Acadia Pharmaceuticals' DAYBUE™ (trofinetide) Receives FDA Approval for Rett Syndrome Treatment

Neuren Pharmaceuticals has announced that its North American partner, Acadia Pharmaceuticals, has been granted FDA approval for the use of DAYBUE™ (trofinetide) in treating Rett syndrome among pediatric and adult patients aged two years and above. DAYBUE is the first and only FDA-approved treatment for Rett syndrome. Acadia expects DAYBUE to become available by the end of April 2023.

Publish Date: 13-03-2023 Source: Neuren Pharmaceuticals

Rett syndrome (RTT) is a rare genetic neurological disorder that primarily affects girls and causes severe impairments in nearly every aspect of the child’s life, including their ability to speak, walk, eat, and even breathe easily. Rett syndrome is usually recognized in children aged 6 to 18 months when they begin to miss developmental milestones or lose previously acquired abilities. RTT is caused by mutations on the X chromosome on a gene called MECP2. The early-onset seizure type (Hanefeld variant) is frequently caused by mutations in the X-linked CDKL5 gene (Xp22). A translocation involving the NTNG1 gene (1p13.2-p13.1) has also been identified in a patient with early seizures and atypical RTT. The congenital variant (Rolando variant) is generally caused by mutations in the FOXG1 gene (14q11-q13).

Rett syndrome varies worldwide; in the USA, the estimated prevalence is 0.45 to 0.76 cases per 10,000 females aged 2 to 18 years. Germany is the leading and followed by France in the EU region in terms of total diagnosed and prevalent cases.

However, the current Rett syndrome (RTT) treatment market share, market uptake, and attribute analysis concerning the most potential emerging therapies (ANAVEX2-73, Trofinetide, etc..) has been provided under the market outlook section of the study covering 8 MM countries; The United States, EU5 (Germany, Spain, France, Italy, UK) Japan and China.

In terms of pharmacologic therapies, several pharmaceutical products are being approved and under different phases of development for the Rett syndrome treatment. The key companies in the advanced development stage are Anavex Australia Pty Ltd, ACADIA Pharmaceuticals Inc., etc..

Based on solid domain and business knowledge, Thelansis Knowledge Partners has published the market outlook forecast report on Rett syndrome (RTT) to provide a clear understanding of disease area background, epidemiology, current and future competitions, the country-specific standard of care, and the complete market forecast for 2021 to 2032.

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