FDA Fast Tracks Approval of Novel Therapy for Rare Neurologic Disorder
The US Food and Drug Administration (FDA) has granted accelerated approval to the new gene therapy elivaldogene autotemcel (Skysona, bluebird bio) for the treatment of cerebral adrenoleukodystrophy (CALD).
The therapy “is indicated to delay the progression of neurological dysfunction in children 4 to 17 years of age” with early active CALD, the manufacturer noted in a news release.
CALD is a rare, progressive neurodegenerative disease that primarily affects young children and causes irreversible neurological impairment, including major functional disabilities such as loss of communication, cortical blindness, need for tube feeding, total incontinence, wheelchair dependence, or complete loss of voluntary movement.
The approval follows a unanimous approval for the therapy by an FDA advisory committee in June, as reported by Medscape Medical News.
Until now, effective options for CALD have been limited to allogeneic hematopoietic stem cell transplantation, which carries the risk of serious complications, including treatment-related death, graft failure, and graft-versus-host disease.
“We have made significant progress in giving children diagnosed with CALD the best chance at life with early identification of ALD through expanded newborn screening. However, with limited treatment options, early diagnosis remains a reason of despair rather than hope for many families,” Elisa Seeger, co-founder of the ALD Alliance, said in the statement.
With this approval, “parents whose children receive a CALD diagnosis can have renewed hope for the future,” Seeger added.
Urgently needed
The neurological disorder is caused by mutations in the ABCD1 gene. Elivaldogene autotemcel works by taking advantage of ex-vivo transduction with a lentiviral vector to insert functional copies of the ABCD1 genes in a patient’s hematopoietic stem cells.
Results from a phase 2/3 study showed that 2 years after receiving a single gene therapy treatment, there was no deterioration in neurological function in 27 of 30 children (90%) with early active CALD, according to data reported on last year at the Annual Meeting of the European Society for Blood and Bone marrow transplant.
The most common non-laboratory related adverse reactions include mucositis, nausea, vomiting, fever neutropeniaalopecia, decreased appetite, abdominal pain, constipationpyrexia, Diarrhea, headacheand rash.
The most common grade 3 or 4 laboratory abnormalities include leukopenia, lymphopenia, thrombocytopenia, neutropenia, anemiaand hypokalemia.
Elivaldogene autotemcel had breakthrough therapy and orphan drug status and received priority review.
As a condition of accelerated approval, the company will provide confirmatory long-term clinical data to the FDA.
The company expects elivaldogene autotemcel to be available later this year through a limited number of qualified treatment centers in the United States, including Boston Children’s Hospital and Children’s Hospital of Philadelphia, which participated in clinical trials.
“After supporting the clinical development of Skysona for nearly a decade as a study site, Boston Children’s Hospital is very pleased that an FDA-approved therapy is now available for children in urgent need of new therapies,” David A. Williams , MD, chief, division of hematology/oncology at Boston Children’s Hospital, in the release.
September 20, 2022
