Wednesday, May 24, 2023
Rocket Pharmaceuticals announced that the U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to RP-L301, the investigational lentiviral-based gene therapy for Pyruvate Kinase Deficiency (PKD), a rare blood disorder characterised by severe anaemia and excessive red blood cell breakdown.
Results from the RP-L301 programme demonstrated robust efficacy in both adult patients for up to 30 months post-infusion demonstrated by normalised haemoglobin (from baseline levels in the 7.0-7.5 g/dL range), improved haemolysis parameters, red blood cell transfusion independence and improved quality of life with documented improvements via formal quality of life assessments.
Receiving RMAT designation from the FDA for RP-L301 is a major achievement to bring the first potentially curative gene therapy treatment to patients living with PKD who have high unmet need.
Pyruvate Kinase Deficiency (PKD) is a rare, monogenic red blood cell disorder resulting from a mutation in the PKLR gene encoding for the pyruvate kinase enzyme, a key component of the red blood cell glycolytic pathway. Patients with PKD have a high unmet medical need, as currently available treatments include splenectomy and red blood cell transfusions, which are associated with immune defects and chronic iron overload.
