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EU approves bluebird bio’s CALD gene therapy Skysona

CALD is a progressive and fatal neurodegenerative disease that overwhelmingly affects males

The European Commission (EC) has approved bluebird bio’s gene therapy Skysona for the rare inherited neurological disease cerebral adrenoleukodystrophy (CALD).

The EC has cleared Skysona (elivaldogene autotemcel) for the treatment of early CALD in patients under the age of 18 years old with an ABCD1 gene mutation who do not have a matched sibling blood stem cell donor.

The approval is supported by data from the phase 2/3 Starbeam study as well as the ongoing phase 3 ALD-104 study.

In the phase 2/3 Starbeam study evaluating Skysona, 90% of CALD patients met the month 24 major functional disability- (MFD) free survival endpoint as of the last data cutoff date.

MFDs are the six severe disabilities commonly attributed to CALD, which have the most severe effect on a patient’s ability to function independently.

In addition, 26 out of 28 evaluable patients maintained a neurologic function score (NFS) less than or equal to one until month 24, with 24 of those patients having no change in their NFS.

All the patients who completed the Starbeam study enrolled for long-term follow-up in the LTF-304 study. The majority of patients that enrolled in LTF-304 – 96.3% - remained alive and maintained their MFD-free status through their last follow-up on study.

The median duration of follow-up was 3.2 years and 14 patients reached at least their year five follow-up visit.

“bluebird bio was founded with the mission of developing a therapy to recode CALD on the genetic level, and today’s announcement represents over twenty years of research and development that has laid the groundwork for future gene therapies to be possible,” said Andrew Obenshain, president of severe genetic diseases at bluebird bio.

CALD is a progressive and fatal neurodegenerative disease that overwhelmingly affects males. It involves the breakdown of myelin – the protective sheath of nerve cells in the brain that is responsible for muscle control and thinking.

The condition is caused by mutations in the ABCD1 gene that affect the production of ALDP which eventually causes damage to the adrenal cortex and white matter of the brain and spinal cord.

Skysona is designed to add functional copies of the ABCD1 gene into a patient’s hematopoietic stem cells (HSC).

Once this functional gene is added to a CALD patient’s stem cells, the patient's body can produce the adrenoleukodystrophy protein (ALDP), which is believed to allow for the breakdown of very-long-chain fatty acids that build up to toxic levels in the brain.

Article by
Lucy Parsons

21st July 2021

From: Regulatory



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