The U.S. Food and Drug Administration (FDA) has approved betibeglogene autotemcel, a one-time gene therapy to treat patients with beta‑thalassemia who require regular red blood cell (RBC) transfusions. This is the first gene therapy to be approved for this condition.
Beta-thalassemia is a rare bleeding disorder caused by mutations in the beta-globin gene. Patients with the most severe form (beta-thalassemia major) experience severe anemia and lifelong dependence on regular red blood cell transfusions every 2-5 weeks. There are approximately 1,300-1,500 individuals with beta-thalassemia major in the U.S. Due to the small number of patients and the need for only one treatment to cure the patient, the cost of the therapy is expected to be high.
The approval of the gene therapy was largely dependent on the a number of clinical trials, including Phase 3 studies HGB-207 (Northstar-2) and HGB-212 (Northstar-3), and the long-term follow-up study LTF-303. To date, 89% (32/36) of evaluable patients in the studies that have received the gene therapy achieved transfusion independence.
The most common non-laboratory adverse reactions (≥20%) were mucositis, febrile neutropenia, vomiting, pyrexia, alopecia, epistaxis, abdominal pain, musculoskeletal pain, cough, headache, diarrhea, rash, constipation, nausea, decreased appetite, pigmentation disorder, and pruritus. The most common Grade 3 or 4 laboratory abnormalities (>50%) include neutropenia, thrombocytopenia, leukopenia, anemia, and lymphopenia.
Due to the complexity of administering this ex vivo gene therapy, it will be available exclusively at Qualified Treatment Centers (QTCs) based on their expertise in relevant areas such as stem cell transplantation, cell and gene therapy, and beta-thalassemia; and receive specialized training to administer betibeglogene autotemcel. Information on the QTC network will be available at mybluebirdsupport.com.
The therapy is approved for pediatric and adults. As such, the company (bluebird bio) received was a Priority Review voucher upon approval. These vouchers can be used for future applications to the FDA or sold to another company. The vouchers can be used for future new drug applications with the FDA to reduce the time for an application review from the standard 10 months to six months. More commonly, the vouchers are sold by smaller companies such as bluebird bio to larger companies wanting a competitive edge. Last year, Rhythm Pharmaceuticals sold their Priority Review Voucher to Alexion for $100 million.
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