Acadia Pharmaceuticals Submits Marketing Authorization Application to the European Medicines Agency for Trofinetide for the Treatment of Rett Syndrome
Acadia Pharmaceuticals (NASDAQ: ACAD) has submitted a Marketing Authorization Application (MAA) to the European Medicines Agency for trofinetide to treat Rett syndrome in patients two years and older. If approved, it would be the first therapy for Rett syndrome in the EU. The drug is already approved in the US and Canada.
The application is supported by positive results from the Phase 3 LAVENDER study of 187 participants. The study met its co-primary endpoints, showing statistically significant improvements in both the Rett Syndrome Behaviour Questionnaire (RSBQ) total score and Clinical Global Impression-Improvement (CGI-I) scale score at week 12. The key secondary endpoint measuring communication and social behavior was also met.
Rett syndrome affects approximately 1 in 10,000-15,000 female births worldwide. It's a rare neurodevelopmental disorder typically caused by a MECP2 gene mutation, characterized by normal early development followed by regression in communication skills and purposeful hand use.
Acadia Pharmaceuticals (NASDAQ: ACAD) ha presentato una domanda di autorizzazione al commercio (MAA) all'Agenzia Europea per i Medicinali per trofinetide, per trattare la sindrome di Rett nei pazienti di età pari o superiore a due anni. Se approvato, sarebbe la prima terapia per la sindrome di Rett nell'UE. Il farmaco è già approvato negli Stati Uniti e in Canada.
La domanda è supportata da risultati positivi dello studio di Fase 3 LAVENDER che ha coinvolto 187 partecipanti. Lo studio ha raggiunto i suoi obiettivi co-primari, mostrando miglioramenti statisticamente significativi sia nel punteggio totale del Questionario sul Comportamento nella Sindrome di Rett (RSBQ) sia nel punteggio della scala di Impressione Clinica-Gli Miglioramenti (CGI-I) alla settimana 12. Anche l'obiettivo secondario chiave che misura la comunicazione e il comportamento sociale è stato raggiunto.
La sindrome di Rett colpisce circa 1 nascita femminile ogni 10.000-15.000 a livello mondiale. È un raro disturbo neuroevolutivo, tipicamente causato da una mutazione del gene MECP2, caratterizzato da uno sviluppo iniziale normale, seguito da una regressione nelle abilità comunicative e nell'uso intenzionale delle mani.
Acadia Pharmaceuticals (NASDAQ: ACAD) ha presentado una Solicitud de Autorización de Comercialización (MAA) a la Agencia Europea de Medicamentos para trofinetide, para tratar el síndrome de Rett en pacientes de dos años o más. Si se aprueba, sería la primera terapia para el síndrome de Rett en la UE. El medicamento ya está aprobado en EE. UU. y Canadá.
La solicitud está respaldada por resultados positivos del estudio de Fase 3 LAVENDER con 187 participantes. El estudio alcanzó sus objetivos co-principales, mostrando mejoras estadísticamente significativas tanto en la puntuación total del Cuestionario de Comportamiento del Síndrome de Rett (RSBQ) como en la puntuación de la Escala de Impresión Clínica - Mejora (CGI-I) a la semana 12. También se alcanzó el objetivo secundario clave que mide la comunicación y el comportamiento social.
El síndrome de Rett afecta aproximadamente a 1 de cada 10.000-15.000 nacimientos femeninos en todo el mundo. Es un trastorno neurodesarrollado raro, típicamente causado por una mutación en el gen MECP2, caracterizado por un desarrollo temprano normal seguido de una regresión en las habilidades de comunicación y el uso intencionado de las manos.
Acadia Pharmaceuticals (NASDAQ: ACAD)는 trofinetide에 대한 의약품 허가 신청서(MAA)를 유럽의약청에 제출했습니다. 이는 2세 이상 환자의 레트 증후군 치료를 위한 것이며, 승인이 이루어질 경우 EU에서 레트 증후군에 대한 첫 번째 치료제가 됩니다. 이 약물은 이미 미국과 캐나다에서 승인되었습니다.
이 신청서는 187명의 참가자가 포함된 3상 LAVENDER 연구의 긍정적인 결과를 뒷받침합니다. 이 연구는 레트 증후군 행동 질문지(RSBQ) 총 점수 및 임상 전반적 인상 - 개선(CGI-I) 척도의 12주 차 점수에서 통계적으로 유의미한 개선을 보여주는 공동 주요 목표를 충족했습니다. 의사소통 및 사회적 행동을 측정하는 주요 2차 목표도 달성되었습니다.
레트 증후군은 전 세계적으로 약 1만-1만5000명의 여성 출생 중 1명에게 영향을 미칩니다. 이는 MECP2 유전자 변이에 의해 발생하는 드문 신경 발달 장애로, 정상적인 초기 발달을 나타내다가 의사소통 능력 및 목적 있는 손 사용의 퇴행을 특징으로 합니다.
Acadia Pharmaceuticals (NASDAQ: ACAD) a soumis une Demande d'Autorisation de Mise sur le Marché (MAA) à l'Agence Européenne des Médicaments pour trofinetide afin de traiter le syndrome de Rett chez des patients âgés de deux ans et plus. Si elle est approuvée, ce serait la première thérapie pour le syndrome de Rett dans l'UE. Le médicament est déjà approuvé aux États-Unis et au Canada.
La demande est soutenue par des résultats positifs de l'étude de Phase 3 LAVENDER portant sur 187 participants. L'étude a atteint ses objectifs co-primaires, montrant des améliorations statistiquement significatives tant dans le score total du Questionnaire de Comportement du Syndrome de Rett (RSBQ) que dans le score de l'Échelle d'Improvement Clinique Globale (CGI-I) à la semaine 12. L'objectif secondaire clé mesurant la communication et le comportement social a également été atteint.
Le syndrome de Rett touche environ 1 naissance féminine sur 10.000-15.000 dans le monde. C'est un trouble neurodéveloppemental rare, généralement causé par une mutation du gène MECP2, caractérisé par un développement précoce normal suivi d'une régression des compétences en communication et de l'utilisation intentionnelle des mains.
Acadia Pharmaceuticals (NASDAQ: ACAD) hat einen Antrag auf Zulassung (MAA) bei der Europäischen Arzneimittelagentur für trofinetide zur Behandlung des Rett-Syndroms bei Patienten ab zwei Jahren eingereicht. Bei Genehmigung wäre es die erste Therapie für das Rett-Syndrom in der EU. Das Medikament ist bereits in den USA und Kanada zugelassen.
Der Antrag wird durch positive Ergebnisse der Phase-3-Studie LAVENDER mit 187 Teilnehmern unterstützt. Die Studie erfüllte ihre ko-primären Endpunkte und zeigte statistisch signifikante Verbesserungen sowohl im Gesamtscore des Rett-Syndrom-Verhaltensfragebogens (RSBQ) als auch im Score der Skala Klinische Gesamteinschätzung - Verbesserung (CGI-I) in der Woche 12. Auch der wichtige sekündäre Endpunkt zur Messung von Kommunikation und sozialem Verhalten wurde erreicht.
Das Rett-Syndrom betrifft weltweit etwa 1 von 10.000-15.000 weiblichen Geburten. Es ist eine seltene neurodevelopmentale Störung, die typischerweise durch eine Mutation des MECP2-Gens verursacht wird und sich durch eine normale frühe Entwicklung auszeichnet, gefolgt von einem Rückgang der Kommunikationsfähigkeiten und des gezielten Einsatzes der Hände.
- First potential approved therapy for Rett syndrome in the EU market
- Successful Phase 3 LAVENDER trial meeting all primary and key secondary endpoints
- Already approved in US and Canada markets
- Unknown mechanism of action for trofinetide
- market size due to rare disease status (1 in 10,000-15,000 female births)
Insights
The EMA submission for trofinetide represents a major regulatory milestone for Acadia Pharmaceuticals, potentially opening up significant market opportunities in the EU. The application is backed by compelling Phase 3 LAVENDER study data, which demonstrated statistically significant improvements in both primary endpoints. With trofinetide already approved in the US and Canada, EU approval would establish a strong global presence for this first-in-class Rett syndrome treatment.
The EU market represents approximately 12,000-18,000 Rett syndrome patients, based on the cited prevalence of 1 in 10,000-15,000 female births. Given the current lack of approved treatments in the EU and the complex, lifelong nature of Rett syndrome requiring constant care, successful approval could translate into substantial recurring revenue streams.
The positive Phase 3 data, particularly the improvements in the RSBQ total score and CGI-I scale, strongly support the likelihood of EMA approval. The standard EMA review timeline suggests a potential approval decision within 12-14 months, setting up a possible commercial launch in early 2026.
This regulatory submission carries strategic importance for Acadia's international expansion plans. The European Rett syndrome market presents a significant untapped opportunity, as there are currently no approved treatments in the region. Success in the EU would strengthen Acadia's position as the global leader in Rett syndrome therapeutics.
The potential approval would create a first-mover advantage in the EU market, allowing Acadia to establish strong relationships with healthcare providers and patient advocacy groups before any competition emerges. The company's existing experience with trofinetide commercialization in North America provides valuable insights for a successful EU launch.
From a market perspective, EU approval would diversify Acadia's revenue streams geographically and reduce dependence on the US market. Given the high unmet need and the chronic nature of Rett syndrome, pricing power in the EU market could be substantial, though subject to individual country reimbursement negotiations.
-- Filing marks company’s first Marketing Authorization Application in
“This application underscores our continued dedication to the Rett community, and our commitment to making a meaningful impact for people living with Rett syndrome and their caregivers in the EU, who currently have no approved treatment options specifically for this condition,” said Catherine Owen Adams, Acadia’s Chief Executive Officer. “We look forward to working with the EMA to address this unmet need in the hopes of potentially bringing this therapy to families who are impacted by Rett syndrome.”
The MAA is 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 five 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. The 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.1
“Rett syndrome is a severe and complex neurodevelopmental disorder that manifests differently in each affected person, leading to a wide range of symptoms over the course of their lives,” said Becky Jenner, President of Rett Syndrome Europe. “The Rett syndrome community 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.2-4 A child with Rett syndrome exhibits an early period of apparently normal development until six to 18 months, when their skills seem to slow down or stagnate. This is typically followed by a duration of regression when the child loses acquired communication skills and purposeful hand use. The child may then experience a plateau period in which they show mild recovery in cognitive interests, but body movements remain severely diminished. As they age, those living with Rett may continue to experience a stage of motor deterioration which can last the rest of the patient’s life.3 Rett syndrome is typically caused by a genetic mutation on the MECP2 gene.5 In preclinical studies, deficiency in MeCP2 function is thought to lead to impairment in synaptic communication, and the deficits in synaptic function may be associated with Rett manifestations.5-7
Symptoms of Rett syndrome may also include development of hand stereotypies, such as hand wringing and clapping, and gait abnormalities.8 Most people living with Rett syndrome typically live into adulthood and require round-the-clock care.2,9
About Trofinetide
Trofinetide is a synthetic analog of the N-terminal tripeptide of insulin-like growth factor 1 (IGF-1). The mechanism by which trofinetide exerts therapeutic effects in patients with Rett syndrome is unknown. In animal studies, trofinetide has been shown to increase branching of dendrites and synaptic plasticity signals.10
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,” “can,” “could,” “would,” “expects,” “plans,” “anticipates,” “believes,” “estimates,” “projects,” “predicts,” “potential” 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 potential approval of trofinetide as the first option in the European Union for the treatment of Rett syndrome, (ii) the efficacy and safety profile of trofinetide for patients with Rett syndrome, (iii) market acceptance in EU, including the importance of trofinetide for the treatment of Rett syndrome, if approved, for Rett patients or families of patients with Rett syndrome and (iv) the ability for trofinetide to address the unmet medical needs of Europeans living with Rett syndrome. Forward-looking statements are subject to known and unknown risks, uncertainties, assumptions, and other factors that may cause our actual results, performance or achievements to differ materially and adversely from those anticipated or implied by our forward-looking statements. Such risks, uncertainties, assumptions and other factors include, but are not limited to: the ability for trofinetide to deliver efficacious and safe results to patients, if approved, our dependency on the continued successful commercialization of DAYBUE™ in
References
1 Neul JL, Percy AK, Benke TA et al. Trofinetide for the treatment of Rett syndrome: a randomized phase 3 study. Nat Med. 2023; 29: 1468–1475
2 Fu C, Armstrong D, Marsh E, et al. Consensus guidelines on managing Rett syndrome across the lifespan. BMJ Paediatrics Open. 2020; 4: 1-14.
3 Kyle SM, Vashi N, Justice MJ. Rett syndrome: a neurological disorder with metabolic components. Open Biol. 2018; 8: 170216.
4 May DM, Neul JL, Satija A, et al. Real-world clinical management of individuals with Rett syndrome: a physician survey. J of Med Econ. 26(1), 1570–1580.
5 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.
6 Fukuda T, Itoh M, Ichikawa T, et al. Delayed maturation of neuronal architecture and synaptogenesis in cerebral cortex of Mecp2-deficient mice. J Neuropathol Exp Neurol. 2005; 64(6): 537-544.
7 Asaka Y, Jugloff DG, Zhang L, et al. Hippocampal synaptic plasticity is impaired in the Mecp2-null mouse model of Rett syndrome. Neurobiol Dis. 2006; 21(1): 217-227.
8 Neul JL, Kaufmann WE, Glaze DG, et al. Rett syndrome: revised diagnostic criteria and nomenclature. Ann Neurol. 2010; 68(6): 944-950.
9 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.
10 Acadia Pharmaceuticals Inc., Data on file.
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