Promise for Rett syndrome

ACADIA Pharmaceuticals and Neuren Pharmaceuticals announce Rare Pediatric Disease designation for trofinetide for the treatment of Rett syndrome

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SAN DIEGO & MELBOURNE, Australia—ACADIA Pharmaceuticals Inc. and Neuren Pharmaceuticals Limited have announced that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease (RPD) designation to trofinetide for the treatment of Rett syndrome, a serious and rare neurological disorder. With FDA approval, ACADIA may now be eligible to receive a Priority Review Voucher, which can be used to obtain FDA review of a New Drug Application for another product in an expedited period of six months.
“We are pleased that the FDA has recognized the unmet need currently experienced by Rett patients and their families and our goal is to bring a treatment option forward as soon as possible,” said Serge Stankovic, M.D., M.S.P.H., ACADIA’s president. “This is an encouraging step forward as we continue to enroll patients in our Phase 3 LAVENDER study, with results expected in 2021.”
Trofinetide is an investigational drug, a novel synthetic analog of the amino‐terminal tripeptide of IGF-1 designed to treat the core symptoms of Rett syndrome. It works by potentially reducing neuroinflammation and supporting synaptic function. In the central nervous system, IGF-1 is produced by both of the major types of brain cells — neurons and glia — and it is critical for both normal development and for response to injury and disease. Trofinetide has been granted both Fast Track Status and Rare Pediatric Disease Designation for the treatment of Rett Syndrome in the U.S., and Orphan Drug Designation in the U.S. and Europe for both Rett syndrome and Fragile X syndrome.
The Phase 3 trofinetide clinical program includes LAVENDER — a 12-week, double-blind, placebo-controlled study — and LILAC, an open-label, long-term extension study. ACADIA and Neuren note that the Phase 3 clinical program is progressing as planned, with 11 study sites recruiting and more sites expected in the future.
Rett syndrome is a debilitating neurological disorder caused by mutations on the X chromosome on a gene called MECP2. It occurs primarily in females following apparently normal development for the first six months of life. Rett syndrome is frequently misdiagnosed as autism, cerebral palsy or non-specific developmental delay. There are over 200 different mutations found on the MECP2 gene that interfere with its ability to generate a normal gene product.
Rett syndrome occurs worldwide in approximately one of every 10,000 to 15,000 female births. In the U.S. it impacts 6,000 to 9,000 patients. Rett syndrome causes problems in brain function that are responsible for cognitive, sensory, emotional, motor and autonomic function. Symptoms typically present between six to 18 months of age. Patients experience a period of rapid decline, with loss of purposeful hand use, spoken communication and inability to independently conduct activities of daily living. Symptoms also include seizures, disorganized breathing patterns, scoliosis and sleep disturbances. Currently there are no FDA-approved medicines for the treatment of Rett syndrome.

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