FDA committee unanimously endorses gene therapy for cerebral adrenoleukodystrophy
Click Here to Manage Email Alerts
Biotechnology company bluebird bio announced that an FDA advisory committee has unanimously endorsed its gene therapy treatment for early active cerebral adrenoleukodystrophy.
According to a company press release, the FDA’s Cellular, Tissue and Gene Therapies Advisory Committee voted 15 to 0 that the benefits of bluebird bio’s elivaldogene autotemcel (eli-cel) therapy outweighed the risks for patients younger than 18 years without an available and willing human leukocyte antigen-matched sibling hematopoietic stem cell donor.
Early active cerebral adrenoleukodystrophy (CALD) is a rare, progressive disease that primarily affects young boys and causes behavioral, cognitive and neurological deficits. Without treatment, nearly 50% of patients die within 5 years of symptom onset, the company stated. Allogenic hematopoietic stem cell transplant is currently the only effective treatment for CALD but is linked to serious complications and mortality in patients without a matched sibling donor.
“For decades, the CALD community has fought for the opportunity to stave off the rapid, irreversible decline associated with this devastating disease,” Andrew Obenshain, bluebird bio CEO, said in the release. “Today we are one step closer to delivering a potentially lifesaving therapy for CALD.”
The committee’s recommendation is based on the biologics license application currently under FDA priority review with a Prescription Drug User Fee Act goal date of Sept. 16, 2022, which is supported by data from the completed phase 2/3 Starbeam study (ALD-102, n = 32), as well as from data for 35 patients in the phase 3 ALD-104 study.
According to bluebird bio, patients treated with eli-cel in clinical studies were more likely to achieve both overall and event-free survival compared with allogenic hematopoietic stem cell transplant patients without a matched sibling donor.
The eli-cel clinical program was placed on a clinical hold following a suspected unexpected serious adverse reaction of myelodysplastic syndrome in August 2021. If approved, eli-cel will be the first approved gene therapy to target the underlying genetic cause of CALD, the company stated.