The UK’s National Institute for Health and Care Excellence (NICE) has rejected bluebird bio’s gene therapy Zynteglo for patients with transfusion-dependent beta-thalassaemia (TDT) in draft guidance.
The draft guidance, published today, said that clinical trials evaluating Zynteglo (betibeglogene autotemcel; beti-cel) were ‘small’, with participants not having been followed up for ‘very long’.
NICE did add, however, that these trials suggest that eventually certain TDT patients treated with Zynteglo may no longer require blood transfusion or may require them less often.
bluebird’s main clinical evidence for Zynteglo came from phase 1/2 studies of the gene therapy in TDT patients with non-beta0/beta0 genotypes.
The primary endpoint of these studies was transfusion independence (TI), with the secondary endpoint being transfusion reduction.
In these trials, data from a total of 24 participants was available for evaluation for TI at the time of the submission, with 83% having reached TI.
Of the remaining participants who had not reached TI, two had ‘substantial’ transfusion reductions and two were transfusion dependent.
NICE also determined that there were uncertainties around the cost-effectiveness of Zynteglo, with the cost estimate for the gene therapy ‘considerably higher than what NICE normally considers an acceptable use of NHS resources’.
The cost of a one-off infusion of Zynteglo is £1.45m, although bluebird has a commercial arrangement which would have been applied if NICE had recommended the therapy.
TDT is a hereditary disease caused by mutation in the β-globin gene that results in significantly reduced or absent adult haemoglobin. Those who suffer with this condition depend on blood transfusions to maintain haemoglobin levels throughout their lifetime.
The one-off gene therapy was approved by the European Medicines Agency (EMA) in June 2019, for patients 12 years and older with transfusion-dependent beta thalassaemia (TDT) for whom haematopoietic stem cell (HSC) transplantation is appropriate but a human leukocyte antigen (HLA)-matched related HSC donor is not available.
“We are shocked and disappointed by this recommendation and strongly believe that NICE has failed to act in the best interests of people with TDT and their families in England and Wales,” said Nicola Redfern, UK general manager at bluebird bio.
“It is baffling that NICE disregarded the testimonies of patients, advocacy groups and clinicians and, despite recognising beti-cel as a potential cure for patients with TDT, has chosen to deny access and has dismissed the obvious unmet need,” she added.
NICE’s draft guidance of Zynteglo is open for consultation until 4 March 2021.