Acadia Pharmaceuticals Faces EU Setback for Rett Syndrome Drug Daybue, Seeks Re-Examination
Acadia Pharmaceuticals (Nasdaq: ACAD) is navigating a regulatory hurdle in Europe as the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has issued a negative opinion regarding its Marketing Authorization Application (MAA) for trofinetide, marketed as Daybue, for the treatment of Rett syndrome.
CHMP’s Negative Trend Vote and Acadia’s Response
The CHMP informed Acadia of a negative trend vote following an oral explanation session.1 Despite this setback, Acadia intends to request a re-examination of the opinion once the CHMP formally adopts it.1
Understanding Rett Syndrome and Trofinetide
Rett syndrome is a rare neurodevelopmental disorder primarily affecting females, impacting approximately 1 in 10,000 to 15,000 female births worldwide.3 Trofinetide aims to address the core symptoms of this debilitating condition.
Previous Approvals and Clinical Data
Trofinetide has already received approval in the United States, Canada, and Israel.1 The FDA approval was based on the pivotal Lavender trial, which involved 187 female patients with Rett syndrome.2 Acadia reports that over 1,000 patients are currently on active treatment globally.1
CHMP Concerns and Acadia’s Perspective
The CHMP’s negative decision stemmed from concerns regarding the magnitude of the treatment effect observed after 12 weeks, the comprehensiveness of symptom capture in the study, and potential limitations in assessing long-term outcomes due to patient discontinuations.3
Catherine Owen Adams, Chief Executive Officer of Acadia, expressed disappointment but emphasized the strength of the data supporting trofinetide’s approval in other regions, highlighting the meaningful benefits the drug can deliver.1
Looking Ahead
Acadia remains committed to working with the EMA and other stakeholders to advance trofinetide as a potential treatment option in the EU.1 The company’s request for a re-examination will be crucial in determining the future availability of Daybue for individuals and families in Europe affected by Rett syndrome.
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