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

[December 20, 2022]  (Reuters) - Bluebird Bio Inc 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.

Signage is seen outside of the Food and Drug Administration (FDA) headquarters in White Oak, Maryland, U.S., August 29, 2020. REUTERS/Andrew Kelly

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)

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