Among a second quarter results announcement full with setbacks, bluebird bio has revealed that it will exit the European market after failing to secure access for its gene therapy with various governments.
In a statement, the US biotech company’s president of severe genetic diseases Andrew Obenshain said bluebird will instead focus on the US market, highlighting the ‘challenges of achieving appropriate value recognition and market access for Zynteglo [betibeglogene autotemcel] in Europe’.
Bluebird received conditional marketing authorisation for Zynteglo in Europe in June 2019 – since then, it has failed to secure access agreements for the beta thalassaemia gene therapy in the market.
That includes the UK’s National Institute for Health and Care Excellence (NICE), which chose not to recommend the beta thalassaemia gene therapy for use on the NHS in February 2021.
NICE determined that there were uncertainties around the cost-effectiveness of Zynteglo – also known as beti-cel – with the estimate for the gene therapy ‘considerably higher’ than what it usually deems an acceptable use of NHS resources.
Looking ahead, bluebird said that it will be exploring options to ensure European patients can gain access to its gene therapies, including potentially out-licensing the ex-US rights to its three lead products to a company ‘with European experience and capabilities’.
“While European regulators have been innovative partners in supporting accelerated regulatory paths for these therapies, European payers have not yet evolved their approach to gene therapy in a way that can recognise the innovation and the expected life-long benefit of these products,” added Obenshain.
Elsewhere in its second quarter update, bluebird said that it had received a reported suspected unexpected serious adverse reaction (SUSAR) of myelodysplastic syndrome (MDS) in a patient treated with its CALD gene therapy eli-cel.
Bluebird added that the SUSAR is ‘likely mediated by Lenti-D lentiviral vector insertion’, with current evidence suggesting the design features of the vector most likely contributed to the event.
“Our hearts go out to this patient and his family, who are dealing with a challenging diagnosis,” said Nick Leschly, chief executive officer of bluebird.
“Given what we know, we remain confident that eli-cel can offer hope for patients and families impacted by this devastating disease who have very few treatment options. We are committed to working with regulators and physicians in order to resolve this hold as soon as possible and bring this important therapy to patients in need,” he added.