By Chris Wack

Rocket Pharmaceuticals said Tuesday that the U.S. Food and Drug Administration has granted a regenerative medicine advanced therapy designation to RP-L301, the company's investigational lentiviral-based gene therapy for pyruvate kinase deficiency.

The Cranbury, N.J.-based late-stage biotechnology company said RMAT designation was granted based on RP-L301's robust safety and efficacy data from an ongoing Phase 1 clinical trial, and its potential to cure a life-threatening disease for which no curative therapies currently exist.

The RMAT designation will provide the benefits of added FDA guidance and expedited review throughout the RP-L301 program's development. RP-L301 has also received the FDA's fast track and orphan drug designations.

Rocket Pharma said RP-L301's safety profile appears highly favorable, with no RP-L301-related serious adverse events in either of the adult patients.

The program's first pediatric patient results suggest efficacy similar to the adult cohort with an initial greater than a five-point increase in hemoglobin. The infusion was well-tolerated, and no RP-L301-related serious adverse events or red blood cell transfusion requirements followed engraftment.

Write to Chris Wack at chris.wack@wsj.com

(END) Dow Jones Newswires

May 23, 2023 07:36 ET (11:36 GMT)

Copyright (c) 2023 Dow Jones & Company, Inc.
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