FDA lifts clinical hold on Rocket's gene therapy for heart failure after 3-month delay
New Jersey’s Rocket Pharmaceuticals hit a major roadblock for its AAV-delivered gene therapy for heart failure in May after the FDA came asking for more details on target patients. Three months down the road, Rocket is clear for liftoff once again.
The FDA on Monday lifted its clinical hold on Rocket’s Phase I trial for gene therapy candidate RP-A501 for Danon disease, an ultra-rare genetic disorder that can lead to heart failure in children and young adults, the company said in a release.
It’s been a little more than three-month wait for Rocket after the agency grounded the trial, asking for a revised protocol on “patient selection and management.”
On Monday, Rocket said it had addressed those issues and was ready to move forward, citing no new safety red flags churned up during the agency’s review. The company expects to resume dosing in the adult cohort “as soon as possible” and is gearing up to begin enrollment for a pediatric cohort as well, Rocket said.
Unlike the rest of its pipeline, which uses lentiviral vectors to transport its gene therapies, RP-A501 is Rocket’s only candidate that uses AAV9 for delivery, a vector that has received intense scrutiny after a wave of safety flags in recent years. The therapy uses that vector to transport a functional version of lysosome-associated membrane protein 2 (LAMP-2) into patients’ cells — the mutated form of the protein causes a buildup of autophagosomes and glycogen in the heart and other tissues that can cause heart failure and death, particularly in boys and young men.
Rocket believes there are somewhere between 15,000 and 30,000 patients with Danon in the US and Europe. The drug would become the first gene therapy approved for monogenic heart failure, Rocket said.