Biologics

Gene Therapy OK’d for Blood Disorder

FDA has approved a bluebird bio BLA for its lentiviral vector gene therapy Zynteglo (betibeglogene autotemcel) (beti-cel) for beta-thalassemia. The agency says Zynteglo is the first cell-based gene therapy for treating adult and pediatric patients with beta-thalassemia who require regular red blood cell transfusions.

The inherited blood disorder causes a reduction in normal hemoglobin and red blood cells that leads to insufficient delivery of oxygen in the body, according to FDA. “The reduced levels of red blood cells can lead to a number of health issues including dizziness, weakness, fatigue, bone abnormalities and more serious complications,” it says. “Transfusion-dependent beta-thalassemia, the most severe form of the condition, generally requires life-long red blood cell transfusions as the standard course of treatment.”

Zynteglo is a one-time gene therapy product administered as a single dose. Approval was based on data that measured transfusion independence, which is attained when the patient maintains a pre-determined level of hemoglobin without needing any red blood cell transfusions for at least 12 months, the agency says, adding that out of 41 patients receiving Zynteglo, 89% achieved transfusion independence.

On the safety side, FDA said the most common adverse reactions associated with the therapy included reduced platelet and other blood cell levels, as well as mucositis, febrile neutropenia, vomiting, pyrexia (fever), alopecia (hair loss), epistaxis (nosebleed), abdominal pain, musculoskeletal pain, cough, headache, diarrhea, rash, constipation, nausea, decreased appetite, pigmentation disorder and pruritus (itch).

“There is a potential risk of blood cancer associated with this treatment; however, no cases have been seen in studies of Zynteglo,” it says. “Patients who receive Zynteglo should have their blood monitored for at least 15 years for any evidence of cancer. Patients should also be monitored for hypersensitivity reactions during Zynteglo administration and should be monitored for thrombocytopenia and bleeding.”

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