The first gene therapy to treat a rare blood disorder is one step closer to approval Friday following a recommendation by European officials.
Lentiglobin, the gene therapy for beta-thalassemia developed by Cambridge, Mass.-based Bluebird Bio, was recommended for approval by the Committee for Medicinal Products for Human Use (CHMP), the drug-reviewing arm of the European Medicines Agency. A final approval decision is expected within the next three months.
Lentiglobin is Bluebird’s first gene therapy, and if approved, will be marketed under the brand name Zynteglo.
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Please update me of the approval and how to get it started.