FDA Accepts for Priority Review Gene Therapy for Rare Blood Disorder

The FDA has accepted Bluebird Bio’s biologics license application (BLA) for betibeglogene autotemcel (beti-cel) for priority review. Beti-cel is a gene therapy for adult, adolescent and pediatric patients with β-thalassemia who require regular red blood cell (RBC) transfusions. If approved, beti-cel will be the first one-time treatment that addresses the underlying genetic cause of disease for patients living with β-thalassemia. The agency has set a Prescription Drug User Fee Act (PDUFA) goal date of May 20, 2022.

The application is based on data from bluebird bio’s phase 3 studies, including Northstar-2 and Northstar-3, as well as the phase 1/2 Northstar and HGB-205 studies, and the long-term follow-up study LTF-303. Together, these studies represent more than 220 patient-years of experience with beti-cel.

Beti-cel adds functional copies of a modified form of the β-globin gene into a patient’s own hematopoietic stem cells in order to correct the deficiency of adult hemoglobin that is the hallmark of β-thalassemia. After treatment, patients have the potential to produce beti-cel-derived adult hemoglobin at levels that may eliminate the need for transfusions.

Anne-Virginie Eggimann

“For too long, people with β-thalassemia who rely on regular transfusions have had to live with extraordinary burdens associated with their disease. beti-cel works uniquely to help patients produce adult hemoglobin at normal or near-normal levels, which can eliminate their need for chronic transfusions and chelation that only temporarily relieve the symptoms of anemia and are associated with serious health risks and reduced quality of life,” Anne-Virginie Eggimann, chief regulatory officer, Bluebird Bio, said in a statement.

β-thalassemia is a severe genetic disease caused by mutations in the β-globin gene, which may cause significantly reduced adult hemoglobin. This can result in severe anemia, and patients require red blood cell transfusions every three to four weeks.

Worldwide, about 1.5% of people are β-thalassemia carrier, and occurs most frequently in people from Mediterranean countries, North Africa, the Middle East, India, Central Asia, and Southeast Asia. In the United States, the prevalence of β-thalassemia has increased about 7.5% over the last 50 years, according to researchers.