Immuno-Oncology Resource Center
Immuno-Oncology Resource Center
January 10, 2020
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FDA grants rare pediatric disease designation to gene therapy for sickle cell disease

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The FDA granted rare pediatric disease designation to ARU-1801, an investigational autologous gene therapy, for the treatment of sickle cell disease, according to the agent’s manufacturer.

ARU-1801 (Aruvant/Roivant Sciences) is under development as a possible one-time treatment for sickle cell disease and beta-thalassemia.

The investigational therapy aims to increase functioning red blood cells by inserting a modified fetal hemoglobin gene into the patient’s CD34-positive hematopoietic stem cells through a lentiviral vector.

Previous studies have shown that patients with sickle cell disease with elevated levels of fetal hemoglobin have fewer vaso-occlusive crises and fewer hospitalizations than patients who have only adult hemoglobin.

ARU-1801 was designed to produce a potent gene payload with a reduced-intensity conditioning approach. The therapy delivers gene-encoding fetal hemoglobin that has been modified to optimize oxygen-carrying capacity and anti-sickling properties, according to the manufacturer.

“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,” Will Chou, MD, CEO of Aruvant, said in a company-issued press release.

“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,” he said. “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 defines a rare pediatric disease as a disease or condition that is serious or life-threatening and affects fewer than 200,000 children in the United States.

The rare pediatric disease designation makes Aruvant eligible for an FDA priority review voucher for ARU-1801 if the agency approves the investigational therapy’s biologics license application.