EMA Accepts Bluebird Bio's Marketing Authorization Application for Gene Therapy for Rare Blood Disorder

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  • LentiGlobin has Orphan Drug status in Europe and the US
  • It has also been granted an accelerated assessment in Europe and Breakthrough Therapy designation in the US
  • LentiGlobin is a one-time gene therapy and is also being studied in sickle cell disease


Bluebird bio, Inc. (Nasdaq: BLUE) announced on 10/5/18 that the European Medicines Agency (EMA) accepted the company’s marketing authorization application (MAA) for its investigational LentiGlobin gene therapy for the treatment of adolescents and adults with transfusion-dependent β-thalassemia (TDT) and a non-β0/β0 genotype.


LentiGlobin was previously granted an accelerated assessment by the Committee for Medicinal Products for Human Use (CHMP) of the EMA in July 2018, potentially reducing the EMA’s active review time of the MAA from 210 days to 150 days. The EMA has also granted Orphan Medicinal Product designation to LentiGlobin for the treatment of TDT. Additionally, the FDA has granted LentiGlobin Orphan Drug status and Breakthrough Therapy designation for the treatment of TDT.

“People living with transfusion-dependent β-thalassemia require frequent blood transfusions that are life-saving but may lead to complications, including organ failure due to iron overload,” said David Davidson, M.D., chief medical officer, bluebird bio. “The acceptance of our marketing authorization application for LentiGlobin is a milestone that advances us toward our goal of providing to patients the first one-time gene therapy that addresses the underlying genetic cause of TDT. We share this important milestone with the patients, families and healthcare providers who made it possible through their participation in our pioneering clinical studies of LentiGlobin.”

The MAA for LentiGlobin is supported by data from the completed Phase 1/2 Northstar (HGB-204) study and the ongoing Phase 1/2 HGB-205 study as well as available data from the Phase 3 Northstar-2 (HGB-207) study and the long-term follow-up study LTF-303.

TDT is an inherited blood disorder caused by a mutation in the β-globin gene, which causes ineffective red blood cell production leading to severe anemia. Supportive care for people with TDT consists of a lifelong regimen of chronic blood transfusions to enable survival and suppress symptoms of the disease, and iron chelation therapy to manage iron overload that results from the transfusions.

Despite the availability of supportive care, many people with TDT experience serious complications and organ damage due to underlying disease and iron overload. By eliminating or reducing the need for blood transfusions, the long-term complications associated with TDT may be reduced.

LentiGlobin is a one-time gene therapy being studied as a potential treatment to address the underlying genetic cause of TDT, which could eliminate or reduce the need for blood transfusions.

Bluebird bio’s clinical development program for LentiGlobin includes ongoing studies around the world with sites in Australia, Germany, Greece, France, Italy, Thailand, the United Kingdom and the United States.

Bluebird is also conducting a long-term safety and efficacy follow-up study (LTF-303) for people who have participated in bluebird bio-sponsored clinical studies of LentiGlobin for TDT and sickle cell disease.