Bluebird bio’s blood disorder therapy is effective – FDA staff

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

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June 7 (Reuters) – Bluebird bio’s (BLUE.O) blood disorder treatment demonstrates “clinically significant” benefits in patients, U.S. Food and Drug Administration staff reviewers have said in court documents. information published on Tuesday.

The company has applied for approval of beti-cel as a single gene therapy for the treatment of blood transfusion-dependent beta-thalassemia patients.

The data provided in the application supports the efficacy of beti-cel for the treatment of these patients, FDA staff saidpushing the company’s shares up 4.1% to $3.1.

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The reviewers also published documents for bluebird’s other gene therapy, eli-cel, for the treatment of a rare neurological condition called cerebral adrenoleukodystrophy (CALD) in patients under the age of 18, which raises safety concerns.

It is unclear whether the benefit of eli-cel outweighs the significant and unknown long-term risk of myelodysplastic syndrome (MDS), the reviewers wrote.

The agency put the eli-cel program on clinical hold last year after three patients in the trial developed MDS, a type of cancer. Staff said on Tuesday there was a causal link between the treatment and two of the cases and expect longer follow-up to uncover additional cases.

A panel of outside FDA experts is scheduled to discuss the approval of eli-cel and beti-cel later this week.

This will mark a watershed moment for the field of gene therapy, as it is the first time the Cell, Tissue and Gene Therapies Advisory Committee has met since 2017. The committee then recommended the approval of Luxturna from Spark Therapeutics , the first gene therapy to be approved in the United States.

The outcome of the meeting is also seen as critical for bluebird after the company flagged “going concern” doubts in March and also unveiled plans in April to cut its workforce by 30%.

“The company is currently in a very precarious financial position, given the cash trail … (the decision) is not only important for the gene therapy space, but also crucial for the company,” said to Reuters William Blair analyst Raju Prasad.

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Reporting by Mrinalika Roy in Bengaluru; Editing by Sriraj Kalluvila

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