Biotechnology company, bluebird bio, Inc. BLUE announced late Friday that the Food and Drug Administration approved its gene therapy for a rare neurodegenerative disease.
What Happened: The FDA granted accelerated approval for Skysona, aka eli-cel, to slow the progression of neurologic dysfunction in boys 4-17 years of age with early, active cerebral adrenoleukodystrophy (CALD), the Somerville, Massachusetts-based company said in a statement.
The approval was expected, given an Adcom that met in June to review the therapy unanimously recommended approval.
CALD is a rare, progressive neurodegenerative disease caused by mutations in a gene called ABCD1 that leads to the build-up of very long-chain fatty acids in the brain and the spinal cord. It affects young boys and causes irreversible, devastating neurologic decline. This often results in loss of communication, cortical blindness, requirement of tube feeding, total incontinence, wheelchair dependence or complete loss of voluntary movement.
Why It’s Important: For bluebird, it is back-to-back approvals, having received the nod for Zynteglo, a one-time gene therapy to treat an inherited bleeding disorder called beta thalassemia in adult and pediatric patients, in August.
About half of the patients who do not receive treatment for CALD die within five years of symptom onset, the company said. Before the Skysona nod, the only option available for patients was an allogenic hematopoietic stem cell transplant, which carries the risk of serious complications, including death.
The European Medicines Agency has already approved Skysona for the same indication.
Wells Fargo Securities said in a note last week that eli-cel’s approval decision could be positive, potentially providing 10-15% upside to the stock based on the value of the pediatric rare disease priority review voucher that is to be granted upon eli-cel's approval.
What’s Next: Bluebird expects to commercially launch Skysona by the end of 2022 through a limited number of “Qualified Treatment Centers” in the U.S. The company has fixed the wholesale acquisition cost of the therapy at $3 million, making it the most expensive drug to date.
Given the accelerated approval granted, the company has agreed to provide confirmatory long-term clinical data from the ongoing long-term follow-up study, which follows patients treated in clinical trials for 15 years.
The company also confirmed that a previous clinical hold imposed on the eli-cel clinical development program has been lifted.
Bluebird shares closed Friday's session at $6.34, down 10.58%, according to Benzinga Pro data. In after-hours, the stock was up about a percent.
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