Rocket Pharmaceuticals gets FDA approval for PKD gene therapy


Rocket Pharmaceuticals, Inc. has announced that the US Food and Drug Administration (FDA) has awarded Regenerative Medicine Advanced Therapy (RMAT) designation for RP-L301, a lentiviral-based gene therapy that the company is currently investigating for pyruvate kinase (PKD) deficiency.

PKD is a rare blood disorder characterized by severe anemia and excessive breakdown of red blood cells.

The RMAT designation was awarded based on strong safety and efficacy data from ongoing Phase 1 clinical trials of RP-L301 and its potential to cure life-threatening diseases for which there are currently no curative therapies. The designation will benefit from additional FDA guidance and expedited review through program development.

“Receiving the FDA’s RMAT designation for RP-L301 is a major accomplishment in our quest to bring the first potentially curative gene therapy treatment to patients living with PKD who have high unmet needs. Notably, PKD has an estimated prevalence of up to 8,000 patients in the US and Europe and is one of the most significant patient opportunities in our LV hematology portfolio,” said Kinnari Patel, president and chief operating officer, Rocket Pharmaceuticals.

“Furthermore, all four Rocket-sponsored programs with clinical data have now received RMAT designation from the FDA on both platforms, a unique showcase of our team’s ability to select the right targets and develop gene therapies for them.”

Patel said the results from the RP-L301 program demonstrated strong efficacy in both adult patients up to 30 months with a very favorable safety profile and were recently presented at the ASGCT.

“The first pediatric patient has shown promising early results similar to adults, and enrollment has been completed in the phase 1 study. We look forward to starting the critical phase 2 trial in the fourth quarter of 2023 as we continue to advance our world-class pipeline for patients who dealing with this rare and deadly disease.

RMAT designation

Established under the 21st Century Cures Act, the RMAT designation is designed to accelerate the drug development and review process for promising pipeline products, including gene therapies. Regenerative drug therapy qualifies for the 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 an unmet medical need for such disease or condition. That.

Apart from RMAT, the RP-L301 has also received Fast Track and Orphan Drug Designation.

About Rocket Pharmaceuticals’ RP-L301 safety profile

Results from a phase 1 program presented recently at the American Society of Gene and Cell Therapy (ASGCT) 26th Annual Meeting demonstrated robust and sustained efficacy in both adult patients up to 30 months post infusion demonstrated by normalized hemoglobin (from baseline levels in the range of 7.0-7.5 g/dL), better hemolysis parameters, red blood cell transfusion independence and improved quality of life with documented improvement through formal quality of life assessments.

The safety profile appeared to be favorable, with no serious side effects associated with RP-L301 in one of our adult patients.

The results of the first pediatric patient demonstrated similar efficacy to the adult cohort with baseline hemoglobin increases of more than five points (from a mean initial level of 7.9 g/dL). The infusion was well tolerated, with engraftment achieved at day +15, discharge from hospital less than one month after infusion, and no serious adverse events related to RP-L301 or RBC transfusion requirements after engraftment.

About pyruvate kinase deficiency

PKD is a rare monogenic red blood cell disorder resulting from mutations in the PKLR gene that encodes the enzyme pyruvate kinase, a key component of the red blood cell glycolytic pathway. Mutations in the PKLR gene result in increased red blood cell destruction and abnormalities range from mild to life-threatening anemia.

PKD has an estimated prevalence of 4,000 to 8,000 patients in the US and Europe. Children are the most frequent and severely affected patient subgroup. Patients with PKD have high unmet medical needs, because currently available treatments include splenectomy and red cell transfusion, which are associated with impaired immunity and chronic iron overload.

Recently, mitapivat, an oral enzyme activator, has been approved for use in adult patients, but its efficacy is limited in more severely ill patients, especially those who are splenectomy, transfusion dependent, or whose disease is caused by deleterious mutations.

The RP-L301 is licensed by the Center for Energy, Environment and Technology Research (CIEMAT), the Biomedical Network Research Center for Rare Diseases (CIBERER) and the Jiménez Díaz Foundation Health Research Institute (IIS-FJD).

Earlier this year, Rocket Pharmaceuticals received the FDA RMAT designation for the gene therapy treatment of Danon’s disease.


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