Aruvant receives EMA orphan designation for sickle cell gene therapy

By The Science Advisory Board staff writers

November 16, 2020 -- Aruvant has been granted orphan drug designation for ARU-1801, an investigational lentiviral gene therapy by the European Medicines Agency (EMA).

The company has been previously granted orphan drug designation and rare pediatric disease designations for the therapy for the treatment of sickle cell disease by the U.S. Food and Drug Administration.

ARU-1801 is a one-time potentially curative investigational gene therapy for individuals living with sickle cell disease. The therapy incorporates a patented modified gamma-globin into autologous stem cells, with the goal of restoring red blood cell function through increased fetal hemoglobin. ARU-1801 engraftment is achieved with only reduced intensity conditioning.

The EMA's decision was based on a positive opinion issued by the EMA Committee for Orphan Medicinal Products. Preliminary clinical data from the Momentum study, an ongoing phase I/II trial, demonstrated durable reductions in disease burden.

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