Acadia Pharmaceuticals Announces Health Canada Approval of DAYBUE™ (trofinetide) for the Treatment of Rett Syndrome
Acadia Pharmaceuticals announced that Health Canada has approved DAYBUE™ (trofinetide) for the treatment of Rett syndrome in patients two years and older. This marks the first and only therapy approved in Canada for this rare neurodevelopmental disorder, which affects an estimated 600 to 900 patients in the country.
The approval was based on positive results from the Phase 3 LAVENDER™ study, which showed statistically significant improvements in the co-primary endpoints: the Rett Syndrome Behaviour Questionnaire (RSBQ) total score and the Clinical Global Impression-Improvement (CGI-I) scale score at week 12.
Rett syndrome primarily affects females and is characterized by regression following normal development in the first two years of life. Symptoms include loss of communication skills, purposeful hand use, and gait abnormalities. The approval of DAYBUE represents a significant milestone for patients and their families, offering a specific therapy for a condition that previously only had symptomatic management options.
Acadia Pharmaceuticals ha annunciato che Health Canada ha approvato DAYBUE™ (trofinetide) per il trattamento della sindrome di Rett nei pazienti di età pari o superiore ai due anni. Questo segna la prima e unica terapia approvata in Canada per questo raro disturbo neuroevolutivo, che colpisce circa 600-900 pazienti nel paese.
L'approvazione si basa su risultati positivi dello studio di Fase 3 LAVENDER™, che ha mostrato miglioramenti statisticamente significativi nei criteri co-principali: il punteggio totale del Rett Syndrome Behaviour Questionnaire (RSBQ) e il punteggio della Clinical Global Impression-Improvement (CGI-I) alla settimana 12.
La sindrome di Rett colpisce principalmente le femmine ed è caratterizzata da una regressione dopo un normale sviluppo nei primi due anni di vita. I sintomi includono perdita delle abilità comunicative, uso intenzionale delle mani e anomalie della deambulazione. L'approvazione di DAYBUE rappresenta un traguardo significativo per i pazienti e le loro famiglie, offrendo una terapia specifica per una condizione che in precedenza aveva solo opzioni di gestione sintomatica.
Acadia Pharmaceuticals anunció que Health Canada ha aprobado DAYBUE™ (trofinetide) para el tratamiento del síndrome de Rett en pacientes de dos años o más. Esto marca la primera y única terapia aprobada en Canadá para este raro trastorno del neurodesarrollo, que afecta a aproximadamente 600 a 900 pacientes en el país.
La aprobación se basó en resultados positivos del estudio de Fase 3 LAVENDER™, que mostró mejoras estadísticamente significativas en los criterios co-principales: la puntuación total del cuestionario de comportamiento del síndrome de Rett (RSBQ) y la puntuación de la Clinical Global Impression-Improvement (CGI-I) en la semana 12.
El síndrome de Rett afecta principalmente a las mujeres y se caracteriza por una regresión tras el desarrollo normal en los primeros dos años de vida. Los síntomas incluyen la pérdida de habilidades de comunicación, uso intencional de las manos y anormalidades en la marcha. La aprobación de DAYBUE representa un hito significativo para los pacientes y sus familias, ofreciendo una terapia específica para una condición que anteriormente solo contaba con opciones de manejo sintomático.
Acadia Pharmaceuticals는 Health Canada가 DAYBUE™ (trofinetide)를 레트 증후군 치료제로 승인했다고 발표했습니다. 이는 캐나다에서 승인된 첫 번째이자 유일한 치료제로, 이 드문 신경 발달 장애는 캐나다에서 약 600~900명의 환자에게 영향을 미칩니다.
이번 승인은 3상 LAVENDER™ 연구의 긍정적인 결과에 기반하며, 여기서 레트 증후군 행동 질문지(RSBQ) 총점과 임상 전반적 인상-개선(CGI-I) 척도 점수가 12주 차에 통계적으로 유의미한 개선을 보였습니다.
레트 증후군은 주로 여성에게 영향을 미치며, 생후 2년 동안 정상적인 발달 후 퇴행을 특징으로 합니다. 증상으로는 의사소통 능력 상실, 목적 있는 손 사용 및 보행 이상이 포함됩니다. DAYBUE의 승인은 환자와 그 가족에게 중요한 이정표가 되어, 이전에 단지 증상 관리 옵션만 있었던 질환에 대한 특정 치료를 제공합니다.
Acadia Pharmaceuticals a annoncé que Santé Canada a approuvé DAYBUE™ (trofinetide) pour le traitement du syndrome de Rett chez les patients âgés de deux ans et plus. Cela marque la première et unique thérapie approuvée au Canada pour ce trouble neurodéveloppemental rare, qui touche environ 600 à 900 patients dans le pays.
Cette approbation est basée sur des résultats positifs de l'étude de phase 3 LAVENDER™, qui a montré des améliorations statistiquement significatives dans les critères co-principaux : le score total du questionnaire de comportement du syndrome de Rett (RSBQ) et le score de l'échelle d'impression globale clinique-amélioration (CGI-I) à la semaine 12.
Le syndrome de Rett affecte principalement les femmes et se caractérise par une régression après un développement normal au cours des deux premières années de la vie. Les symptômes incluent la perte des compétences en communication, l'utilisation intentionnelle des mains et des anomalies de la démarche. L'approbation de DAYBUE représente une étape importante pour les patients et leurs familles, offrant une thérapie spécifique pour une condition qui n'avait auparavant que des options de gestion symptomatique.
Acadia Pharmaceuticals hat bekannt gegeben, dass Health Canada DAYBUE™ (trofinetide) zur Behandlung des Rett-Syndroms bei Patienten ab zwei Jahren genehmigt hat. Dies ist die erste und einzige Therapie, die in Kanada genehmigt wurde für diese seltene neurodevelopmentale Störung, die schätzungsweise 600 bis 900 Patienten im Land betrifft.
Die Genehmigung basierte auf positiven Ergebnissen der Phase-3-Studie LAVENDER™, die statistisch signifikante Verbesserungen der primären Endpunkte zeigte: den Gesamtwert des Rett Syndrome Behaviour Questionnaire (RSBQ) und den Wert der Clinical Global Impression-Improvement (CGI-I) Skala in Woche 12.
Das Rett-Syndrom betrifft hauptsächlich Frauen und ist gekennzeichnet durch eine Regression nach normalem Entwicklung in den ersten zwei Lebensjahren. Zu den Symptomen gehören der Verlust von Kommunikationsfähigkeiten, gezielte Handnutzung und Gangabweichungen. Die Genehmigung von DAYBUE stellt einen bedeutenden Meilenstein für Patienten und deren Familien dar und bietet eine spezifische Therapie für einen Zustand, der zuvor nur symptomatische Behandlungsmöglichkeiten hatte.
- First and only therapy approved in Canada for Rett syndrome
- Positive results from Phase 3 LAVENDER™ study showing statistically significant improvements
- Addresses an unmet medical need for a rare disorder affecting 600-900 patients in Canada
- Expands Acadia's market reach and potential revenue stream
- patient population may restrict revenue potential
- Mechanism of action for therapeutic effects is unknown
Insights
Health Canada's approval of DAYBUE™ (trofinetide) for Rett syndrome is a significant breakthrough in the treatment of this rare neurodevelopmental disorder. This marks the first and only approved therapy for Rett syndrome in Canada, addressing an unmet medical need for an estimated 600 to 900 patients in the country.
The approval is based on positive results from the Phase 3 LAVENDER™ study, which demonstrated statistically significant improvements in both caregiver-assessed and clinician-assessed measures. Key findings include:
- Improvement in Rett Syndrome Behaviour Questionnaire (RSBQ) total score
- Positive changes in Clinical Global Impression-Improvement (CGI-I) scale score
- Enhanced Communication and Symbolic Behavior Scales Development Profile™ Infant-Toddler Checklist – Social Composite Score
This approval could potentially transform the treatment landscape for Rett syndrome in Canada, moving beyond symptomatic management to a targeted therapy. The broad indication for patients 2 years and older allows for early intervention, which may be important in managing this progressive disorder.
The Health Canada approval of DAYBUE™ represents a significant market opportunity for Acadia Pharmaceuticals (NASDAQ: ACAD). As the first and only approved treatment for Rett syndrome in Canada, DAYBUE™ has a potential monopoly in this niche market.
Key financial implications include:
- Market Exclusivity: As the sole approved therapy, Acadia can potentially command premium pricing.
- Revenue Potential: With an estimated 600-900 patients in Canada, even a modest penetration could translate to meaningful revenue growth.
- Global Expansion: This approval strengthens Acadia's position in the global rare disease market, potentially paving the way for approvals in other jurisdictions.
- Diversification: DAYBUE™ adds to Acadia's product portfolio, reducing reliance on its Parkinson's disease psychosis drug.
Investors should monitor the drug's launch, uptake rates and potential inclusion in public and private drug plans, as these factors will significantly impact the revenue trajectory and Acadia's financial performance in the coming quarters.
-- DAYBUE is the first and only therapy approved in
Rett syndrome is a rare, complex, neurodevelopmental disorder affecting primarily females, in which patients demonstrate significant regression following apparently normal development in the first two years of life.1,2 In
“Health Canada’s authorization of DAYBUE is a significant milestone for the Rett syndrome community in
“Rett syndrome is a debilitating and complex neurodevelopmental disorder that presents differently across patients and can lead to a range of symptoms throughout a patient’s life,” said Anita Datta, MD, FRCPC, Pediatric Neurologist and Epileptologist, Co-Director of the Rett Program at BC Children’s Hospital, and Clinical Associate Professor at the Faculty of Medicine at UBC. “Until now, treatment options have primarily focused on symptomatic management, as specific therapies for Rett syndrome have not existed.”
The Health Canada filing was supported by positive results from the pivotal Phase 3 LAVENDER™ study evaluating the efficacy and safety of trofinetide versus placebo in 187 girls and young women 5-to-20 years of age with Rett syndrome. The co-primary endpoints were change from baseline in the Rett Syndrome Behaviour Questionnaire (RSBQ) total score, a caregiver assessment, and Clinical Global Impression-Improvement (CGI-I) scale score, clinician perspective, at week 12; both were statistically significant. RSBQ is a 45-item rating scale completed by the caregiver that assesses a range of symptoms of Rett syndrome (breathing, hand movements or stereotypies, repetitive behaviors, night-time behaviors, vocalizations, facial expressions, eye gaze, and mood). The key secondary endpoint measuring the change from baseline to Week 12 in the Communication and Symbolic Behavior Scales Development Profile™ Infant-Toddler Checklist – Social Composite Score (CSBS-DP-IT Social) was also statistically significant when compared to placebo.6
“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,” said Sabrina Millson, President of the Ontario Rett Syndrome Association (O.R.S.A.). “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.”
In
For more information, in
About Rett Syndrome
Rett syndrome is a rare, complex, neurodevelopmental disorder that may occur over four stages and occurs in approximately one of every 10,000 to 15,000 female births worldwide.1,2,8 In
About DAYBUE™ (trofinetide)
Trofinetide is a synthetic analog of the N-terminal tripeptide of insulin-like growth factor 1. The mechanism by which trofinetide exerts therapeutic effects in patients with Rett syndrome is unknown.11
For more information, please refer to the Product Monograph for DAYBUE in
About Acadia Pharmaceuticals Inc.
Acadia is advancing breakthroughs in neuroscience to elevate life. Since our founding we have been working at the forefront of healthcare to bring vital solutions to people who need them most. We developed and commercialized the first and only FDA-approved drug to treat hallucinations and delusions associated with Parkinson’s disease psychosis and the first and only approved drug in
Forward-Looking Statements
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Forward-looking statements include all statements other than statements of historical fact and can be identified by terms such as “intends,” “may,” “will,” “should,” “could,” “would,” “expects,” “plans,” “anticipates,” “believes,” “estimates,” “projects,” “predicts,” “potential,” “hope” and similar expressions (including the negative thereof) intended to identify forward-looking statements. Forward-looking statements contained in this press release, include, but are not limited to, statements about: (i) the efficacy and safety profile of trofinetide for patients with Rett syndrome, (ii) market acceptance in
References
1 Fu et al. Consensus guidelines on managing Rett syndrome across the lifespan. BMJ Paediatrics Open. 2020;4:1-14
2 Kyle SM, Vashi N, Justice MJ. Rett syndrome: a neurological disorder with metabolic components. Open Biol. 2018; 8:170216.
3 Acadia Pharmaceuticals Inc. Data on File.
4 Neul JL, Kaufmann WE, Glaze DG, et al. Rett syndrome: revised diagnostic criteria and nomenclature. Ann Neurol. 2010;68(6):944-950.
5 Daniel C, Tarquinio DO, Hou W, et al. The changing face of survival in Rett syndrome and MECP2-related disorders. Pediatr Neurol. 2015; 53(5): 402-411.
6 Neul JL, Percy AK, Benke TA et al. Trofinetide for the treatment of Rett syndrome: a randomized phase 3 study. Nature Medicine. 2023; 29: 1468–1475
7 DAYBUE Canadian Product Monograph, [October] 2024
8 May DM, Neul JL, Satija A, et al. Real-world clinical management of individuals with Rett syndrome: a physician survey. J of Med Econ. 2023; 26(1):1570–1580
9 Tarquinio DC, Hou W, Neul JL, et al. Age of Diagnosis in Rett Syndrome: Patterns of Recognition Among Diagnosticians and Risk Factors for Late Diagnosis. Pediatric Neurology. 2015;52:585-591.
10 Amir RE, Van den Veyver IB, Wan M, et al. Rett syndrome is caused by mutations in X-linked MECP2, encoding methyl-CpG-binding protein 2. Nat Genet. 1999; 23(2):185-188.
11 Acadia Pharmaceuticals Inc., Data on file.
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Investor Contact:
Acadia Pharmaceuticals Inc.
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Acadia Pharmaceuticals Inc.
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Source: Acadia Pharmaceuticals Inc.
FAQ
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