- Bluebird bio, a Cambridge, Massachusetts-based drug company, on Friday said it will resume selling one of its gene therapies in Europe after voluntarily halting marketing in February over safety concerns.
- Known as Zynteglo, the therapy was conditionally approved in Europe about two years ago as a treatment for certain people with beta thalassemia, a blood disorder that can negatively affect growth, organ health and red blood cell count. Zynteglo marketing has been on hold, though, since one patient in a clinical trial of another, related Bluebird medicine developed leukemia.
- After an investigation, Bluebird said in March that its medicine was "very unlikely" to have caused the cancer case. European regulators appear to agree. Based on a review of all available data, the European Medicines Agency's Pharmacovigilance Risk Assessment Committee has concluded that Zynteglo's benefits outweigh its risks, leading Bluebird to lift its marketing suspension.
Zynteglo was Bluebird's first product cleared for market. Yet, in the two years since its European approval, the therapy has struggled commercially.
In the European Union, each member state works out how — or if — it's going to pay for medicines approved through the European Commission. But Bluebird has had trouble convincing the states to reimburse Zynteglo, in large part because it set the therapy's initial price at $1.8 million, making it one of the most expensive drugs in the world. Just a few months ago, Bluebird decided to withdraw Zynteglo in Germany, the EU's most populous member, after it couldn't reach an agreement on pricing with health authorities.
Those challenges, combined with delays from manufacturing problems, have resulted in very little use of Zynteglo. It wasn't until February of this year that the first patient was treated outside of a clinical trial. Bluebird ended 2020 with a $619 million net loss on $251 million in revenue, the vast majority of which came from collaborations with larger pharmaceutical companies.
This year, the Zynteglo franchise has also had to contend with issues surrounding an experimental Bluebird medicine called LentiGlobin, which is being studied as a treatment for sickle cell disease.
Zynteglo and LentiGlobin use the same type of viral vector — a tool used to deliver helpful genetic material — to deliver the same engineered beta-globin gene. So when researchers identified a cancer case in an ongoing LentiGlobin study, Bluebird decided to stop treating patients with both therapies until an investigation was conducted.
According to the company, the investigation ultimately found it "very unlikely" that LentiGlobin was tied to the patient's leukemia. By early June, the Food and Drug Administration had given Bluebird the go-ahead to resume two studies of LentiGlobin in sickle cell and two others of Zynteglo in beta thalassemia.
Bluebird affirmed in a Friday statement that no cases of blood cancer have been reported in any patient who's been treated with Zynteglo.
Looking ahead, Bluebird said the recommendation from the Pharmacovigilance Risk Assessment Committee will be sent to two other regulatory committees for adoption. Afterward, it will be up to the European Commission to make a ruling, which would be applicable in all member states.
Bluebird's share value ticked up by as much as 2.5% Friday morning. Since the start of the year, however, the stock has fallen 28%.