Today, the Rett community in Europe has a reason to feel hopeful.
Rett Syndrome Europe welcomes the positive opinion issued by the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency for Daybu (trofinetide), a medicine intended for the treatment of Rett syndrome.
For our community, this is a meaningful and emotional milestone.
For decades, families affected by Rett syndrome in Europe have lived without a medicine specifically approved for Rett syndrome. At the same time, they have faced the daily reality of a complex condition that can affect communication, movement, breathing, sleep, feeding, epilepsy, anxiety, autonomic symptoms and many other aspects of life.
This moment does not remove the challenges families face every day. It does not answer every question. But it does represent progress. And progress matters.
For decades, families in Europe affected by Rett syndrome have had no medicine specifically approved for the condition, despite the profound impact Rett syndrome has on almost every aspect of daily life. This positive CHMP opinion represents an important milestone and a source of hope for people living with Rett syndrome, their families and caregivers across Europe.
At the same time, we want to communicate clearly and responsibly.
A positive CHMP opinion is an important step, but it is not the final step in the European process. The final decision on European marketing authorisation will be taken by the European Commission. After that, access will depend on national processes in each country, including pricing, reimbursement, clinical guidance and healthcare system readiness.
This means that families should not expect immediate access everywhere in Europe. Timelines may be different from country to country.
As a European patient organization, RSE will continue to advocate for clear information, responsible communication and fair access for families, wherever they live. Families need to understand what is known, what remains uncertain, how treatment decisions will be made, and how safety and real-world outcomes will be followed over time.
Today, we also want to recognise the many people who have contributed to this moment: families who participated in research, clinicians, researchers, national Rett associations, advocates, and everyone who has kept pushing for progress in Rett syndrome over many years.
Today is a day for hope.
It is also a reminder that our work continues: to improve care, strengthen evidence, support research, and ensure that the voices of people living with Rett syndrome and their families remain at the centre of every decision.
Rett Syndrome Europe will continue to share information with our member associations and the wider community as official updates become available.
Important note for families
Families and caregivers should not make treatment decisions based only on this announcement. Questions about treatment suitability, possible benefits, side effects, monitoring and access should be discussed with a qualified clinician familiar with Rett syndrome and the individual medical history of the person affected.
