Aruvant Receives Orphan Designation from European Medicines Agency for Gene Therapy ARU-1801 for Sickle Cell Disease
November 16, 2020

NEW YORK and BASEL, Switzerland, Nov. 16, 2020 /PRNewswire/ -- 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.

About ARU-1801
ARU-1801 is a one-time potentially curative investigational gene therapy for individuals living with sickle cell disease. This product candidate was designed to address the limitations of current treatment options including myeloablative chemotherapy toxicity, donor availability, and chronic administration, and replace it with a curative therapy. ARU-1801 incorporates a patented modified gamma-globin into autologous stem cells, with the aim of restoring normal red blood cell function through increased levels of fetal hemoglobin. The high potency of the modified gamma globin enables ARU-1801 engraftment with only reduced intensity conditioning (RIC). Preliminary clinical data from the MOMENTUM study, an ongoing Phase 1/2 trial of ARU-1801 in patients with severe sickle cell disease, demonstrate continuing durable reductions in disease burden.

About Aruvant
Aruvant Sciences, part of the Roivant family of companies, is a clinical-stage biopharmaceutical company focused on developing and commercializing gene therapies for the treatment of rare diseases. The company has a talented team with extensive experience in the development, manufacturing and commercialization of gene therapy products. Aruvant has an active research program with a lead product candidate, ARU-1801, in development for individuals suffering from sickle cell disease (SCD). ARU-1801 is an investigational lentiviral gene therapy currently in a Phase 1/2 clinical trial, the MOMENTUM study, as a potential one-time curative treatment for SCD. Preliminary clinical data demonstrate engraftment of ARU-1801 and amelioration of SCD is possible with one dose of low intensity chemotherapy. For more information on the clinical study, please visit and for more on the company, please visit

About Roivant
Roivant aims to improve health by rapidly delivering innovative medicines and technologies to patients. Roivant does this by building Vants – nimble, entrepreneurial biotech and healthcare technology companies with a unique approach to sourcing talent, aligning incentives, and deploying technology to drive greater efficiency in R&D and commercialization. For more information, please visit .

SOURCE Aruvant Sciences

For further information: E. Blair Clark-Schoeb, SVP, Communications,