The EMA’s Committee for Medicinal Products for Human Use (CHMP) recommended approval of Bluebird’s Zynteglo, for transfusion-dependent beta thalassemia, which can lead to severe anemia. The therapy is recommended for patients who are at least 12 years old who need regular transfusions and who can’t find a stem cell donor. If the approval is granted, it would be the first gene therapy of its kind in Europe.
The committee’s recommendation, which now passes to the EMA, came after two clinical trials showed the majority of patients who took the medicine were able to get off the transfusion treadmill.
Bluebird has high expectations for Zynteglo, saying that a one-time infusion is worth about $2.1 million per patient. The company hopes to have full European approval later this year.