2023-06-08 08:38:34 ET
Rocket Pharmaceuticals ( NASDAQ: RCKT ) announced Thursday that the FDA granted Fast Track and Orphan Drug designations for its gene therapy candidate RP-A601 as a treatment for the rare heart disorder, plakophilin-2 related arrhythmogenic cardiomyopathy (PKP2-ACM).
PKP2-ACM is an inherited disorder linked to abnormal structural changes in the heart, irregular heart rates, and sudden cardiac death.
No curative options are currently available as standard of care to treat PKP2-ACM, which affects an estimated ~50K people in the U.S. and Europe.
The FDA recently cleared Rocket ( RCKT ) Investigational New Drug (IND) application for RP-A601, and the company expects to start a Phase 1 dose-escalation trial for the candidate targeting six adults with PKP2-ACM.
The companies with Fast Track Designations, subject to conditions, can secure the FDA’s Accelerated Approval and Priority Review, enabling patients to access those treatments sooner.
In addition to tax credits for clinical trial costs and waiver of the user fee for marketing applications, the developers of orphan drugs can claim seven years of marketing exclusivity in the U.S. after regulatory approval.
More on Rocket
- Rocket's gene therapy RP-L301 for rare blood disorder gets FDA regenerative medicine tag
- Rocket Pharmaceuticals: A Catalyst-Rich Year Ahead
For further details see:
Rocket Pharma wins key FDA designations for heart disease candidate