ACADIA Pharmaceuticals and Neuren Pharmaceuticals Announce Rare Pediatric Disease Designation for Trofinetide for the Treatment of Rett Syndrome
With No Approved Treatments in Rett Syndrome, FDA Decision Highlights Significant Unmet Need
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"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
RPD designation is granted by the FDA in the case of serious or life-threatening diseases affecting fewer than 200,000 people in the
About Rett Syndrome
Rett syndrome is a debilitating neurological disorder that occurs primarily in females following apparently normal development for the first six months of life. Rett syndrome has been most often misdiagnosed as autism, cerebral palsy, or non-specific developmental delay. 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.
Rett syndrome occurs worldwide in approximately one of every 10,000 to 15,000 female births and in
Trofinetide is an investigational drug. It is a novel 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. Trofinetide has been granted Fast Track Status and Rare Pediatric Disease Designation for the treatment of Rett Syndrome in the
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. The Phase 3 clinical program is progressing as planned with 11 study sites recruiting and more sites expected in the future.
ACADIA is a biopharmaceutical company focused on the development and commercialization of innovative medicines to address unmet medical needs in central nervous system disorders. ACADIA has developed and commercialized the first and only medicine approved for the treatment of hallucinations and delusions associated with Parkinson’s disease psychosis. ACADIA also has ongoing clinical development efforts in additional areas with significant unmet need, including dementia-related psychosis, the negative symptoms of schizophrenia, major depressive disorder, and Rett syndrome. This press release and further information about ACADIA can be found at www.acadia-pharm.com.
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 the license agreement between ACADIA and Neuren, Neuren is eligible to receive one third of the market value of any Rare Pediatric Disease Priority Review Voucher, if awarded by the
Statements in this press release that are not strictly historical in nature are forward-looking statements. These statements include but are not limited to statements regarding the Phase 3 clinical trial evaluating trofinetide; the likelihood of success of such clinical trial; the prospects for FDA approval of trofinetide for Rett syndrome and other indications; and the success of any efforts to commercialize trofinetide in
This ASX-announcement contains forward-looking statements that are subject to risks and uncertainties. Such statements involve known and unknown risks and important factors that may cause the actual results, performance or achievements of Neuren to be materially different from the statements in this announcement.
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