A First for Rett: FDA Approves Trofinetide for Treatment of Rett Syndrome!

A First for Rett: FDA Approves Trofinetide for Treatment of Rett Syndrome!

Categories: Blog, Clinical Trials, Clinics, Family Empowerment, Homepage, News, Research

This first-ever treatment is approved for individuals with Rett syndrome.

(Updated March 19, 2023)

On March 10, 2023, Acadia Pharmaceuticals announced that their investigational drug, Trofinetide, has been approved by the U.S. Food and Drug Administration (FDA) for the treatment of Rett syndrome. The drug, marketed under the name DAYBUE, is expected to be commercially available in the United States by the end of April, 2023. To jump to resources, links & recent news, click here

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Quick Facts:

  • DAYBUE (trofinetide) is the first treatment for Rett syndrome to receive FDA approval.
  • DAYBUE is approved for the treatment of Rett syndrome in individuals two years of age and older.
  • DAYBUE is expected to be available in the U.S. by the end of April, 2023.
  • Trofinetide works by reducing inflammation in the brain, stopping certain types of cells from becoming overactive, and increasing the amount of the naturally occurring protein called IGF-1.
  • In studies, participants taking Trofinetide saw improvement across several key domains including social communication, fine motor skills, and breathing.
  • For Acadia Pharmaceuticals’ full press release, click here.
  • Acadia Pharmaceuticals hosted an educational webinar for caregivers to learn more about DAYBUE™ and support services offered by Acadia Connect® on March 28, 2023, at 7 p.m. ET. (Click here to view the recording)


This is truly a historic day for the Rett syndrome community, the culmination of a journey that began more than a decade ago and succeeded only thanks to the perseverance and commitment of the families, researchers, and donors who were there every step of the way. We at IRSF are honored to have been a partner and advocate for families throughout this journey, identifying Trofinetide as applicable in Rett syndrome under then-CSO Dr. Steven Kaminsky in 2012, providing nearly $2 million in funding for the Phase 2 adult and pediatric trials, and facilitating study enrollment at our Center of Excellence network trial sites. Today, we celebrate with you and thank you for participating, donating, supporting, and raising your voice for Rett syndrome.

We are grateful for the investments made by Acadia to bring this first approved treatment through Phase 3 and to FDA approval. We are grateful to all of our partners—doctors, researchers, scientists, our board of directors, and donors—who supported our vision to relentlessly pursue the development of trofinetide. But mostly, we celebrate and thank those courageous families who participated in the clinical trial process of trofinetide. It is because of their bravery that the Rett syndrome community is able to celebrate this milestone.
– Melissa Kennedy, IRSF Chief Executive Officer


Rett is not one-size-fits-all; because Rett syndrome has a broad range of presentations, we need a broad range of treatments and cures. No one treatment can benefit every single person with Rett, so we must continue to fund the most promising research. This is just the first of many treatments needed.

And there is so much reason for hope. Forty years ago, no one was investing in Rett syndrome research. Now that landscape has dramatically changed, positioning us for more breakthroughs like this. In 2023 alone, multiple gene therapy clinical trials will begin enrolling, 20+ companies are investing in treatments, and there are more than 40 active research projects accelerating our knowledge of what causes Rett syndrome.

With this momentum, we are positioned for more success and more ways to provide treatments that will make a profound difference in overcoming Rett and helping those with Rett reach their fullest potential. IRSF is with the community leading the fight, recently approving the largest research investment in our history–over $4.4M! –as part of our ongoing commitment to invest broadly and smartly in the most promising research at every stage.


We know you have many questions about DAYBUE, 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 DAYBUE’s benefit to your child and its addition to your treatment plan.

We promise to continue advocating at the federal and state level to ensure all families have affordable access to DAYBUE. This will be the first fight to access an FDA-approved treatment for Rett, but it won’t be the last.

With this announcement comes an opportunity 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.

“We are thrilled that families now have a treatment option and the hope that their children may be able to do more and struggle less,” shared Kennedy. “Our mission of accelerating research continues until we can bring treatments including cures to ALL Rett syndrome patients, male and female, and with different presentations of this disease.”


Learn More About DAYBUE (trofinetide)
Visit Acadia Pharmaceuticals’ just-launched website, DAYBUE.com, for prescribing information, FAQs, and more for DAYBUE as they become available. Registration for their March 28th webinar for caregivers is also now open by clicking here.

Make Your Voice Heard
FDA approval is just the beginning. In many cases, access to new treatments like DAYBUE often lies with decisions made at the state level. Now more than ever, it’s critical that your elected state officials hear from you and learn about Rett syndrome. Use our simple Advocacy Tool to find your state representatives and raise awareness for all those living with Rett.

Give to Support Rett Syndrome Research
Today is an incredible milestone representing the culmination of more than a decade of strategically funding promising, innovative research thanks to the many donors who make our mission possible. Give now to help us continue to support the development of treatments like Trofinetide.

IRSF is with you every step of the way. We will do everything we can to answer your questions in the coming weeks and months or direct you to the resources you need. You can click the button below to use our contact form, email us directly at [email protected], or reach out to one of our 18 IRSF-designated Rett syndrome Center of Excellence clinics.


Additional Resources & Links:

Video Series: What you Need to Know About DAYBUE (trofinetide)
Acadia Pharmaceuticals Press Release – 3.10.23
IRSF Press Release – 3.11.23
Acadia Pharmaceuticals Press Release – 4.17.23
Message from IRSF CEO, Melissa Kennedy

DAYBUE Website (for patients)
DAYBUE Website (for health professionals)
Acadia Connect™ Support Program

What Center of Excellence Directors & PIs are saying about DAYBUE:

Robin Ryther, MD, PhD – Rett Spectrum Clinic at WashU in St. Louis School of Medicine
Alan Percy, MD & Amitha Ananth, MD – UAB Rett Syndrome Clinic
Steve Skinner, MD – Greenwood Genetic Center
Jennifer Martelle Tu, MD, PhD – UCSF Benioff Children’s Hospital Oakland (Article | Podcast)

Plain Language Summaries & Publications by COE Directors:

Results from the LAVENDER study: trofinetide treatment for Rett syndrome
Recommendations for managing diarrhea from trofinetide use in individuals with Rett syndrome
Trofinetide for the treatment of Rett syndrome: a randomized phase 3 study