Acadia seeks regulatory approval in EU of trofinetide as Rett treatment
Therapy already approved in US, Canada as Daybue for patients 2 and older
Acadia Pharmaceuticals has submitted a marketing authorization application for trofinetide to European Union regulators, seeking the therapy’s approval for adults and children, ages 2 and older, with Rett syndrome.
The application to the European Medicines Agency (EMA) is Acadia’s first such submission in Europe, according to a company press release.
If it’s ultimately approved, trofinetide would become the first treatment for Rett syndrome to win approval in the European Union. The therapy already is approved for this indication in the U.S. and Canada, where it is marketed under the name Daybue.
“This application underscores our continued dedication to the Rett community, and our commitment to making a meaningful impact for people living with Rett syndrome and their caregivers in the EU, who currently have no approved treatment options specifically for this condition,” said Catherine Owen Adams, CEO of Acadia. “We look forward to working with the EMA to address this unmet need in the hopes of potentially bringing this therapy to families who are impacted by Rett syndrome.”
Application for trofinetide in EU based on LAVENDER trial data
News of the application was celebrated by Rett Syndrome Europe, a volunteer-led organization that aims to support families affected by Rett.
“The Rett syndrome community in Europe is excited by the prospect of having an available treatment option,” said Becky Jenner, the nonprofit’s president. “Rett syndrome has a significant impact on people living with this condition, and also impacts caregivers and families who often have to navigate challenging symptoms that impact their daily lives.”
Rett is a genetic disorder that causes symptoms including abnormal behaviors and difficulty with communication. Trofinetide is thought to work to ease these symptoms by lessening inflammation and improving the health of connections between nerve cells in the brain.
The solution therapy is taken daily by mouth or via a feeding tube.
The Rett syndrome community in Europe is excited by the prospect of having an available treatment option.
Acadia’s application in the EU is based mainly on data from a Phase 3 clinical trial called LAVENDER (NCT04181723). The results of that study, completed in 2021, showed trofinetide was better than a placebo at improving measures of communication and reducing scores related to fear, anxiety, body rocking, and blank facial expressions in girls and young women with Rett. The positive results from LAVENDER were also the basis for last year’s approvals of the therapy in the U.S. and Canada.
Participants who completed the LAVENDER study had the option to continue into the extension studies LILAC-1 (NCT04279314) and LILAC-2 (NCT04776746). In both, participants were given trofinetide for up to three years, with the results indicating continuous improvements in communication and normalization of behaviors. The most common side effects of the therapy seen in trials were vomiting and diarrhea.
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