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bluebird bio’s beti-cel accepted for priority review by FDA

The tough journey to approval for beta-thalassemia gene therapy (beti-cel) in the US may be coming to an end, with approval possible by 2022

bluebird bio

The US Food and Drug Administration (FDA) has finally accepted bluebird bio’s gene therapy beti-cel (betibeglogene autotemcel) for priority review after years of setbacks.

The one-off gene therapy is for adult, adolescent and paediatric patients with beta-thalassemia across all genotypes who require regular red blood cell (RBC) transfusions. If approved, the therapy – known as Zynteglo in Europe – will be the first one-time treatment that addresses the underlying genetic cause of disease for patients living with β-thalassemia.

Beta-thalassemia is a severe genetic disease where mutations cause significantly reduced haemoglobin levels, which can result in severe anaemia and lifelong dependence on blood transfusions. While transfusions temporarily relieve the symptoms of anaemia, including fatigue, weakness and shortness of breath, they do not address the underlying genetic cause of the condition and can have serious side effects.

By adding functional copies of a modified form of the mutant gene into a patient’s own haematopoietic (blood) stem cells, beti-cel (pronounced BEH-tee-cell) can correct the haemoglobin deficiency that causes the condition.

The FDA priority review ruling is based on data from two phase 3 studies of the therapy, Northstar-2 and Northstar-3, as well as a long-term follow-up study LTF-303. Togetherthe studies represent more than 220 patient-years of experience, following 63 paediatric, adolescent and adult patients for more than seven years.

“For too long, people with beta-thalassemia who rely on regular transfusions have had to live with extraordinary burdens associated with their disease,” said Anne-Virginie Eggimann, chief regulatory officer at bluebird bio.

“Beti-cel works uniquely to help patients produce adult haemoglobin at normal or near-normal levels, which can eliminate their need for chronic transfusions and chelation that only temporarily relieve the symptoms of anaemia and are associated with serious health risks and reduced quality of life,” she added.

It has been a long road to this point for Massachusetts-based bluebird bio, which reported its first phase 3 data back in June 2017 and gained approval in Europe two years later. Bbluebird bio has since withdrawn its entire gene therapy portfolio from the European market citing the reluctance of European healthcare systems to pay its eye-popping $1.8m price tag.

In Germany, the only country where the pricing details are known, the authorities offered to pay $790,000 per patient, rising to $950,000 after five years if the therapy is still working. Bbluebird bio insisted on receiving $1.8m per patient although the amount could be paid over five years conditional on the patient’s response to the therapy.

Article by
Hugh Gosling

23rd November 2021

From: Research



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