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bluebird bio anticipates lift of clinical holds on LentiGlobin in mid-2021

Previously reported case of MDS in phase 1/2 study has been re-classified as transfusion-dependent anaemia

bluebird bio is anticipating a potential lift of all clinical holds on its LentiGlobin gene therapy for sickle cell disease (SCD) following the re-classification of one of the previously reported suspected unexpected serious adverse reactions (SUSAR).

In February, bluebird bio said that it had received reports of a case of myelodysplastic syndrome (MDS) in a patient from group C of the phase 1/2 HGB-206 study of LentiGlobin (betibeglogene autotemcel; beti-cel) for SCD.

At the same time, bluebird bio said that another SUSAR of acute myeloid leukaemia (AML) had been reported in the HGB-206 study.

Today, bluebird bio announced that after further assessment following the review of results from additional tests of the MDS case, the treating investigator has concluded the patient did not have MDS and revised the diagnosis instead to transfusion-dependent anaemia.

The biotech company said it is continuing to work with the treating investigator to identify the cause of the anaemia.

bluebird bio also reported last month that it is ‘very unlikely’ that the SUSAR of AML was related to the BB305 lentiviral vector (LVV) used for LentiGlobin gene therapy.

With this assessment, as well as the revision of the MDS case, bluebird bio is hoping to potentially see a lift of all clinical holds on the HGB-206 and HGB-210 clinical studies of LentiGlobin for SCD, as well as the holds on the HGB-207 and HGB-212 studies of beti-cel for beta-thalassaemia.

In relation to  its beta-thalassaemia gene therapy Zynteglo, bluebird bio said this would be withdrawn from the German market after unsuccessful negotiations with health authorities in that country.

“The price proposed by the German health authorities fails to recognise the severe burden of living with TDT or the innovation and benefit Zynteglo brings to patients who are impacted every day, throughout their lives, by this severe genetic disease,” the biotech company said.

bluebird bio went on to say that further negotiations concerning the gene therapy are continuing in countries in Europe, and that it expected to provide updates on the negotiation processes in the second half of 2021.

In response to these and other events over the past year, bluebird bio has also announced that it plans to reduce its workforce, with the majority of the reductions occurring in Europe.

The company said that this will enable resources to be reallocated, to allow bluebird bio to focus on ‘priority’ European markets and streamline global operations.

Article by
Lucy Parsons

20th April 2021

From: Regulatory



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