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FDA halts studies for bluebird bio's sickle cell therapy LentiGlobin

Published on 24/02/21 at 10:49am

The FDA has put bluebird bio’s LentiGlobin gene therapy programme for sickle cell disease (SCD) on clinical hold, following Suspected Unexpected Serious Adverse Reactions (SUSARs) in clinical trials.

The agency’s decision comes a week after trials for the drug were temporarily halted due to a SUSAR of acute myeloid leukaemia (AML) in a patient treated with LentiGlobin five years ago. Another SUSAR of myelodysplastic syndrome possibly linked to the drug is also being investigated by researchers.

The company announced the suspension, which affects both the Phase I/II HGB-206 and Phase III HGB-210 trials, in a quarterly update published yesterday.

It said: “Due to a SUSAR of acute myeloid leukemia and a SUSAR of myelodysplastic syndrome in our HGB-206 clinical study, the FDA has placed our clinical studies of LentiGlobin for SCD on clinical hold.

“We are investigating these events and plan to continue to work closely with the FDA in their review of these events. In addition, we are also engaged with the EMA in discussions regarding our proposed development plans for LentiGlobin for SCD in Europe.”

Bluebird bio has also temporarily suspended sales of its approved gene therapy Zynteglo, a treatment for the blood disorder beta thalassemia. While no cases of haematologic malignancy have been reported in patients who received the medication, it is produced using the same lentiviral vector as LentiGlobin.

The company stated: “The EMA has paused the renewal procedure for Zynteglo's conditional marketing authorisation while the EMA's pharmacovigilance risk assessment committee reviews the risk-benefit assessment for Zyntelgo and determines whether any additional pharmacovigilance measures are necessary.”

Darcy Jimenez

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