EU stamps historic OK on bluebird’s gene therapy for β-thalassemia — now sit back and wait for the price
It’s official: bluebird bio has scored its first drug approval with an EU marketing authorization for its gene therapy for transfusion-dependent β-thalassemia.
Zynteglo is the new name for a treatment once known as LentiGlobin, a process that takes CD34+ cells from a patient’s hematopoietic stem cells, inserts functional copies of the βA-T87Q-globin gene, then reinfuses the modified HSCs. Patients are given chemotherapy to prime their bone marrows before the reinfusion.
It’s a one-shot therapy designed to replace a lifetime of transfusions and medication, indicated for patients 12 years or older. The approval follows an EMA recommendation in March.
The Cambridge, MA-based biotech plans to provide an update on the roll out plans and pricing by the end of next week, a spokesperson confirmed.
No matter the figure, the sticker price is likely to trigger a fresh wave of debate on pricing of supposedly once-and-done therapies while Novartis’ decision to charge $2.1 million for its spinal muscular atrophy treatment still reverberates.
While bluebird has produced compelling results — dramatically reducing the need for blood transfusions in a 22-patient study and eliminating it in most of the patients with a less severe form of the disease — Zynteglo has fallen short of a cure for more severe patients. Phase III trials are ongoing.
Chief executive Nick Leschly has previously proposed a plan to pay for the therapy by installment and stated that Zynteglo will be priced lower than its “intrinsic value” — coincidentally estimated at $2.1 million
SVB Leerink analysts have previously modeled the launch price of LentiGlobin as $1.2 million in the US and $0.9 million in the EU.
Image: Nick Leschly. International Society for Pharmaceutical Engineering