The Food and Drug Administration (FDA) has accepted for Priority Review the Biologics License Application for elivaldogene autotemcel (eli-cel) for the treatment of cerebral adrenoleukodystrophy in patients less than 18 years of age without a matched sibling donor.

Cerebral adrenoleukodystrophy (CALD) is a rare, X-linked metabolic disorder caused by mutations in the ABCD1 gene, which affects the production of adrenoleukodystrophy protein (ALDP). Eli-cel is an investigational one-time gene therapy designed to add functional copies of the ABCD1 gene into a patient’s own hematopoietic stem cells, resulting in the production of ALDP.

The application is supported by data from the phase 2/3 Starbeam study (ClinicalTrials.gov Identifier: NCT01896102), which evaluated the efficacy and safety of eli-cel in 32 patients with active CALD. Findings showed that 90.6% (n=29/32) of patients achieved major functional disabilities (MDR)-free survival at 24 months.

The application also includes data for 23 patients being evaluated in the phase 3 ALD-104 study (ClinicalTrials.gov Identifier: NCT03852498), which has now completed enrollment. Patients who completed Starbeam, along with those who complete ALD-104, are eligible to enroll in a long-term follow-up study (ClinicalTrials.gov Identifier: NCT02698579).


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In clinical trials, myelodysplastic syndrome, viral cystitis, pancytopenia, and vomiting were observed in patients treated with eli-cel.

A Prescription Drug User Fee Act (PDUFA) target date of June 17, 2022 has been set for the application.

Reference

bluebird bio announces FDA Priority Review of Biologics License Application for eli-cel gene therapy for cerebral adrenoleukodystrophy (CALD) in patients without a matched sibling donor. News release. bluebird bio, Inc. December 17, 2021. Accessed December 20, 2021. https://www.businesswire.com/news/home/20211217005659/en/bluebird-bio-Announces-FDA-Priority-Review-of-Biologics-License-Application-for-eli-cel-Gene-Therapy-for-Cerebral-Adrenoleukodystrophy-CALD-in-Patients-Without-a-Matched-Sibling-Donor. 

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