharmaceuticals press release

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