BIG NEWS for our international Rett syndrome community! Today, Acadia Pharmaceuticals announced they have acquired the rights to make trofinetide available for the treatment of Rett syndrome across the globe.
This is an essential starting point in ensuring ALL Rett families have access to this treatment. Acadia already retains the rights in North America and earned FDA approval in March to make trofinetide, under the name DAYBUE, commercially available for the treatment of Rett in the United States. Now, with this updated agreement with Neuren Pharmaceuticals, Acadia can begin exploring opportunities to make trofinetide available everywhere.
While this is a critical first step in achieving global availability, this process could take some time and there is much that is unknown right now. Acadia intends to submit a New Drug Submission (NDS) for trofinetide in Canada in the next 18 months with plans for Europe, Asia and other regions to be announced at a later date. We promise to keep the community updated as more information becomes available.
OUR PROMISE TO FAMILIES
We know you have many questions about trofinetide, and we promise to be a resource of timely, updated information on when and how your child can access this treatment. While we will keep you informed, your doctor is the best resource to discuss this treatment’s potential benefit to your child and its addition to your treatment plan.
We also want to restate our commitment that we will press harder for more research, more trials, and more FDA-approved drugs to help in overcoming Rett. We promise to continue to relentlessly pursue treatments and curative approaches to create a world without Rett syndrome.
MORE FOR YOU
IRSF Resources on DAYBUE™ (trofinetide)
Read our post from the day trofinetide was approved by the FDA and watch our video series “What You Need to Know About DAYBUE,” featuring panel conversations with parents and physicians who participated in the trofinetide US clinical trials. You can also reach out to us with any questions at [email protected].
Resources from Acadia
Visit Acadia Pharmaceuticals’ website, DAYBUE.com, for prescribing information, FAQs, and more about DAYBUE in the United States. Healthcare providers can also access an HCP-specific website on DAYBUE and sign-up for an upcoming webinar.
Give to Support Rett Syndrome Research
Today is another incredible milestone representing thanks to the many donors who allow us to strategically fund promising and innovative research. Give now to help us continue to support the development of treatments like Trofinetide.