Health Canada OKs Acadia's DAYBUE for Rett Syndrome

Acadia Pharmaceuticals has secured marketing authorization from Health Canada for its drug DAYBUE (trofinetide) to treat Rett syndrome in both adult and pediatric patients aged two years and older. Rett syndrome is a rare neurodevelopmental disorder that predominantly affects females. This condition is marked by significant developmental regression after the initial two years of normal growth, leading to a requirement for lifelong, round-the-clock care for most patients.

The approval of DAYBUE through Health Canada's priority review process is notable as it is the first and only drug approved in the country specifically for this condition. Catherine Owen Adams, CEO of Acadia, remarked that this authorization is a major milestone for the Canadian Rett syndrome community and underscores Acadia's ongoing efforts to make this therapy more accessible to patients and their families. She emphasized anticipation in making DAYBUE available to Canadian patients with Rett syndrome promptly.

The authorization of DAYBUE in Canada applies to patients who weigh at least nine kilograms. This approval was based on favorable results from the Phase III LAVENDER trial, which was a critical study that evaluated the safety and efficacy of trofinetide compared to a placebo. The trial involved 187 girls and young women aged between five and 20 years old.

The study had two primary endpoints: the first was the change in the total score from baseline in the Rett Syndrome Behavior Questionnaire (RSBQ) as reported by caregivers, and the second was the score of clinical global impression-improvement (CGI-I) scale as assessed by clinicians after 12 weeks. The RSBQ is a caregiver-completed 45-item scale that measures various symptoms of Rett syndrome, including hand movements, repetitive behaviors, nighttime behaviors, and vocalizations.

In March 2023, DAYBUE also received approval from the US Food and Drug Administration for the treatment of Rett syndrome, further validating the drug's potential and effectiveness in managing this challenging condition.