Pharma Focus Asia

ACADIA Pharmaceuticals and Neuren Pharmaceuticals Announce Rare Pediatric Disease Designation for Trofinetide for the Treatment of Rett Syndrome

Friday, March 06, 2020

CADIA Pharmaceuticals Inc. and Neuren Pharmaceuticals Limited announced today that the U.S. Food and Drug Administration (FDA) granted Rare Pediatric Disease (RPD) designation to trofinetide for the treatment of Rett syndrome, a serious and rare neurological disorder. Upon FDA approval of a product with RPD designation, the sponsor may 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.”

RPD designation is granted by the FDA in the case of serious or life-threatening diseases affecting fewer than 200,000 people in the U.S. and primarily in individuals 18 years of age and younger. Trofinetide was previously granted Fast Track Status and Orphan Drug Designation for Rett syndrome in the U.S. and Orphan Drug Designation for Rett syndrome in Europe.

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