EU approves first medicine for early treatment of childhood cALD
EU approves first medicine for early treatment of childhood cALD
The European Commission has approved Nezglyal for the treatment of cerebral adrenoleukodystrophy (cALD) in boys aged 2–12 years.
It is the first pharmacological treatment approved in the EU for this rapidly progressing neurodegenerative disease.
cALD is a rare inherited condition that damages the protective covering of nerve fibres in the brain. The disease can progress quickly and lead to severe neurological decline.
Nezglyal:
- is taken orally;
- is given once daily;
- is intended for early-stage disease in children who already have brain lesions but do not yet show signs of active inflammation.
Experts stress that neurological damage in cALD can be irreversible, making early treatment especially important.
Until now, there were no medicines available for early intervention. More advanced cases may be treated with hematopoietic stem cell transplantation, but this option depends on finding a donor and can only be used within a limited treatment window.
The approval applies across all 27 EU Member States, as well as Norway, Iceland and Liechtenstein. The first commercial launch is expected in Germany by the end of 2026.