The US Food and Drug Administration (FDA) approved the company’s one-time beta thalassaemia treatment Zynteglo, also known as betibeglogene autotemcel (beti-cel), two days ahead of the regulator’s approval action date of August 19.
The green light is “monumental” for both patients and the company itself, Tom Klima, Bluebird’s Chief Commercial Officer, said in an interview. Bluebird has endured its share of “bumps and bruises over the last year-and-a-half or so,” including the therapy’s withdrawal from Europe, but things have been looking up since a positive FDA advisory committee meeting earlier this summer, Klima said.
The approval clears the drug for kids and adults with beta thalassaemia who require regular red blood cell transfusions, the FDA said in a release. As a one-time bespoke treatment, each Zynteglo dose is created by genetically modifying a patient’s own bone marrow stem cells. Specifically, the patient’s cells are modified to produce functional beta-globin, which is a component of haemoglobin.
Across two clinical studies looking at adults and kids with transfusion-dependent beta thalassemia, 89% of the 41 patients on Zynteglo achieved transfusion independence, the FDA said.
Regarding launch plans, bluebird aims to bring its first wave of qualified treatment centers online by September, Klima said. Meanwhile, bluebird plans to start collecting patients’ cells by 2022’s fourth quarter, with the aim to kick off treatment of its first patients in early 2023.
Zynteglo will roll out at a price of $2.8 million per patient, Klima told Fierce Pharma, adding that the company feels that price tag “reflects the value of a one-time treatment in the U.S.” The list price doesn’t include the cost of hospitalization required for patients receiving the therapy Stat News noted.