EU Approves bluebird bio's Skysona as First Drug Therapy for Rare Disease cALD
核心洞察
The European Commission has approved Nezglyal (leriglitazone), making it the first drug therapy cleared in the EU for cerebral adrenoleukodystrophy (搜索).
The oral PPAR gamma (搜索) agonist is indicated for male cALD patients aged two to 12 with gadolinium-negative brain lesions, a narrower label than requested.
Neuraxpharm (搜索) and Minoryx Therapeutics (搜索) expect a first launch in Germany by the end of the year, with other countries to follow reimbursement talks.
The European Commission has granted Neuraxpharm (搜索) and Minoryx Therapeutics (搜索) approval for Nezglyal (leriglitazone), the first drug therapy authorised in the EU for cerebral adrenoleukodystrophy (搜索) (cALD). The oral PPAR gamma (搜索) agonist is indicated for male cALD patients aged two to 12 who have gadolinium-negative brain lesions, and the companies expect to launch before the end of the year.
cALD is a fatal neurodegenerative condition that destroys the myelin sheath surrounding nerve cells, typically causing acute neurological decline and death within three to four years. The companies originally developed leriglitazone for children and adults but hit a setback in the ADVANCE trial, and their first marketing application was rejected in 2024. Data from the paediatric NEXUS trial, which showed the drug slowed disease progression, supported a second filing. The CHMP recommended the narrower paediatric indication in July rather than the broader children and adults label the companies sought.
Neuraxpharm (搜索) will commercialise the product in Europe, with the first launch expected in Germany by the end of the year and other markets following reimbursement negotiations. The partners are also running a phase 3 trial in adult cALD (CALYX) and a phase 2a trial in children with Rett syndrome (搜索).