The European Commission has authorised Acadia Pharmaceuticals’ trofinetide for treating neurobehavioural symptoms of Rett syndrome in patients aged five and above, marking a significant breakthrough for a rare and severe neurodevelopmental disorder.
The European Commission has cleared Acadia Pharmaceuticals’ trofinetide for the treatment of neurobehavioural symptoms linked to Rett syndrome in adults and children aged five and over, making it the first therapy approved for the condition in the European Union. European Pharmaceutical Review reported that the oral medicine is an analogue of glycine-proline-glutamate, while Acadia said the authorisation now applies across all 27 EU member states, as well as Iceland, Liechtenstein and Norway.
The company said the decision was based largely on the Phase 3 LAVENDER study, which it said showed statistically significant and clinically meaningful improvements on the key measures used in Rett syndrome research, including the Rett Syndrome Behaviour Questionnaire and the Clinical Global Impression-Improvement scale. Acadia chief executive Catherine Owen Adams said the approval marked an important advance for families who have long had very limited treatment options. Professor Nadia Bahi, a paediatric neurologist at Necker Enfants Malades University Hospital in Paris, said the ruling recognised a major unmet medical need and gave clinicians a new option for managing neurobehavioural symptoms.
Before the Commission’s decision, the European Medicines Agency’s Committee for Medicinal Products for Human Use had already issued a positive opinion on trofinetide after a re-examination procedure, according to Pharmabiz. Acadia said it will now begin pricing and reimbursement discussions with national authorities, a step that will determine how quickly patients can actually access the medicine in different countries.
Rett syndrome is a rare and severe neurodevelopmental disorder that affects almost exclusively girls. Medical literature cited in the related reports says it is often first noticed after a period of apparently typical early development, usually between six and 18 months, when skills begin to stall or regress. Loss of communication abilities and purposeful hand use is common, and many patients develop repetitive hand movements and walking difficulties. Most people with Rett syndrome live into adulthood but require round-the-clock care. The condition is usually caused by mutations in the MECP2 gene, and earlier research has estimated its prevalence at roughly one in 10,000 to 15,000 female births.
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