bluebird bio (BLUE) Granted FDA Priority Review of Biologics License Application for eli-cel Gene Therapy

bluebird bio, Inc. (Nasdaq: BLUE) today announced that the U.S. Food and Drug Administration (FDA) has accepted for priority review the Biologics License Application (BLA) for elivaldogene autotemcel (eli-cel, Lenti-D®), the company’s gene therapy for cerebral adrenoleukodystrophy (CALD) in patients less than 18 years of age. Eli-cel is an investigational one-time gene therapy, custom-designed to treat the underlying cause of this irreversible neurodegenerative disease and to stabilize neurologic function. The agency set a Prescription Drug User Fee Act (PDUFA) goal date of June 17, 2022.

“Eli-cel is an important potential therapeutic option for patients with CALD—a devastating neurodegenerative disease—and we are encouraged to be moving forward given the urgent unmet need for these children and their families,” said Andrew Obenshain, chief executive officer, bluebird bio. “As the second BLA acceptance for bluebird bio this year, this is a meaningful milestone in our work to deliver one-time treatments for severe genetic diseases.”

If approved, eli-cel will be the first approved treatment to address the underlying genetic cause of disease for patients living with CALD in the U.S. – offering appropriate patients an alternative to allogeneic hematopoietic stem cell transplant (allo-HSCT), which is associated with serious potential complications and mortality that increase in patients without a matched sibling donor. It is estimated that more than 70% of patients diagnosed with CALD do not have a matched sibling donor.

The BLA for eli-cel is supported by efficacy and safety data from the completed Phase 2/3 Starbeam study (ALD-102) (N=32). Additionally, the BLA contains data for 23 subjects dosed in the Phase 3 ALD-104 study. Study ALD-104 has subsequently completed enrollment and follow-up is ongoing. All patients who completed ALD-102, as well as those who will complete ALD-104, are invited to participate in a long-term follow-up study (LTF-304).

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 study ALD-102 at investigator discretion, and one additional subject experienced rapid disease progression early in the study, resulting in MFDs and subsequent death. All patients who completed ALD-102 enrolled in follow-up study LTF-304. The median duration of follow-up is 3.5 years (42.3 months; 13.4, 83.7).

Adverse reactions attributed to eli-cel observed in clinical trials include myelodysplastic syndrome, cystitis viral, pancytopenia, 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 55 patients who received eli-cel in clinical studies (ALD-102/LTF-304 and ALD-104).

On August 9, bluebird bio announced that the eli-cel clinical program was placed on a clinical hold, following a Suspected Unexpected Serious Adverse Reaction (SUSAR) of myelodysplastic syndrome (MDS). Available evidence suggests that the event was likely mediated by Lenti‑D lentiviral vector (LVV) insertion. Consistent with this known risk, two additional cases of MDS have subsequently been reported and details have been shared with the FDA and study investigators. The FDA clinical hold for eli-cel is ongoing and all patients who received eli-cel in the clinical program continue to be closely monitored, per study protocols. Given the devastating and fatal nature of CALD and lack of other treatment options for patients without a matched sibling donor, bluebird bio continues to assess the overall benefit/risk profile of the product as favorable for patients with CALD who do not have a matched sibling donor.

The FDA’s Priority Review designation is granted to therapies that have the potential to provide significant improvements in the treatment, diagnosis or prevention of serious conditions, and targets a review timeline of six months after the 60-day FDA BLA filing decision, compared to a standard review timeline of 10 months after the 60-day FDA filing decision.

The FDA previously granted eli-cel Orphan Drug status, Rare Pediatric Disease designation, and Breakthrough Therapy designation.

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