FDA Decision Due March 2023 on Trofinetide as 1st-ever Rett Treatment

Prior FDA designations had put trofinetide on fast track for approval

Marisa Wexler, MS avatar

by Marisa Wexler, MS |

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The U.S. Food and Drug Administration (FDA) has set an action date of March 12, 2023 to decide whether or not to approve the experimental oral therapy trofinetide for Rett syndrome.

If approval is granted, trofinetide would become the first treatment for Rett ever approved by the FDA.

The twice-daily therapy was shown in a clinical trial to improve behavior and communication among Rett patients.

Acadia Pharmaceuticals, which submitted an application seeking FDA approval of trofinetide earlier this year, now announced that the regulatory agency has accepted that filing. The FDA also informed Acadia that it is not planning to hold an advisory committee meeting at this time.

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“We’re pleased that the FDA has accepted our NDA [new drug application] filing and we will be working closely with them to facilitate completion of the review in a timely manner,” Steve Davis, CEO of Acadia, said in a press release.

“This milestone reinforces Acadia’s ongoing commitment to advancing research into high unmet needs in disorders affecting the central nervous system [brain and spinal cord],” Davis said.

Trofinetide contains a modified version of glypromate, a protein fragment thought to reduce inflammation and improve the health of connections between nerve cells. It was originally developed by Neuren Pharmaceuticals, which licensed it to Acadia in 2018.

Acadia’s application to the FDA is supported by data from the Phase 3 LAVENDER clinical trial (NCT04181723), which tested trofinetide against a placebo in 187 girls and young women with Rett syndrome. The participants in the study, conducted at 21 locations across the U.S., ranged in age from 5 to 20.

Results showed that, after about three months, participants given trofinetide scored significantly better than those given the placebo across several standardized measures of communication and behavior. The positive findings were met with excitement in the Rett community, which noted that there currently are no approved treatments for Rett in the U.S.

“Rett is a complex disease that can present with a diverse array of symptoms,” said Jeffrey Neul, MD, PhD, a professor at Vanderbilt University Medical Center and investigator in the LAVENDER study.

“In clinical trials, trofinetide demonstrated a significant improvement in a range of Rett syndrome symptoms,” Neul said.

Nearly all participants who completed LAVENDER continued into the ongoing LILAC open-label extension study (NCT04279314), where all participants are being treated with trofinetide.

Acadia also is running an open-label Phase 2/3 trial called DAFFODIL (NCT04988867) that is testing trofinetide in 15 girls with Rett syndrome ages 2 to 5. The results of that trial are expected by July 2023.

The FDA previously granted trofinetide fast track status as well as orphan drug and rare pediatric disease designations. All are designed to incentivize and expedite the development and review of therapies that can improve care for rare and/or serious conditions.