Aruvant Receives Orphan Designation from European Medicines Agency for Gene Therapy ARU-1801 for Sickle Cell Disease

Aruvant, a clinical-stage biopharmaceutical company focused on developing and commercializing transformative gene therapies for the treatment of rare diseases, today announced that the European Medicines Agency (EMA) has granted orphan designation for ARU-1801, an investigational lentiviral gene therapy. The U.S. Food and Drug Administration (FDA) previously granted Orphan Drug and Rare Pediatric Disease Designations to ARU-1801 for the treatment of sickle cell disease (SCD).

“The EMA orphan designation highlights the importance of ARU-1801, our potentially curative gene therapy for individuals living with severe SCD,” said Will Chou, M.D., chief executive officer of Aruvant. “We are encouraged by the preliminary clinical data from our ongoing Phase 1/2 study that demonstrates ARU-1801, administered with only reduced intensity conditioning, can achieve durable reductions in disease burden.”

Orphan drug designation in the European Union (EU) is granted by the European Commission based on a positive opinion issued by the EMA Committee for Orphan Medicinal Products. To qualify, an investigational medicine must be intended to treat a seriously debilitating or life-threatening condition that affects fewer than five in 10,000 people in the EU, and there must be sufficient non-clinical or clinical data to suggest the investigational medicine may produce clinically relevant outcomes. EMA orphan drug designation provides companies with certain benefits and incentives, including clinical protocol assistance, differentiated evaluation procedures for Health Technology Assessments in certain countries, access to a centralized marketing authorization procedure valid in all EU member states, reduced regulatory fees and 10 years of market exclusivity upon marketing authorization.

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