FDA

U.S. FDA lifts hold on Bluebird’s sickle cell disease therapy

Dec 19 (Reuters) – Bluebird Bio Inc (BLUE.O) said on Monday that the U.S. health regulator had lifted a partial clinical hold on studies for its gene therapy to treat blood disorder sickle cell disease.

The U.S. Food and Drug Administration’s decision ends a year-long halt on future studies and a pause in enrollment for ongoing studies of lovo-cel after one case of persistent anemia in a patient.

The move also signals a more favorable regulatory environment for cell and gene therapies after a spate of clinical holds by the regulator in the last few years, said SVB Securities analyst Mani Foroohar.

Bluebird said its investigation showed that patients with persistent anemia had a genetic trait called the alpha-thalassemia trait. Such patients would be excluded from future studies of the drug.

Sickle cell disease leads to a shortage of healthy blood cells due to the sickle-shape instead of the round shape of red blood cells, and can cause complications such as infection, acute chest syndrome and stroke.

Bluebird is developing the drug as a potential one-time treatment for the disorder, which affects an estimated 100,000 people in the United States.

The therapy works by adding a gene to a patient’s blood stem cells that can help the body produce anti-sickling hemoglobin.

The company, which flagged going concern doubts earlier this year, is working to resume enrollment and treatment of patients aged between 2 and 17.

Reporting by Aditya Samal in Bengaluru; Editing by Krishna Chandra Eluri and Shailesh Kuber

Our Standards: The Thomson Reuters Trust Principles.

Source: Reuters