Eli-Cel Gene Therapy Approved for Early, Active Cerebral Adrenoleukodystrophy

The US Food and Drug Administration (FDA) granted accelerated approval to elivaldogene autotemcel, also known as eli-cel (Skysona), to slow the progression of neurologic dysfunction in boys 4 through 17 years of age with early, active cerebral adrenoleukodystrophy (CALD), maker bluebird bio Inc. recently announced.

“After supporting the clinical development of Skysona for nearly a decade as a study site, Boston Children’s Hospital is extremely pleased that an FDA-approved therapy is now available for children who urgently need new therapies,” said David A. Williams, MD, chief of the division of hematology/oncology at Boston Children’s Hospital.

A rare, progressive neurodegenerative disease that primarily affects young boys, CALD leads to irreversible neurologic decline, and nearly half of patients die within 5 years of symptom onset without treatment. Prior to the approval of eli-cel gene therapy, the only effective treatment option was allogeneic hematopoietic stem cell transplant, which is associated with the risk of serious complications including death.

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The approval of the gene therapy is based on data from bluebird bio’s phase 2/3 ALD-102 study and phase 3 ALD-104 study. The open-label, single-arm studies enrolled a total 67 patients with early, active CALD. Efficacy of eli-cel was compared to a natural history population.

A post-hoc enrichment analysis in symptomatic patients that assessed survival free of major functional disabilities (MFD-free survival) found that 11 patients treated with eli-cel had an estimated 72% likelihood of MFD-free survival at 24 months from time of their first neurologic function score of 1 or more, compared with a 43% likelihood in seven untreated patients.

The most common nonlaboratory adverse reactions (20% or higher incidence) with eli-cel are mucositis, nausea, vomiting, febrile neutropenia, alopecia, decreased appetite, abdominal pain, constipation, pyrexia, diarrhea, headache, and rash. The most common grade 3 or 4 laboratory abnormalities (40% or higher incidence) include leukopenia, lymphopenia, thrombocytopenia, neutropenia, anemia, and hypokalemia.

bluebird has agreed to provide confirmatory long-term clinical data to the FDA as a condition of the accelerated approval. The company expects to include data from an ongoing study that follows patients treated in clinical trials for 15 years, and data from commercially treated patients.

Eli-cel should be available by the end of 2022 through a limited number of qualified treatment centers in the United States, including Boston Children’s Hospital and Children’s Hospital of Philadelphia, according to bluebird bio. The company has set the wholesale acquisition cost of the therapy at $3 million.

Reference

bluebird bio receives FDA accelerated approval for SKYSONA gene therapy for early, active cerebral adrenoleukodystrophy (CALD). News release. bluebird bio Inc.; September 16, 2022. Accessed October 11, 2022.