Neuren Pharmaceuticals experienced a share price decline on Tuesday following an announcement from its partner, Acadia Pharmaceuticals. Acadia stated it will request a re-examination of the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) decision to deny marketing authorisation for trofinetide, a drug intended to treat Rett syndrome in patients aged two years and older. Neuren Pharmaceuticals focuses on developing and commercialising novel therapies to treat neurological disorders. Their aim is to address unmet medical needs through innovative pharmaceutical solutions.
The CHMP’s refusal was reportedly based on concerns regarding the limited extent of the treatment’s effect over a 12-week period, incomplete coverage of key Rett syndrome symptoms, and the influence of patient discontinuations on long-term results. This decision came despite the pivotal LAVENDER trial having successfully met its primary and key secondary endpoints. The EMA noted these concerns after their review of the submitted data.
Trofinetide has already secured approval in the United States, Canada, and Israel. In these countries, it stands as the first and only approved treatment option specifically for Rett syndrome, marking a significant advancement in managing the condition. Rett syndrome is a rare genetic neurological and developmental disorder that affects brain development, primarily in females.
Acadia and Neuren have indicated their intention to collaborate with European regulators to address the concerns raised by the CHMP. Their aim is to provide additional data and arguments in support of trofinetide’s approval in Europe, highlighting the potential benefits for patients with Rett syndrome. As of Tuesday, Neuren’s shares were down by 5.5 per cent following this news.
