Pharma Focus Asia

UCB Collaborates with Veeva Systems

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.

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