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.