Aruvant announced the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease designation to ARU-1801, Aruvant's investigational therapy for the treatment of sickle cell disease.
The FDA defines a rare pediatric disease as a serious or life-threatening disease that impacts fewer than 200,000 individuals in the United States and whose serious or life-threatening manifestations primarily affect individuals 18 years or younger. With this designation, Aruvant will be eligible to receive a priority review voucher upon approval by the FDA of a Biologics License Application for ARU-1801.
"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, M.D., Chief Executive Officer 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 (RIC) 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."
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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.
β-thalassemia is an inherited red blood cell disorder distinguished by reduced or nonexistent production of functional β-globin, a critical component of adult hemoglobin. Patients with the disorder suffer from anemia, which can cause weakness, fatigue, and more serious complications. Individuals with β-thalassemia are at an increased risk of developing abnormal blood clots.