US-based biopharmaceutical company Acadia Pharmaceuticals has received Health Canada marketing authorisation for Daybue (trofinetide) to treat a rare genetic neurological disorder.
Daybue is a synthetic analog of the N-terminal tripeptide of insulin-like growth factor 1, approved by the US Food and Drug Administration (FDA) approval in March last year.
Health Canada indicated the drug for the treatment of Rett Syndrome in adults and children, aged two years and above, and weighing at least 9kg.
Rett syndrome is a rare, complex, neurodevelopmental disorder that affects primarily females, causing significant regression after normal development in the first two years of life.
Its symptoms may include loss of communication skills, purposeful hand use, gait abnormalities and stereotypic hand movements such as hand wringing, clapping, and rubbing.
With the regulatory approval, granted under the priority review process, Daybue becomes the first and only drug approved for the treatment of Rett syndrome in Canada.
Acadia CEO Catherine Owen Adams said: “Health Canada’s authorisation of Daybue is a significant milestone for the Rett syndrome community in Canada and another step forward in Acadia’s commitment to increase access to this therapy for patients and their families.”
Health Canada approved Acadia’s regulatory submission based on positive results from Phase 3 LAVENDER study in 187 females aged between 5 and 20 years with Rett syndrome.
The Phase 3 study evaluated the efficacy and safety of Daybue compared to placebo.
The co-primary endpoints include change from baseline in the Rett Syndrome Behaviour Questionnaire (RSBQ) and Clinical Global Impression-Improvement (CGI-I) scale scores.
RSBQ is a rating scale that assesses symptoms of Rett syndrome, including breathing, hand movements, repetitive behaviours, night-time behaviours, vocalisations, facial expressions, eye gaze, and mood.
The key secondary endpoint includes measuring the change in the Communication and Symbolic Behavior Scales Development Profile Infant-Toddler (CSBS-DP-IT) score.
Ontario Rett Syndrome Association (ORSA) president Sabrina Millson said: “Rett syndrome not only has a significant impact on the individuals living with this disorder, but on their families, friends and loved ones as well.
“Today’s news is a historic milestone for the Canadian Rett syndrome community, who have eagerly awaited an approved treatment.
“Now that this therapy has been approved, we hope it will be made accessible through private and publicly funded drug plans as quickly as possible for all those who could benefit from it.”
