Growing Trofinetide’s Long-Term Profile as First Approved Rett Syndrome Therapy: Ponni Subbiah, MD, MPH


The senior vice president, global head of medical affairs, and chief medical officer at Acadia Pharmaceutical, provided an overview of LILAC-2, a long-term, open-label study assessing the treatment benefits of trofinetide (Daybue). [WATCH TIME: 6 minutes]

WATCH TIME: 6 minutes

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Rett syndrome, a neurodevelopmental disorder, has been traditionally managed through a combination of physical and occupational therapy, as well as medications that may help with seizures, muscle stiffness, issues with breaking, sleep, the gastrointestinal tract or the heart. A diagnosis of Rett syndrome includes core symptoms such as partial or complete loss of purposeful hand skills, spoken language, difficulty walking, and repetitive hand movements, among others. If Rett syndrome is suspected after evaluation, genetic testing may be needed to confirm the diagnosis.

After years of no approved treatments, the FDA gave greenlight to Acadia Pharmaceuticals’ trofinetide earlier this year. Trofinetide, a novel synthetic analog of the amino-terminal tripeptide of IGF-1 designed to potentially reduce neuroinflammation and supporting synaptic function, was approved earlier this year based on data from the phase 3 LAVENDER study (NCT04181723). Months later, at the 2023 American Epilepsy Society (AES) annual meeting, held December 1-5, in Orlando, Florida, new data from LILAC-2, an open-label extension study (NCT04776746), continued to demonstrate the long-term benefits of trofinetide in this patient population.

Over a 32-month period, trofinetide continued to improve symptoms of Rett syndrome while maintaining a safety profile that was consistent with its previous studies. To learn more about these findings, NeurologyLive® sat down with Ponni Subbiah, MD, MPH, senior vice president, global head of medical affairs, and chief medical officer at Acadia Pharmaceutical. In the conversation, Subbiah spoke about the data leading up to LILAC-2, the importance of growing trofinetide’s treatment profile, and some of the promising safety findings observed.

Click here for more coverage of AES 2023.

1. Percy AK, Neul JL, Benke TA, et al. Trofinetide for the treatment of Rett syndrome: long-term safety and efficacy results from the open-label LILAC-2 study. Presented at: AES 2023; December 1-5; Orlando, FL.

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