EMA recommends approval for first gene therapy to treat children with rare inherited neurological disease

Based on evidence from a single-arm clinical trial, the European Medicines Agency (EMA) has recommended granting a marketing authorisation in the European Union for the gene therapy SKYSONA™ for the treatment of children with cerebral adrenoleukodystrophy (CALD), a severe form of a rare inherited neurological disease.

SKYSONA™, developed by bluebird bio, is a one-time treatment which can only be given in a specialised hospital by doctors who are experienced in treating patients with CALD, transplanting bone marrow, and using gene therapy medicines. This therapy was accepted into PRIME, a support scheme developed by EMA for promising new medicines that address an unmet medical need.

Path to patient access: The EMA opinion will now be sent to the European Commission (EC) for the adoption of a decision on an EU-wide marketing authorisation. Once a marketing authorisation has been granted, decisions about price and reimbursement will take place at the level of each Member State in the context of the national healthcare system.

More information here: https://www.ema.europa.eu/en/news/first-gene-therapy-treat-children-rare-inherited-neurological-disease