Cell and gene therapy company bluebird bio became the subject of international confusion Monday amid conflicting reports from the company and two European beta-thalassemia advocacy groups about the regulatory status of one of its lead product candidates.
The UK Thalassaemia Society and Italy’s Associazione Veneta Lotta alla Talassemia – issued a press release Monday stating that the European Medicines Agency had given conditional approval to the gene therapy LentiGlobin for transfusion-dependent beta-thalassemia, or TDT, under the brand name Zynteglo. Later in the day, however, bluebird clarified that the product was still under review as part of an EMA Committee on Human Medicinal Products meeting that started Monday and lasts through Thursday. The bluebird clarification did not include the alleged brand name of LentiGlobin or name the two organizations.
Under EMA policy, the CHMP can issue a recommendation of whether or not to approve a drug, though the approval is up to the European Commission. The EMA covers all the European Union countries and the European Economic Area nations of Norway, Iceland and Liechtenstein. Beta thalassemia is relatively rare in the US, but more prevalent in the Mediterranean, as well as the Middle East, Africa and Asia.
Following the erroneous announcement, an analyst note to investors stated that the supposed decision was within expectations, and that the accelerated assessment LentiGlobin received in July 2018 meant the EMA review time could be reduced from 210 to 150 days. Assuming a positive CHMP opinion, approval would likely take about two months.
The application for EMA approval of LentiGlobin is based on data from two Phase I/II studies, Northstar and HGB-205, the Phase III Northstar-2 study and the long-term follow-up study LTF-303. The most recent data presentation on the gene therapy was for Northstar, at the American Society of Hematology meeting in December. It showed that of 10 TDT patients treated, eight became transfusion-independent. The therapy is also being developed for sickle cell disease, for which it is in a Phase I/II clinical trial.
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