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bluebird bio to restart marketing of Zynteglo in EU after previous suspension

EMA’s safety committee PRAC has confirmed favourable risk-benefit balance of gene therapy

bluebird bio will resume marketing of Zynteglo in the EU, after a review from the European Medicines Agency’s (EMA) safety committee PRAC confirmed the favourable benefit-risk profile of the beta-thalassemia gene therapy.

Earlier this year, bluebird voluntarily withdrew marketing of Zynteglo (betibeglogene autotemcel) in the EU after safety events relating to LentiGlobin for sickle cell disease (SCD) were reported.

Although no cases of haematologic malignancy have been reported in any Zynteglo-treated patients, it is manufactured using the same BB305 lentiviral vector use in LentiGlobin, prompting the voluntary marketing pause.

At the time of the pause, bluebird said that it had received reports of a suspected unexpected serious adverse reaction (SUSAR) of myelodysplastic syndrome (MDS) in a patient from group C of the phase 1/2 HGB-206 study of LentiGlobin for SCD.

At the same time, bluebird announced that another SUSAR of acute myeloid leukaemia (AML) had been reported in the HGB-206 study – as a result, the FDA placed clinical holds on the gene therapy.

Since then, the FDA has lifted all clinical holds on studies evaluating LentiGlobin for SCD, after the treating investigator concluded the patient in the HGB-206 study did not have MDS and revised the diagnosis instead to transfusion-dependent anaemia.

In March bluebird reported that it is ‘very unlikely’ that the SUSAR of AML was related to the BB305 lentiviral vector used for LentiGlobin gene therapy.

The PRAC also investigated and assessed these safety events as part of its review into Zynteglo.

“Patient safety remains our top priority. To this end, we are grateful to the PRAC for its comprehensive review of the available evidence and positive recommendation for Zynteglo,” said Andrew Obenshain, president, severe genetic diseases, bluebird bio.

“We are pleased to resume offering Zynteglo to patients living with transfusion-dependent beta-thalassemia, offering the potential to live free from transfusions which is evidenced by our clinical studies where patients are maintaining normal or near-normal haemoglobin levels over the course of up to seven years of follow-up,” he added.

Article by
Lucy Parsons

12th July 2021

From: Marketing



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