Blue­bird's one-shot gene ther­a­py for be­ta tha­lassemia of­fi­cial­ly se­cures EMA back­ing — ap­proval ex­pect­ed in the sec­ond quar­ter

Days af­ter two char­i­ties abrupt­ly pre­an­nounced that the EMA had en­dorsed the ap­proval of blue­bird bio’s keen­ly an­tic­i­pat­ed gene-re­place­ment ther­a­py Lenti­Glo­bin, for be­ta tha­lassemia — a rare, in­her­it­ed blood dis­or­der — the Eu­ro­pean reg­u­la­tor made the rec­om­men­da­tion of­fi­cial on Fri­day.

The one-shot ther­a­py for this pa­tient pop­u­la­tion — who usu­al­ly re­quire life­long treat­ment with blood trans­fu­sions and med­ica­tion — is ex­pect­ed to win EC ap­proval by the sec­ond quar­ter, and US ap­proval next year.

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