bluebird bio Receives Second FDA Approval for a Gene Therapy in Two Months with Skysona for CALD

Anna Osborne
20 September 2022
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bluebird bio’s Skysona has become the first therapy targeting early active cerebral adrenoleukodystrophy (CALD) to be approved, under an accelerated timeline, by the FDA.

The neurodegenerative disease, CALD, causes progressive neurological dysfunction in boys causing major functional disabilities such as loss of communication, cortical blindness, wheelchair dependence or total loss of voluntary movement.

Skysona has been granted accelerated approval by the FDA to slow the progression of neurological symptoms in boys aged 4-17 years old – the first therapy shown to slow this debilitating disease’s progress.

This approval is based on data from bluebird’s Phase II/III and Phase III open-label single-arm studies. Patients in the early stages on CALD were treated with Skysona and assessed for the onset of six Major Functional Disabilities (MFDs).

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These studies showed that the patients who received Skysona had an estimated 72% likelihood of MFD-free survival at 24 months compared to a 43% likelihood in untreated patients.

Concurrent with this news, bluebir

d bio also reported that the clinical hold on its Skysona program has also been lifted.Before Skysona’s approval treatment options for patients with CALD were limited to allogeneic hematopoietic stem cell transplants, which carry the risk of serious complications including death. Nearly half of the patients who don’t receive any treatment die within five years of symptom onset – a bleak outlook.

“For the ALD community, this long-awaited approval represents significant hope and offers families a new option where, for many, there had been none. We are grateful to every individual who was involved in the development of SKYSONA and are committed to working with providers and payers to make this important treatment option available to patients and their families,” said Andrew Obenshain, chief executive officer, bluebird bio.

Prior to the FDA’s Skysona Accelerated Approval, bluebird received Orphan Drug designation, Rare Pediatric Disease designation, and Breakthrough Therapy designation, from the administrator. Alongside the approval proposal, Skysona has also been granted a rare pediatric priority review voucher.

As a condition of this approval bluebird will provide the FDA with long-term clinical data, including data from the ongoing long-term follow-up study (LTF-304) following patients currently 15 years post-admission as well as those to receive commercial Skysona.  

The wholesale acquisition cost of Skysona has been set by bluebird at $3m in the U.S., where they plan to make Skysona commercially available by the end of 2022 at select Qualified Treatment Centres.

“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 progress we’re making to treat these rare and devastating diseases,” concluded Paul Orchard, Pediatric Blood and Marrow Transplant Physician at the University of Minnesota Medical School and M Health Fairview Masonic Children’s Hospital.

Source: bluebird bio Press Release


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