CHMP recommends approval of Oczyesa® for treatment of acromegaly in the EU
Camurus (NASDAQ STO: CAMX) has received a positive recommendation from the European Medicines Agency's CHMP for market authorization of Oczyesa®, a monthly octreotide depot treatment for adult acromegaly patients. The recommendation is supported by comprehensive clinical data from seven studies, including two Phase 3 trials.
The ACROINNOVA 1 study showed Oczyesa achieved higher normalization rates of insulin growth-factor-1 (IGF-1) levels versus placebo. ACROINNOVA 2 confirmed sustained IGF-1 improvements and demonstrated enhanced symptoms, quality of life, and treatment satisfaction over 52 weeks compared to standard care. Common side effects included gastrointestinal, nervous system, and hepatobiliary disorders.
A final European Commission decision on marketing authorization is expected in mid-2025.
Camurus (NASDAQ STO: CAMX) ha ricevuto una raccomandazione positiva dal CHMP dell'Agenzia Europea per i Medicinali per l'autorizzazione al commercio di Oczyesa®, un trattamento depot mensile a base di octreotide per pazienti adulti con acromegalia. La raccomandazione si basa su dati clinici completi provenienti da sette studi, inclusi due studi di Fase 3.
Lo studio ACROINNOVA 1 ha evidenziato che Oczyesa ha raggiunto tassi più elevati di normalizzazione dei livelli di fattore di crescita insulino-simile 1 (IGF-1) rispetto al placebo. ACROINNOVA 2 ha confermato miglioramenti sostenuti dell’IGF-1 e ha dimostrato un miglioramento dei sintomi, della qualità della vita e della soddisfazione al trattamento nell’arco di 52 settimane rispetto alla terapia standard. Gli effetti collaterali più comuni includevano disturbi gastrointestinali, del sistema nervoso e epatobiliare.
La decisione finale della Commissione Europea sull’autorizzazione alla commercializzazione è prevista per metà 2025.
Camurus (NASDAQ STO: CAMX) ha recibido una recomendación positiva del CHMP de la Agencia Europea de Medicamentos para la autorización de comercialización de Oczyesa®, un tratamiento mensual de depósito de octreótido para pacientes adultos con acromegalia. La recomendación está respaldada por datos clínicos exhaustivos de siete estudios, incluidos dos ensayos de Fase 3.
El estudio ACROINNOVA 1 mostró que Oczyesa logró tasas más altas de normalización de los niveles de factor de crecimiento similar a la insulina 1 (IGF-1) en comparación con el placebo. ACROINNOVA 2 confirmó mejoras sostenidas en IGF-1 y demostró mejoras en los síntomas, la calidad de vida y la satisfacción con el tratamiento durante 52 semanas en comparación con la atención estándar. Los efectos secundarios comunes incluyeron trastornos gastrointestinales, del sistema nervioso y hepatobiliares.
Se espera una decisión final de la Comisión Europea sobre la autorización de comercialización para mediados de 2025.
Camurus (NASDAQ STO: CAMX)는 성인 말단비대증 환자를 위한 월 1회 옥트레오타이드 주입제인 Oczyesa®의 유럽 의약품청 CHMP로부터 시장 허가에 대한 긍정적인 권고를 받았습니다. 이 권고는 2건의 3상 임상시험을 포함한 7건의 광범위한 임상 데이터에 기반합니다.
ACROINNOVA 1 연구에서는 Oczyesa가 위약 대비 인슐린 유사 성장 인자 1(IGF-1) 수치 정상화 비율이 더 높게 나타났습니다. ACROINNOVA 2 연구는 IGF-1 개선이 지속됨을 확인했으며, 52주 동안 표준 치료 대비 증상, 삶의 질, 치료 만족도 향상을 입증했습니다. 흔한 부작용으로는 위장관, 신경계, 간담도계 장애가 포함되었습니다.
시장 허가에 대한 유럽 위원회의 최종 결정은 2025년 중반에 예상됩니다.
Camurus (NASDAQ STO: CAMX) a reçu une recommandation positive du CHMP de l'Agence européenne des médicaments pour l'autorisation de mise sur le marché de Oczyesa®, un traitement mensuel en dépôt d'octréotide destiné aux patients adultes atteints d'acromégalie. Cette recommandation est appuyée par des données cliniques complètes issues de sept études, dont deux essais de phase 3.
L'étude ACROINNOVA 1 a montré qu'Oczyesa obtenait des taux de normalisation plus élevés des niveaux de facteur de croissance analogue à l'insuline 1 (IGF-1) par rapport au placebo. ACROINNOVA 2 a confirmé une amélioration soutenue de l'IGF-1 et a démontré une amélioration des symptômes, de la qualité de vie et de la satisfaction du traitement sur 52 semaines par rapport aux soins standards. Les effets secondaires courants comprenaient des troubles gastro-intestinaux, du système nerveux et hépatobiliaires.
Une décision finale de la Commission européenne concernant l'autorisation de mise sur le marché est attendue pour la mi-2025.
Camurus (NASDAQ STO: CAMX) hat eine positive Empfehlung des CHMP der Europäischen Arzneimittel-Agentur für die Marktzulassung von Oczyesa® erhalten, einer monatlichen Depotbehandlung mit Octreotid für erwachsene Akromegalie-Patienten. Die Empfehlung stützt sich auf umfassende klinische Daten aus sieben Studien, darunter zwei Phase-3-Studien.
Die ACROINNOVA 1-Studie zeigte, dass Oczyesa höhere Normalisierungsraten der Insulinähnlichen Wachstumsfaktor-1 (IGF-1)-Spiegel im Vergleich zu Placebo erreichte. ACROINNOVA 2 bestätigte anhaltende IGF-1-Verbesserungen und zeigte über 52 Wochen verbesserte Symptome, Lebensqualität und Behandlungszufriedenheit im Vergleich zur Standardbehandlung. Häufige Nebenwirkungen umfassten gastrointestinale, neurologische und hepatobiliäre Störungen.
Eine endgültige Entscheidung der Europäischen Kommission zur Marktzulassung wird Mitte 2025 erwartet.
- CHMP positive recommendation increases likelihood of EU market approval
- Superior efficacy demonstrated with higher IGF-1 normalization rates vs placebo
- Sustained clinical benefits shown over 52 weeks
- Patient-friendly monthly self-administration option
- Multiple side effects reported including gastrointestinal and nervous system disorders
- Final EU approval still pending
- Market launch not expected until mid-2025
Insights
CHMP's positive opinion for Camurus' Oczyesa significantly advances EU approval process, enhancing their acromegaly treatment portfolio with strong clinical data.
The CHMP's positive recommendation for Oczyesa represents a critical regulatory milestone for Camurus, effectively clearing the penultimate hurdle before full EU market authorization. This recommendation carries substantial weight—historically, the European Commission almost invariably follows CHMP opinions in its final decisions, expected by mid-2025.
What sets this application apart is the robust clinical evidence package supporting it. The dossier includes data from seven clinical studies, with two pivotal Phase 3 trials demonstrating clear efficacy. ACROINNOVA 1 showed statistically significant improvements in normalizing insulin growth-factor-1 levels (the key biomarker for acromegaly control) versus placebo, while ACROINNOVA 2 confirmed sustained efficacy over 52 weeks with meaningful improvements in patient-reported outcomes.
The innovation here lies in the drug delivery mechanism—a subcutaneous depot formulation allowing once-monthly self-administration via autoinjector. This represents a potential advancement in the acromegaly treatment landscape, where current somatostatin analogues often require healthcare professional administration or more frequent dosing.
The safety profile appears consistent with the octreotide class, with disclosed side effects encompassing gastrointestinal, nervous system, hepatobiliary, and metabolic disorders—expected for this therapeutic class. The maintenance treatment indication specifically targets patients who have already demonstrated response and tolerance to somatostatin analogues, appropriately positioning this therapy within the treatment algorithm.
Oczyesa's EU advancement offers significant clinical benefits for acromegaly patients through improved hormone control, convenient administration, and enhanced quality of life.
From a clinical perspective, Oczyesa addresses several fundamental challenges in acromegaly management. Acromegaly—a rare but serious condition caused by excessive growth hormone production—requires precise biochemical control to prevent significant morbidity and mortality. The ACROINNOVA 1 study results demonstrating superior IGF-1 normalization compared to placebo represents meaningful clinical efficacy, as IGF-1 is the gold standard biomarker for disease control.
The drug's formulation brilliantly solves a persistent treatment adherence problem. Current somatostatin analogs typically require either frequent injections or complex intramuscular administration by healthcare professionals. Oczyesa's once-monthly subcutaneous self-administration via autoinjector represents a genuine quality-of-life advancement, potentially improving treatment compliance while reducing healthcare utilization.
Particularly noteworthy are the 52-week ACROINNOVA 2 results showing improvements in symptoms, quality of life, and treatment satisfaction scores versus standard care. Acromegaly symptoms—including debilitating fatigue, joint pain, and metabolic disturbances—significantly impact daily functioning, so these patient-reported outcomes carry substantial clinical relevance.
The side effect profile aligns with the expected pharmacological action of octreotide, a somatostatin analog that inhibits various hormones beyond growth hormone. The gastrointestinal effects (likely including nausea, abdominal pain, and diarrhea), nervous system effects (likely headache), and potential metabolic alterations are expected class effects that most endocrinologists managing acromegaly are thoroughly familiar with.
"We are pleased with the CHMP's positive recommendation for market authorization for Oczyesa octreotide monthly depot for the treatment of acromegaly", says Fredrik Tiberg, President & CEO at Camurus. "Oczyesa has the potential to advance the standard of care for patients living with acromegaly by enhancing octreotide plasma exposure and enabling easy and convenient once-monthly self-administration by patients using an autoinjector pen."
The CHMP positive opinion is backed by a comprehensive clinical program comprising seven clinical studies, including two Phase 3 studies. The ACROINNOVA 1 study demonstrated that treatment with Oczyesa results in a significantly higher proportion of patients achieving normalized insulin growth-factor-1 (IGF-1) levels compared to placebo. The persistence of mean IGF-1 values and reduction of symptoms were confirmed over 52 weeks in the ACROINNOVA 2 study. Furthermore, the study showed improvements in symptoms, quality of life, and treatment satisfaction scores after 52 weeks of treatment with Oczyesa compared to standard of care (SoC) at study baseline.2,3 The most common side effects included gastrointestinal disorders, nervous system disorders, hepatobiliary disorders, metabolism and nutritional disorders, and injection site reactions.1
A final decision on the marketing authorization of Oczyesa based on the CHMP recommendation is anticipated from the European Commission in mid-2025.
For more information
Fredrik Tiberg, President & CEO
Tel. +46 (0)46 286 46 92
fredrik.tiberg@camurus.com
Fredrik Joabsson, Chief Business Development Officer
Tel. +46 (0)70 776 17 37
ir@camurus.com
About acromegaly
Acromegaly is a rare, progressive disease, typically caused by a tumor of the pituitary gland producing excess growth hormone and stimulating increased insulin growth factor-1 (IGF-1) levels. This results in abnormal growth of bone and tissue, enlarged hands, feet, facial features and inner organs, and symptoms such as fatigue, joint pain, headache, visual field defects, excessive sweating, and paresthesia.4 Inadequate biochemical and symptom control can have detrimental impacts on quality of life and mortality of patients with acromegaly.5,6 The prevalence of acromegaly is estimated to about 60 cases per million.7
About Oczyesa® (CAM2029)
CAM2029 is a ready-to-use, long-acting subcutaneous depot of octreotide under development for the treatment of three chronic and severe disease indications: acromegaly, gastroenteropancreatic neuroendocrine tumors (GEP-NET), and polycystic liver disease (PLD).
The CAM2029 clinical program for acromegaly comprises of seven clinical trials, including four Phase 1 studies, one Phase 2 study, and two Phase 3 studies within the ACROINNOVA clinical program. CAM2029 has demonstrated an approximate five-fold higher bioavailability compared to the currently approved, long-acting, intramuscular (IM) octreotide.8 In the Phase 3 ACROINNOVA program, CAM2029 showed superior biochemical control compared to placebo as well as improvements in symptom control, treatment satisfaction, and quality of life compared to standard-of-care (SoC) at baseline with first-generation somatostatin receptor ligands (SRLs), octreotide and lanreotide. The safety profile of CAM2029 was consistent with SoC with no new findings.2,3
CAM2029 is formulated using Camurus' proprietary FluidCrystal® technology. The product is designed for convenient once-monthly, subcutaneous self-administration using a pen with a hidden, small-gauge needle. The product is stored at room temperature and should not be refrigerated.
CAM2029 has received orphan drug designation for acromegaly (EU) and for polycystic liver disease (EU and US).
About Camurus
Camurus is an international, science-led biopharmaceutical company committed to developing and commercializing innovative, long-acting medicines for improving the lives of patients with severe and chronic diseases. New drug products with best-in-class potential are conceived based on the company's proprietary FluidCrystal® technology and its extensive R&D expertise. The R&D pipeline includes products for the treatment of dependence, pain, cancer, and endocrine diseases. Camurus has operations across
References
- Oczyesa® Summary of Opinion.
- Ferone, D., et al. J Clin Endocrinol Metab. Published 8 October, 2024.
- Press release 15 July, 2024: https://www.camurus.com/media/press-releases/2024/camurus-announces-positive-phase-3-results-from-the-acroinnova-2-study-of-octreotide-sc-depot-cam2029-in-acromegaly-patients/
- Colao A., et al. Acromegaly. Nat Rev Dis Primers. 2019;5(1):20.
- Webb SM, et al. Quality of Life in Acromegaly. Neuroendocrinology. 2016;103(1):106-111.
- Fleseriu M, et al Acromegaly: pathogenesis, diagnosis, and management. Lancet Diabetes Endocrinol. 2022 Nov;10(11):804-826.
- Crisafulli S., et al. Global epidemiology of acromegaly: a systematic review and meta-analysis. Eur J Endocrinology. 2021; 185:251-63.
- Summary of Product Characteristics, Sandostatin LAR 20 mg: https://assets.hpra.ie/products/Human/22656/Licence_PA0896-028-005_03012024152159.pdf
This information was submitted for publication at 4:00 pm CET on 25 April 2025.
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SOURCE Camurus AB
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