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 ( 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.

Continue Reading

The application also includes data for 23 patients being evaluated in the phase 3 ALD-104 study ( 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 ( Identifier: NCT02698579).

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.


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.

This article originally appeared on MPR