FDA Grants ACADIA and Neuren Rare Pediatric Disease Designation for Trofinetide in Rett Syndrome

The U.S. Food and Drug Administration has granted ACADIA Pharmaceuticals and Neuren Pharmaceuticals Rare Pediatric Disease designation for trofinetide, their experimental treatment for Rett syndrome, a serious and rare neurological disorder.

“This is an encouraging step forward as we continue to enroll patients in our Phase 3 LAVENDER study with results expected in 2021,” said Serge Stankovic, president of ACADIA.

Rett syndrome is a non-inherited, genetic, post-natal, progressive neurodevelopmental disorder that occurs almost exclusively in girls and leads to severe impairments, affecting nearly every aspect of the child’s life. Rett syndrome is caused by mutations on the X chromosome on a gene called MECP2. There are more than 200 different mutations found on the MECP2 gene that interfere with its ability to generate a normal gene product.

The hallmark of Rett syndrome is near constant repetitive hand movements while awake. The disease is characterized by normal early growth and development (6 to 18 months) followed by a slowing of development, loss of purposeful use of the hands, distinctive hand movements, autistic features, slowed brain and head growth, ataxia, seizures, and intellectual disability. There is currently no cure for Rett syndrome.

Trofinetide is an experimental synthetic analog of the amino‐terminal tripeptide of IGF-1 designed to treat the core symptoms of Rett syndrome 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. IGF-1 in the brain is critical for both normal development and for response to injury and disease. The phase 3 trofinetide clinical program includes a 12-week, double-blind, placebo-controlled study, and LILAC, an open-label, long-term extension study, which are progressing as planned.

The FDA grants Rare Pediatric Disease designation for serious or life-threatening diseases with manifestations in individuals aged from birth to 18 years, including access to the FDA’s expedited review and approval process. The designation makes trofnetide eligible for a Rare Pediatric Disease Priority Review voucher upon approval of the therapy by the FDA.

The vouchers can be used to reduce the time of an FDA new drug approval review to six months from ten months. The vouchers are potentially lucrative because they are transferable. Most recently, Sarepta sold its Rare Pediatric Disease voucher to Vifor Pharma $111 million in February 2020.

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.

In 2018, ACADIA entered into an exclusive North American license agreement with Neuren for the development and commercialization of trofinetide for Rett syndrome and other indications. Under the terms of their agreement, Neuren is eligible to receive one third of the market value of any Rare Pediatric Disease Priority Review Voucher for trofinetide.

Australia-based Neuren has completed mid-stage clinical trials of trofinetide for both Rett syndrome and Fragile X syndrome.

Photo: Serge Stankovic, president of ACADIA

Editor’s note: This story was updated to correct the most recent sales of a rare pediatric disease voucher.

Author: Rare Daily Staff