Rare Daily Staff

The U.S. Food and Drug Administration has granted Aruvant Sciences the Rare Pediatric Disease designation for ARU-1801 for the treatment of sickle cell disease.

Sickle cell disease is a progressively debilitating and life-threatening inherited red blood cell disorder that causes a patient’s oxygen-carrying cells to be abnormally inflexible and sickle-shaped. This genetic defect leads to lowered oxygen-carrying capacity in the hemoglobin protein of these red blood cells. Sickle cell disease can cause inflammation of extremities, bacterial infections, stroke, anemia, and attacks of pain called sickle cell crises.

Aruvant’s lead candidate, ARU-1801, is being developed as a potential one-time treatment for patients suffering from sickle cell disease and β-thalassemia. It utilizes proprietary technology intended to increase functioning red blood cells by inserting a modified fetal hemoglobin gene into autologous stem cells through a lentiviral vector.

Studies have indicated that sickle cell patients with elevated levels of fetal hemoglobin have fewer vaso-occlusive crises and hospitalizations, due in part to improved characteristics of fetal hemoglobin relative to adult hemoglobin. ARU-1801 is currently the only known clinical-stage gene therapy to deliver the gene encoding fetal hemoglobin, which has been modified to optimize oxygen carrying capacity and anti-sickling properties.  

ARU-1801 is currently being tested in an ongoing phase 1/2 study in patients with sickle cell disease conducted using a reduced intensity conditioning regimen that is enable by the unique properties of modified fetal hemoglobin at the Cincinnati Children’s Hospital Medical Center.

“The FDA’s Rare Pediatric Disease designation for ARU-1801 highlights the large and unmet need currently experienced by patients suffering from sickle cell disease,” said Will Chou, CEO of Aruvant. “Other investigational gene therapies for sickle cell disease require the use of high intensity myeloablative conditioning regimens, which are associated with lengthy hospital stays and a host of possibly serious complications. Our reduced intensity conditioning approach aims to provide patients a cure with an improved risk-benefit profile, including a lower risk of infertility and fewer days in the hospital.”

The FDA grants Rare Pediatric Disease designation for serious or life-threatening diseases with manifestations in individuals aged from birth to 18 years, including access to the FDA’s expedited review and approval process. The designation makes ARU1801 eligible for a Rare Pediatric Disease Priority Review voucher upon approval of the therapy by the FDA.

The vouchers can be used to reduce the time of an FDA new drug approval review to six months from ten months. The vouchers are potentially lucrative because they are transferable. Most recently, Swedish Orphan Biovitrum sold its Priority Review voucher to AstraZeneca for $95 million.

Photo: Will Chou, CEO of Aruvant

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