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bluebird bio announces positive recommendation by EU PRAC committee regarding safety referral review of Zynteglo for transfusion-dependent beta-thalassemia and marketing to resume in EU.bluebird bio announces positive recommendation by EU PRAC committee

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Published:10th Jul 2021
bluebird bio, Inc announced that the European Medicines Agency's (EMA) Pharmacovigilance Risk Assessment Committee (PRAC) has concluded based on the review of all available data that the benefit-risk balance of medicinal products containing Zynteglo (betibeglogene autotemcel gene therapy) remains favorable. As of today, bluebird bio has informed the EMA that the company is lifting the voluntary marketing suspension.

“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 ?-thalassemia, offering the potential to live free from transfusions which is evidenced by our clinical studies where patients are maintaining normal or near-normal hemoglobin levels over the course of up to seven years of follow-up.”

No cases of hematologic malignancy have been reported in any patient who has received treatment with Zynteglo. However because it is manufactured using the same BB305 lentiviral vector used in LentiGlobin for sickle cell disease (SCD; investigational drug product bb1111), bluebird bio decided to temporarily suspend marketing of Zynteglo while the root cause of the safety events reported earlier this year for LentiGlobin for SCD were investigated by the company and assessed by the PRAC.

As previously announced on June 7, 2021, the FDA lifted the clinical holds on the Phase 1/II HGB-206 and Phase III HGB-210 studies of LentiGlobin for SCD following the Agency’s review of the data.

Condition: Beta-thalassemia
Type: drug

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