Bluebird bio has announced that their SKYSONA therapy has been approved for pediatric use by the FDA.
The US Food and Drug Administration (FDA) has approved SKYSONA (elivaldogene autotemcel; Bluebird Bio) for use in boys aged 4 to 17 years with early, active cerebral adrenoleukodystrophy (CALD).
SKYSONA can be used to slow the neurological decline in boys caused by CALD. This neurological decline is irreversible and leads to major functional disabilities such as loss of communication, requirement for tube feeding, cortical blindness, wheelchair dependence, total incontinence, or complete loss of voluntary movement.
Without treatment, about half of patients with CALD die within 5 years. Allogeneic hematopoietic stem cell transplant was the most effective treatment in the past, which greatly increased risk of severe complications and death. These risks were magnified in patients without a human leukocyte antigen matched donor.
“The agony of watching your child slip away is something no parent should have to bear,” said Elisa Seeger, co-founder of ALD Alliance. “We have made significant strides in providing children diagnosed with CALD the best chance at life with early identification of ALD through expanded newborn screening. Yet with limited treatment options, early diagnosis is still cause for despair instead of hope for many families. Today, parents whose boys receive a CALD diagnosis can have renewed hope for the future.”
Bluebird will also be providing long-term clinical data on SKYSONA to the FDA, including data on an ongoing long-term follow-up study, LTF-304 (NCT02698579). This will allow data on patients followed for 15 years after clinical trials to be submitted.
SKYSONA is set to be released by the end of 2022 throughout Qualified Treatment Centers in the United States. The wholesale acquisition cost of SKYSONA has been set to $3 million in the United States. For additional information, patients can follow the bluebird patient support program, My Bluebird Support.
bluebird bio receives FDA accelerated approval for SKYSONA® gene therapy for early, active cerebral adrenoleukodystrophy (CALD). bluebirdbio. Accessed September 21, 2022. https://investor.bluebirdbio.com/news-releases/news-release-details/bluebird-bio-receives-fda-accelerated-approval-skysonar-gene