EC Grants Marketing Authorization for bluebird bio’s Gene Therapy SKYSONA

The European Commission (EC) has granted marketing authorization to bluebird bio for its gene therapy SKYSONA, which is a one-time therapy for the treatment of early cerebral adrenoleukodystrophy (CALD) in patients under 18 years old without matched sibling donor.

CALD is a rare, progressive, and irreversible neurodegenerative disease that involves the breakdown of myelin. SKYSONA is custom designed to treat the underlying cause of CALD with the goal of halting disease progression and preserving as much neurological function as possible. The gene therapy works via ex-vivo transduction with the Lenti-D lentiviral vector to add the functional copies of the ABCD1 gene into the patient’s own hematopoietic stem cells.

The marketing authorization of SKYSONA was supported by clinical data that demonstrated the treatment had a durable effect on major functional disabilities-free survival. The therapy has been evaluated as part of the European Medicines Agency’s Priority Medicines scheme (PRIME) and had previously been granted orphan medicinal product status.

“SKYSONA is the first and only one-time gene therapy approved in the EU [European Union] for patients with CALD, a devastating neurodegenerative disease, and we are immensely grateful to all who have brought us to this milestone, from the patients and their loved ones, to all study investigators, regulators, the ALD community and, of course, the extended bluebird family,” said Andrew Obenshain, president, severe genetic diseases, bluebird bio, in a July 21, 2021 press release. “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.”

Source: bluebird bio