Data shows bluebird bio’s blood disorder therapy effective – FDA staff

Signage is seen outside of 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 meaningful” benefit in patients, staff reviewers at the U.S. Food and Drug Administration said in briefing documents published on Tuesday.

The company has applied for approval of beti-cel as a one-time gene therapy for the treatment of Beta-thalassemia patients dependent on blood transfusions.

Data provided in the application supports the effectiveness of beti-cel for treatment of these patients, the FDA staff said.

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The reviewers also published documents for bluebird’s other gene therapy, eli-cel, for treatment of a rare neurologic disease called cerebral adrenoleukodystrophy (CALD) in patients below 18 years.

Agency reviewers said it was unclear whether eli-cel’s efficacy is non-inferior to stem cell transplants, which are currently the only treatment available for CALD patients.

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

The meeting marks a defining moment for the gene therapy field, as it is the first time the Cellular, Tissue, and Gene Therapies Advisory Committee has met since 2017. The panel had then recommended approval for Spark Therapeutics’ Luxturna, the first gene therapy to be approved in the United States.

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Reporting by Mrinalika Roy in Bengaluru; Editing by Sriraj Kalluvila

Our Standards: The Thomson Reuters Trust Principles.

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