Only a month after their arrest Zynteglo approved for the treatment of beta thalassemiaBluebird SKYSONA (elivaldogene autotemcel), also known as eli-cel, has received rapid approval from the Food and Drug Administration. The company says the drug slows the progression of neurological dysfunction in boys ages 4 to 17 who have early active cerebral adrenal dystrophy (CALD). Bluebird says a previous clinical suspension on the eli-cel clinical development program has been lifted.
The company expects its product to be available by the end of 2022 through a limited number of qualified treatment centers (QTCs) in the United States, including Children’s Hospital Boston and Children’s Hospital of Philadelphia. The treatment is expected to cost $3 million.
These approvals are necessary for the start-up of rare diseases, which laid off 30% of its workforce earlier this year.
Cerebral ALD is a rare, progressive, neurodegenerative disease that primarily affects young boys and causes devastating and irreversible neurological deterioration, including major functional impairments such as communication loss, cortical blindness, tube feeding requirements, complete enuresis, wheelchair dependence, or complete loss of voluntary movement. About half of patients who do not receive treatment die within five years of onset of symptoms.
The disorder is caused by mutations in ABCD1 A gene that influences the production of the adrenocorticotropic protein (ALDP) and consequently leads to the accumulation of long-chain fatty acids (VLCFAs), primarily in the white matter of the brain and spinal cord.
Prior to SKYSONA’s approval, the only treatment options were allogeneic hematopoietic stem cell transplantation (allo-HSCT), which is associated with a risk of potentially serious complications including death, which can be significantly increased in patients who do not have their HWC-matched antigen. (HLA). .
“Children with ALD and their families have been at the heart of Bluebird’s mission since the company was founded more than a decade ago,” said Andrew Openshine, CEO of Bluebird Bio.
“For the ALD community, this long-awaited approval represents great hope and offers families a new option that, for many, has not been there. We are grateful to everyone who was involved in developing SKYSONA and are committed to working with providers and pushers to make this important treatment option available to patients and their families.”
“The agony of watching your child slip away is something that no parent should put up with,” said Elisa Seeger, co-founder of ALD Alliance. “We have made great strides in providing children diagnosed with CALD with the best chance in life with early recognition of ALD through expanded newborn screening. But with limited treatment options, early diagnosis remains a cause of despair rather than hope for many families today. Parents who receive a CALD diagnosis for their children can renew hope for the future.”
While gene therapy slows the progression of devastating neurological dysfunction, its label also has a black box to warn of hematological malignancies.
“As one of the largest and most experienced pediatric gene therapy and stem cell transplantation programs in the world, the University of Minnesota is committed to expanding access to care, research, and development for patients with rare diseases such as ALD,” said Paul Orchard, MD, PhD. Pediatric blood and marrow transplant physician at the University of Minnesota College of Medicine and M Health Fairview Masonic Children’s Hospital.
He added, “It is imperative that these patients and families have another treatment option for cerebral ALD other than a blood stem cell transplant using cells from another donor, and we have seen first-hand the impact of gene therapy on our patients. We are encouraged by the progress we are making in treating these rare and devastating diseases.” .