Rocket Pharmaceuticals Obtains FDA RMAT Designation for Pyruvate Kinase Deficiency Gene Therapy

Kadeja Johnson
25 May 2023
Gene Therapy
Rocket Pharmaceuticals Receives FDA Regenerative Medicine Advanced Therapy (RMAT) Designation for RP-L301 Gene Therapy for Pyruvate Kinase Deficiency (PKD), a rare blood disorder characterized by severe anemia and excessive red blood cell breakdown.

Leading late-stage biotechnology company, Rocket Pharmaceuticals, Inc., (Rocket Pharma), advancing an integrated and sustainable pipeline of genetic therapies for rare disorders, announced that the FDA has granted Regenerative Medicine Advanced Therapy (RMAT) designation to their investigational lentiviral-based gene therapy for Pyruvate Kinase Deficiency (PKD), RP-L301. RP-L301 reportedly has the potential to cure PKD, for which currently no curative therapies exist.

PKD is a rare blood disorder. It affects an estimated 4000 – 8000 patients across the U.S and Europe. This life-threatening genetic disorder results from a mutation in the PKLR gene encoding for the pyruvate kinase enzyme causing increased red blood cell destruction.

Currently, for patients with this rare blood disorder available treatments are limited and include splenectomy and red blood cell transfusions, which are associated with immune defects and chronic iron overload.

The RMAT designation was established based on safety and efficacy data from the ongoing Phase I RP-L301 clinical trial which will provide FDA guidance and expedited review through the development of the program. According to the 21st Century Cures Act, ‘Regenerative medicine therapy is eligible for RMAT designation if it is intended to treat, modify, reverse or cure a serious or life-threatening disease or condition, and preliminary clinical evidence indicates that the drug or therapy has the potential to address unmet medical needs for such disease or condition.’

“Receiving RMAT designation from the FDA for RP-L301 is a major achievement in our pursuit to bring the first, potentially curative gene therapy treatment to patients living with PKD who have high unmet need. Notably, PKD has an estimated prevalence of up to 8,000 patients in the US and Europe and represents one of the most significant patient opportunities in our LV hematology portfolio,” noted Kinnari Patel, PharmD, MBA, President and CEO of Rocket Pharma.

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Results from Phase I ‘demonstrated robust efficacy in both adult patients for up to 30 months with a highly favorable safety profile’ and were presented at the 26th Annual Meeting of the American Society of Gene and Cell Therapy (ASCGT).

“The first pediatric patient has shown promising initial results similar to the adults, and enrollment has been completed in the Phase I study. We look forward to initiating the Phase II pivotal trial in the fourth quarter of 2023 as we continue to advance our world-class pipeline for patients facing such rare and devastating diseases,” concluded Dr. Patel.

Having provided sufficient preclinical data for its potential to treat the unmet need of PKD, Rocket Pharma’s RP-L301 new RMAT status joins the Fast Track and Orphan Drug Designation’s it already holds.

Source: Rocket Pharmaceuticals, Inc. Press Release

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