FDA Approves Betibeglogene Autotemcel for Beta-Thalassemia Treatment in Adults and Children

Approval has been granted by the FDA to betibeglogene autotemcel for the treatment of patients with beta-thalassemia based on multiple phase 3 studies.

The FDA has granted approval to betibeglogene autotemcel (Beti-cel; Zynteglo) for the treatment of adult and pediatric patients with beta-thalassemia who require regular red blood cell transfusions. Beti-cel is the first cell-based gene therapy for the treatment of this patient population.1

Approval was granted to the agent based on results from 5 studies, including the phase 3 Northstar-2 (NCT02906202) and Northstar-3 (NCT03207009) studies, and the phase 1/2 studies Northstar (NCT01745120) and HGB-205 (NCT02151526), and the long-term LTF-303 (NCT02633943).2

In the collective studies, 89% of evaluable patients, including pediatric patients and those with the most severe genotypes, achieved transfusion independence. Transfusion independence was defined as no longer needing red blood cell transfusions for at least 12 months while maintaining a weighted average hemoglobin of at least 9 g/dL.

“Today’s approval is an important advance in the treatment of beta-thalassemia, particularly in individuals who require ongoing red blood cell transfusions,” said Peter Marks, MD, PhD, director of the FDA’s Center for Biologics Evaluation and Research, in a press release. “Given the potential health complications associated with this serious disease, this action highlights the FDA’s continued commitment to supporting development of innovative therapies for patients who have limited treatment options.”

Beti-cel is intended to be a one-time gene therapy product administered as a single dose. It was a custom creation from the patient’s own cell that are then genetically modified to be able to develop bet-globin.

According to the collective research, the most common adverse events observed with beti-cel include reduced platelet and other blood cell levels, as well as mucositis, febrile neutropenia, vomiting, pyrexia, alopecia, epistaxis, abdominal pain, musculoskeletal pain, cough, headache, diarrhea, rash, constipation, nausea, decreased appetite, pigmentation disorder and pruritus. There is also a possibility of blood cancer associated with beti-cel. The FDA recommends that patients receiving the drug be monitored for at least 15 years signs of cancer.


1. FDA approves first cell-based gene therapy to treat adult and pediatric patients with beta-thalassemia who require regular blood transfusions. News release. FDA. August 17, 2022. Accessed August 17, 2022. https://bit.ly/3wbgprn

2. bluebird bio announces FDA priority review of biologics license application for beti-cel gene therapy for patients with β-thalassemia who require regular red blood cell transfusions. News release. bluebird bio. November 22, 2021. Accessed August 17, 2022. https://bit.ly/32trmIT