CANADA—Health Canada has granted Acadia Pharmaceuticals marketing authorisation for DAYBUE (trofinetide), which is intended to treat Rett syndrome in adults and children aged two years and above.

This comes shortly after the therapy received approval from the US Food and Drug Administration for treating Rett syndrome in March 2023, further solidifying its significance in neurodevelopmental disorders.

This approval is particularly important as it establishes DAYBUE as the first and only medication authorized in Canada specifically for this rare neurodevelopmental disorder.

Rett syndrome predominantly affects females and is characterized by a profound regression in development after an initial two years of seemingly normal growth.

As a result, most individuals diagnosed with this condition will require lifelong, 24-hour care, underscoring the critical need for effective treatment options.

The approval of DAYBUE is based on the promising outcomes from the Phase III LAVENDER trial, which evaluated the drug’s safety and efficacy against a placebo.

 This pivotal study involved 187 girls and young women, aged between five and 20, providing essential data for regulatory approval.

The study’s co-primary endpoints were twofold: the change in the total score from baseline in the Rett Syndrome Behavior Questionnaire (RSBQ) as reported by caregivers, and the score on the Clinical Global Impression-Improvement (CGI-I) scale, as assessed by clinicians at week 12.

 The RSBQ, a comprehensive 45-item scale filled out by caregivers, evaluates various symptoms associated with Rett syndrome, including hand movements, repetitive behaviors, night-time disturbances, and vocalizations.

Commenting on this authorization, Acadia CEO Catherine Owen Adams expressed that Health Canada’s decison represents a significant milestone for the Rett syndrome community in Canada.

 She reiterated Acadia’s commitment to enhancing access to this therapy for patients and their families, emphasising their eagerness to bring DAYBUE to Canadian patients as quickly as possible.

Adding to the dialogue, Dr. Anita Datta, Co-Director of the Rett Program at BC Children’s Hospital and Clinical Associate Professor at the University of British Columbia, emphasized the complexity and debilitating nature of Rett syndrome.

She noted that the disorder manifests differently across patients and leads to a wide array of symptoms throughout their lives.

Until now, treatment options have largely focused on symptomatic management, as specific therapies for Rett syndrome had been lacking.

Sabrina Millson, President of the Ontario Rett Syndrome Association (O.R.S.A.), highlighted the extensive impact Rett syndrome has not only on individuals living with the disorder but also on their families, friends, and loved ones.

Millson described the authorization of DAYBUE as a historic milestone for the Canadian Rett syndrome community, who have long awaited an approved treatment.

She expressed hope that this new therapy will be made accessible through both private and publicly funded drug plans as swiftly as possible for those who could benefit.

In Canada, the estimated prevalence of Rett syndrome ranges between 600 to 900 patients.

Symptoms may include loss of communication skills, difficulties with purposeful hand use, gait abnormalities, and stereotypic hand movements, such as hand-wringing, clapping, and rubbing.

Most patients with Rett syndrome can expect to live into adulthood but will require round-the-clock care to support their complex needs.

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