Shares of Rocket Pharmaceuticals
RCKT,
gained 2.3% premarket on Thursday after the late-stage biotech company said the U.S. Food and Drug Administration granted fast-track and orphan-drug designations to its gene therapy candidate for treatment of an inherited heart disease. Rocket’s RP-A601 gene therapy is designed to treat plakophilin-2 related arrhythmogenic cardiomyopathy, which is caused by genetic mutations and is associated with cardiac structural abnormalities and sudden cardiac death. FDA’s fast-track designation is designed to streamline development and review of drug candidates for serious conditions when there’s an unmet medical need. The orphan drug designation is available for drugs intended to treat or prevent rare diseases and comes with certain benefits such as tax credits for qualified clinical trials.
