new hope for families as health canada approves first rett syndrome treatment
newly-approved daybue sparks hope in canadians living with or caring for someone with rett syndrome.
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the newly-approved drug, daybue, has been approved in canada to be used in children over the age of two and adults living with rett syndrome.
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health canada has approved a new treatment for rett syndrome, daybue, that has shown great promise in helping to limit symptoms and restore some parameters of health in children over the age of two living with the condition.
rett syndrome is a debilitating disease affecting 600 to 900 canadians. it starts in infancy and affects predominantly females.
symptoms of the disease, which can include loss of mobility and hand function, slowed development and growth, loss of communication abilities, breathing problems, seizures, irritability, and unusual behaviours or moods, typically begin to show at 12 to 18 months of age.
the treatments available for rett syndrome typically focus on addressing specific symptoms, such as anti-convulsants for seizures.
the first and only treatment for rett syndrome
daybue, also known as trofinetide, was created as a joint effort by neuren pharmaceuticals and acadia pharmaceuticals. it was approved by the u.s. food and drug administration (fda) in march of 2023 due to its clinical trial results showing great promise in helping to safely treat rett syndrome symptoms.
the drug has been approved in canada to be used in children over the age of two and adults living with rett syndrome.
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the positive results were based on the work done in the phase 3 lavender trial. the trial, which included 187 participants between the ages of five and 20, showed that the drug passed safety checks and made significant changes in the presentation of symptoms of rett syndrome.
“we are excited to see this first approval outside the united states, which is a significant milestone in the ongoing program to expand access to daybue,” said jon pilcher, ceo of neuren, in a press release.
the daybue study
daybue is a synthetic analog form of the n-terminal tripeptide of insulin-like growth factor 1, a hormone that works alongside growth hormones to promote the development of bone and tissue within the body.
over the course of 12 weeks, each participant was given the drug or a placebo and evaluated for changes in symptoms and adverse events. the clinical characteristics examined included communication, ambulation, hand use, seizures, attentiveness, eye gaze, and breathing. caregiver burden was also assessed during this 12-week period.
each characteristic endpoint was scored on a scale of zero to seven, with zero indicating no impairment and seven indicating no change or severe impairment.
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positive changes were observed throughout the study in all areas in participants taking the drug versus the placebo group, showing the significant potential daybue has for helping alleviate symptoms and caregiver burden in those living with and caring for someone with rett syndrome.
adverse events were not common after using daybue, with the most notable side effects being diarrhea, weight loss, and vomiting. fevers and an increase in seizures, anxiety, decreased appetite, and tiredness were also noted.
the drug’s mechanism of action is still unknown, leading to more in-depth research into how the drug works as it does. it’s also worth noting that not all patients experienced improvement, so it may not work the same for everyone.
providing hope to canadians with rett syndrome
according to the daybue website and the results of the study, canadians who can access the treatment may experience many improvements, including more hand control, more purposeful hand use, fewer breath-holding episodes, less frequent night terrors, and increased nonverbal communication.
while the approval just passed, and the drug may not provide the same results for everyone who takes it, having the first treatment for rett syndrome in canadian pharmacies could potentially make living with rett syndrome more manageable.
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“health canada’s authorization 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,” said catherine owen adams, acadia ceo catherine, in a press release. “we look forward to bringing daybue to canadian patients with rett syndrome as soon as possible.”
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