Only three weeks after gaining approval in Europe for its gene therapy treatment Lenti-D (elivaldogene autotemcel or eli-cel), bluebird bio has announced it will “scale back operations in Europe to focus on the US market”.
The announcement comes after its trial in the rare neurological condition cerebral adrenoleukodystrophy (CALD) was halted after a boy was diagnosed with myelodysplastic syndrome, a condition that can develop into leukaemia.
The company said that current evidence suggested that “specific design features” of its therapy “contributed to this event”.
“Our hearts go out to this patient and his family, who are dealing with a challenging diagnosis,” said bluebird bio chief executive, Nick Leschly. “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.”
Two other patients are also being monitored, and the FDA has placed the eli-cel programme on clinical hold.
This is not the first such incident for bluebird bio’s gene therapies. Earlier this year, two clinical studies in sickle cell disease were put on hold after a patient developed leukaemia and another was diagnosed with myelodysplastic syndrome. The trials were later resumed when the case of myelodysplastic syndrome was assessed to be a misdiagnosis and the cancer case was “very unlikely” to be linked to bluebird bio’s therapy.
However, this latest setback for the company will inevitably raise questions about the future of gene therapy. Shares in bluebird bio dropped 25.9% on the latest news, meaning its stock has now fallen 57.4% so far this year.
The company’s situation in Europe was already difficult following its failure to reach agreements with governments on pricing for its one-time treatments. While the company has not announced how much it asked for Lenti-D, earlier this year, it withdrew its beta-thalassemia therapy from Germany after the government offered $790,000 upfront, rising to $950,000 if the therapy worked after five years. Even though the price was in line with analysts’ expectations, bluebird bio asked for $1.8 million per patient.
While announcing the company’s Q2 results, bluebird bio’s president of severe genetic diseases Andrew Obenshain told investors that continuing to market therapies as a small biotech would have been “untenable”. The company now aims to either license out US rights to its gene therapies or find other ways to make them available on the continent.
Analysts are reacting to the news. SVB Leerink analyst, Mani Foroohar, said: “The aborted launch in the EU raises questions on commercial execution and management’s understanding of the reimbursement landscape in key end-markets.” He added that the closure of its EU operations effectively returns bluebird bio to a “pre-commercial state”.
At present, only one its therapies, cancer treatment Abecma (marketed in partnership with Bristol Myers Squibb), has received approval in the US.
The company will continue its planned split into two independent, publicly traded companies – bluebird bio and 2seventy bio – by the end of 2021.