Neuren Pharmaceuticals (ASX:NEU) has been granted Rare Pediatric Disease (RPD) designation for its Rett syndrome treatment, a serious and rare neurological disorder mainly affecting little girls.

The US Food and Drug Administration (FDA) granted RPD designation to trofinetide, an investigational drug designed to treat the core symptoms of Rett syndrome by reducing neuroinflammation and supporting synaptic function.

RPD designation is granted by the FDA for serious or life-threatening childhood diseases affecting fewer than 200,000 people in the US. Trofinetide was previously granted Fast Track Status and Orphan Drug Designation for Rett syndrome in the US, and Orphan Drug Designation for Rett syndrome in Europe.

Neuren is working on a Rett treatment with NASDAQ-listed ACADIA Pharmaceuticals, after the latter bought an exclusive licence for trofinetide in 2018 for the development and commercialisation in North America.


Under the terms of the license agreement, Neuren is eligible to receive one third of the market value of any Rare Pediatric Disease Priority Review Voucher.

In January 2020, the Report to Congressional Committees on the FDA’s Priority Review Voucher Programs noted that vouchers were sold in April 2019 and July 2019 for $US105m ($159.3m) and $US95m respectively.

“We are pleased that the FDA has recognised 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,” ACADIA president Serge Stankovic said.

“This is an encouraging step forward as we continue to enrol patients in our phase three LAVENDER study with results expected in 2021.”

The phase three trofinetide clinical trials includes LAVENDER, a 12-week, double-blind, placebo controlled study, and LILAC, an open-label, long-term extension study.

Neuren says the program is progressing as planned with 11 study sites recruiting and more sites expected in the future.

The disease Neuren and ACADIA are trying to treat is a debilitating neurological disorder that occurs primarily in girls between six and 18 months old, and symptoms include the loss of purposeful hand use and spoken communication, seizures, and scoliosis.

Rett syndrome has been misdiagnosed as autism, cerebral palsy, or non-specific developmental delay, and is caused by mutations on the X chromosome on a gene called MECP2.

Rett syndrome occurs worldwide in approximately one of every 10,000 to 15,000 female births. Currently, there are no FDA-approved medicines for the treatment of Rett syndrome.


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