Bluebird Bio plans to bring its gene therapies to market in Europe before the U.S., thanks to a favorable regulatory pathway.
Bluebird’s head of Europe, Andrew Obenshain, told the Daily Telegraph that the company is already in negotiations with the EMA and the U.K.’s Medicine and Healthcare products Regulatory Agency (MHRA) on possible regulatory filings.
The EMA’s adaptive pathways process—which allows new therapies to be approved in stages based on stepwise collection of data—is a key part of that decision, as is the fact that the agency “works very closely with companies coming forward with new methodologies,” said Morgan. And with Brexit looming, it makes sense to discuss these plans with the MHRA separately.
Two years ago, Bluebird—which targets severe genetic diseases and cancer—was hit hard when the NorthStar trial of lead therapy LentiGlobin failed to hit the mark in sickle cell disease and beta thalassemia, mainly because of variable patient responses to the treatment.
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