Zynteglo, Bluebird Bio’s ex vivo gene therapy for β-thalassaemia, got a green light from the EMA for conditional marketing authorization making it the 1st gene therapy for patients with thalassaemia to win such an approval.
The one-time gene therapy has been approved for patients 12 years and older with transfusion-dependent β-thalassaemia (TDT), and has been shown in a series of small studies to free a majority of patients from the need to have these regular blood transfusions.
This first conditional approval is in the moderately severe TDT form of the disease (and not those with the more acute β0/β0 genotype) for whom haematopoietic stem cell (HSC) transplantation is appropriate but a human leukocyte antigen (HLA)-matched related HSC donor is not available.
There’s no word yet on pricing, which is a thorny issue for gene therapy developers as they try to balance a high upfront cost – sometimes millions of dollars as with Novartis’ new Zolgensma therapy for rare disease spinal muscular atrophy – with what should be long-term clinical benefits that can save health systems money.
The manufacturing company has stated it will carry out a “continue the country-by-country reimbursement process to help ensure access to Zynteglo for appropriate patients,” adding that it plans to reveal more detail on its pricing plans shortly.
Read the company’s full Press Release