bluebird bio, Inc. (NASDAQ: BLUE) (“bluebird bio” or the “Company”) today announced that the U.S. Food and Drug Administration (FDA) posted briefing documents for the FDA Cellular, Tissue and Gene Therapies Advisory Committee Meeting to review elivaldogene autotemcel (eli-cel) for early active cerebral adrenoleukodystrophy (CALD) in patients without a matched sibling donor and betibeglogene autotemcel (beti-cel) for the treatment of people with β-thalassemia who require regular red blood cell transfusions.

The advisory committee meeting will take place June 9-10, 2022. Briefing materials, agendas and webcast information for the meeting can be accessed here. The Company is not responsible for the content of, nor the statements made in, the briefing materials that were prepared by the FDA.

The Prescription Drug User Fee Act (PDUFA) goal dates for a decision on approval of beti-cel for people with β-thalassemia who require regular red blood cell transfusions and eli-cel for patients with early active CALD without a matched sibling donor are August 19, 2022, and September 16, 2022, respectively.

About elivaldogene autotemcel (eli-cel)

eli-cel (pronounced ELL-ee-cell) uses ex-vivo transduction with the Lenti-D lentiviral vector (LVV) to add functional copies of the ABCD1 gene into a patient’s own hematopoietic stem cells (HSCs). The addition of the functional ABCD1 gene allows patients to produce the ALD protein (ALDP), which is thought to facilitate the breakdown of very long-chain fatty acids (VLCFAs). The expression of ALDP and effect of eli‑cel is expected to be life-long. The goal of treatment with eli-cel is to stop the progression of CALD and, consequently, preserve as much neurological function as possible, including the preservation of motor function and communication ability. Importantly, with eli-cel, there is no need for donor HSCs from another person.

bluebird bio’s clinical development program for eli-cel includes the completed pivotal Phase 2/3 Starbeam study (ALD-102) and the ongoing Phase 3 ALD-104 study, which has completed enrollment and treatment of all patients. Additionally, bluebird bio is conducting a long-term safety and efficacy follow-up study (LTF-304) for patients who have received eli-cel for CALD and completed two years of follow-up in ALD-102 or ALD-104. Clinical studies of eli-cel were placed on hold by the FDA and follow-up of all patients continues, per protocol.

In ALD-102, 90.6% (29/32) of patients met the primary endpoint of Major Functional Disabilities (MFD)-free survival at 24 months. As previously reported, two patients withdrew from ALD-102 at investigator discretion, and one additional subject experienced rapid disease progression early after treatment, resulting in MFDs and subsequent death. All patients who completed ALD-102 enrolled in a long-term follow-up study (LTF-304). The median duration of follow-up is approximately four years (49 months; 13.4, 88.1 months).

Adverse reactions attributed to eli-cel observed in clinical trials include myelodysplastic syndrome, viral cystitis, pancytopenia, and nausea and vomiting. There have been no reports of graft-versus-host-disease, graft failure or rejection, transplant-related mortality, or replication-competent lentivirus in the 67 patients who received eli-cel and are being followed in clinical studies (ALD-102, ALD-104, LTF-304).