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Bluebird’s Gene Therapy Costs $3 Million per Treatment
Bluebird’s newly approved gene therapy, Skysona, costs $3 million per treatment, making it the most expensive therapeutic intervention worldwide.
On September 16, 2022, the FDA granted bluebird bio accelerated approval for their gene therapy technology, Skysona. This technology is meant to treat active cerebral adrenoleukodystrophy (CALD) in males between 4 and 17 years old. This new therapeutic intervention costs approximately $3 million per treatment, making it more expensive than Zynteglo and the most expensive therapy in the world.
The approval of Skysona was based on data collected from phase II and III clinical trials. Patients enrolled had an initial neurological function score of less than one. Throughout the course of the two-year trial, patients’ neurological function scores and major functional disabilities were monitored.
The study found that patients treated with Skysona had a 72% chance of survival without any major functional disabilities. That percentage is approximately 29% greater than the 43% chance of survival found in untreated patients.
Even though the company received approval, the FDA requires bluebird to continue long-term clinical trials and provide confirmatory data.
“As one of the largest and most experienced pediatric gene therapy and stem cell transplant programs in the world, the University of Minnesota is committed to expanding access and advancing care and research for patients with rare diseases like ALD,” said Paul Orchard, MD, pediatric blood and marrow transplant physician at the University of Minnesota Medical School and M Health Fairview Masonic Children’s Hospital, in the bluebird press release. “It’s crucial for these patients and families to have another therapeutic option for cerebral ALD beyond blood stem cell transplantation utilizing cells from another donor, and we’ve seen firsthand the impact that gene therapy has on our patients. We are encouraged by the progress we’re making to treat these rare and devastating diseases.”
The gene therapy will become available at the end of this year. However, the treatment will only be available in the United States at qualified centers such as Boston Children’s Hospital and Children’s Hospital of Philadelphia (CHOP). Despite the potential life-saving outcomes of this technology, the costs are astronomical. Each treatment will cost $3 million in the US. While bluebird is offering a patient support program, the final prices of each treatment are unclear.
Editor's Note: This article has been edited to reflect an error in the first paragraph which originally stated that Zynteglo was the most expensive therapy in the world.
