On Friday, March 10, 2023, the United States Food and Drug Administration (FDA) approved Acadia Pharmaceuticals’ drug for genetic Rett syndrome, the company announced in a press release.
DAYBUE™ (trofinetide), the first and only drug approved for the treatment of the condition, is approved “for the treatment of Rett syndrome in adult and pediatric patients two years of age and older.”
“As the first FDA-approved drug for the treatment of Rett syndrome, DAYBUE now offers the potential to make meaningful differences in the lives of patients and their families who have lacked options to treat the diverse and debilitating array of symptoms caused by Rett syndrome,” said Steve Davis, Acadia’s Chief Executive Officer.
In addition, Acadia has received “a Rare Pediatric Disease Priority Review Voucher, which can be used to obtain priority review for a subsequent application.”
The company expects DAYBUE to be available in the United States by the end of April 2023.
Profoundly debilitating condition
Rett syndrome, a complex, rare neurodevelopmental disorder typically caused by a genetic mutation on the MECP2 gene, is believed to affect 6,000 to 9,000 patients in the U.S., with a diagnosed population of approximately 4,500 U.S. patients, per Acadia data.
This profoundly debilitating condition “is characterized by a period of normal development until six to 18 months of age, followed by significant developmental regression with loss of acquired communication skills and purposeful hand use,” as well as “an array of unpredictable symptoms throughout the course of a patient’s life.”
The results from DAYBUE’s pivotal Phase 3 LAVENDER study showed statistically significant improvement in patients impacted by Rett “as measured by the change from baseline in Rett Syndrome Behaviour Questionnaire (RSBQ) total score (p=0.018) and the Clinical Global Impression-Improvement (CGI-I) scale score (p=0.003) at week 12.”
Study data showed that DAYBUE significantly outperformed the placebo on both co-primary efficacy endpoints.
“With today’s FDA decision, those impacted by Rett have a promising new treatment option that has demonstrated benefit across a variety of Rett symptoms, including those that impact the daily lives of those living with Rett and their loved ones,” said Melissa Kennedy, Chief Executive Officer of the International Rett Syndrome Foundation (IRSF).
More treatments needed for Rett syndrome
While lauding FDA’s decision as truly historic for the Rett syndrome community, IRSF pointed out that DAYBUE is just the first of many treatments needed; “no one treatment can benefit every single person with Rett,” since the condition has a broad range of presentations. IRSF called for continued funding of the most promising research.
“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,” the foundation said on its website.