Crinetics Pharmaceuticals Announces European Medicines Agency (EMA) Validation of Marketing Authorization Application (MAA) and Orphan Drug Designation (ODD) for Paltusotine in Acromegaly
Crinetics Pharmaceuticals (CRNX) announced two major regulatory milestones for paltusotine, its novel once-daily oral treatment for acromegaly. The European Medicines Agency (EMA) has validated the Marketing Authorization Application (MAA) for review and granted Orphan Drug Designation (ODD) on February 27, 2025.
Paltusotine, the first oral selectively-targeted somatostatin receptor type 2 nonpeptide agonist, demonstrated success in two Phase 3 trials, meeting all primary and secondary endpoints. The trials showed statistically significant biochemical control and symptom improvement compared to placebo, with good tolerability.
The MAA submission includes data from 18 clinical trials. This follows the FDA's acceptance of the New Drug Application with a PDUFA date of September 25, 2025. The ODD status provides potential benefits including 10-year market exclusivity in the EU and reduced regulatory fees.
Crinetics Pharmaceuticals (CRNX) ha annunciato due importanti traguardi normativi per paltusotine, il suo nuovo trattamento orale una volta al giorno per l'acromegalia. L'Agenzia Europea dei Medicinali (EMA) ha convalidato la Domanda di Autorizzazione alla Commercializzazione (MAA) per la revisione e ha concesso la Designazione di Farmaco Orfano (ODD) il 27 febbraio 2025.
Paltusotine, il primo agonista non peptidico selettivamente mirato al recettore della somatostatina di tipo 2, ha dimostrato successo in due studi di Fase 3, raggiungendo tutti gli obiettivi primari e secondari. Gli studi hanno mostrato un controllo biochimico statisticamente significativo e un miglioramento dei sintomi rispetto al placebo, con una buona tollerabilità.
La presentazione della MAA include dati provenienti da 18 studi clinici. Questo segue l'accettazione da parte della FDA della Nuova Domanda di Farmaco con una data PDUFA del 25 settembre 2025. Lo stato di ODD offre potenziali vantaggi, tra cui 10 anni di esclusività di mercato nell'UE e riduzione delle tasse regolatorie.
Crinetics Pharmaceuticals (CRNX) anunció dos hitos regulatorios importantes para paltusotine, su nuevo tratamiento oral diario para la acromegalia. La Agencia Europea de Medicamentos (EMA) ha validado la Solicitud de Autorización de Comercialización (MAA) para revisión y ha otorgado la Designación de Medicamento Huérfano (ODD) el 27 de febrero de 2025.
Paltusotine, el primer agonista no péptido selectivamente dirigido al receptor tipo 2 de somatostatina, demostró éxito en dos ensayos de Fase 3, cumpliendo con todos los objetivos primarios y secundarios. Los ensayos mostraron un control bioquímico estadísticamente significativo y una mejora de los síntomas en comparación con el placebo, con buena tolerabilidad.
La presentación de la MAA incluye datos de 18 ensayos clínicos. Esto sigue la aceptación por parte de la FDA de la Nueva Solicitud de Medicamento con una fecha PDUFA del 25 de septiembre de 2025. El estatus de ODD proporciona beneficios potenciales, incluyendo 10 años de exclusividad en el mercado de la UE y tarifas regulatorias reducidas.
Crinetics Pharmaceuticals (CRNX)는 그들의 새로운 하루 한 번 복용하는 구강 치료제인 팔투소틴에 대한 두 가지 주요 규제 이정표를 발표했습니다. 유럽 의약품청 (EMA)는 2025년 2월 27일 마케팅 승인 신청서 (MAA)를 검토를 위해 검증하고, 희귀의약품 지정 (ODD)을 부여했습니다.
팔투소틴은 선택적으로 타겟팅된 비펩타이드 형태의 소마토스타틴 수용체 타입 2의 첫 번째 경구용 작용제로, 두 개의 3상 시험에서 성공을 거두며 모든 주요 및 부차적 목표를 달성했습니다. 이 시험들은 플라세보와 비교하여 통계적으로 유의미한 생화학적 조절 및 증상 개선을 보여주었으며, 좋은 내약성을 보였습니다.
MAA 제출에는 18개의 임상 시험 데이터가 포함되어 있습니다. 이는 FDA가 새로운 약물 신청을 수락한 후 진행된 것으로, 2025년 9월 25일 PDUFA 날짜가 설정되어 있습니다. ODD 상태는 EU에서 10년의 시장 독점 및 규제 수수료 감소를 포함한 잠재적 혜택을 제공합니다.
Crinetics Pharmaceuticals (CRNX) a annoncé deux grandes étapes réglementaires pour le paltusotine, son nouveau traitement oral quotidien pour l'acromégalie. L'Agence Européenne des Médicaments (EMA) a validé la Demande d'Autorisation de Mise sur le Marché (MAA) pour examen et a accordé la Désignation de Médicament Orphelin (ODD) le 27 février 2025.
Le paltusotine, le premier agoniste non peptidique ciblant sélectivement le récepteur de la somatostatine de type 2, a montré son succès dans deux essais de Phase 3, atteignant tous les objectifs primaires et secondaires. Les essais ont montré un contrôle biochimique statistiquement significatif et une amélioration des symptômes par rapport au placebo, avec une bonne tolérance.
La soumission de la MAA comprend des données de 18 essais cliniques. Cela fait suite à l'acceptation par la FDA de la Nouvelle Demande de Médicament avec une date PDUFA du 25 septembre 2025. Le statut ODD offre des avantages potentiels, y compris 10 ans d'exclusivité sur le marché dans l'UE et des frais réglementaires réduits.
Crinetics Pharmaceuticals (CRNX) hat zwei bedeutende regulatorische Meilensteine für Paltusotin, seine neuartige einmal täglich oral einzunehmende Behandlung von Akromegalie, bekannt gegeben. Die Europäische Arzneimittel-Agentur (EMA) hat den Antrag auf Marktzulassung (MAA) zur Prüfung validiert und am 27. Februar 2025 die Orphan Drug Designation (ODD) erteilt.
Paltusotin, der erste oral einnehmbare selektiv zielgerichtete nicht-peptidische Agonist des Somatostatin-Rezeptors vom Typ 2, zeigte in zwei Phase-3-Studien Erfolge und erreichte alle primären und sekundären Endpunkte. Die Studien zeigten eine statistisch signifikante biochemische Kontrolle und Symptomverbesserung im Vergleich zu Placebo, mit guter Verträglichkeit.
Die MAA-Einreichung umfasst Daten aus 18 klinischen Studien. Dies folgt der Annahme des New Drug Application durch die FDA mit einem PDUFA-Datum am 25. September 2025. Der ODD-Status bietet potenzielle Vorteile, darunter 10 Jahre Marktexklusivität in der EU und reduzierte regulatorische Gebühren.
- Successful Phase 3 trials meeting all primary and secondary endpoints
- EMA validation of Marketing Authorization Application (MAA)
- Granted Orphan Drug Designation providing 10-year market exclusivity potential
- FDA NDA acceptance with PDUFA date set
- Strong clinical data from 18 trials supporting regulatory submissions
- None.
Insights
The EMA's validation of Crinetics' MAA for paltusotine represents a significant regulatory milestone in their European approval pathway. This validation confirms the application is complete and formally begins the scientific review process by the CHMP. Combined with the Orphan Drug Designation (ODD), Crinetics has secured two crucial regulatory advantages in Europe.
The ODD provides substantial benefits beyond regulatory fee reductions - most notably the potential for 10 years of market exclusivity upon approval, which would protect paltusotine from similar competitors regardless of patent status. This exclusivity period represents significant commercial value for a rare disease therapy.
The regulatory package appears robust, with data from 18 clinical trials including two successful Phase 3 studies meeting all primary and secondary endpoints. This comprehensive clinical program demonstrates Crinetics' thorough development approach and increases probability of approval. The parallel US regulatory review with a September 2025 PDUFA date suggests potential near-simultaneous approvals in major markets, creating operational efficiencies for global commercialization.
Paltusotine's position as the first oral, daily somatostatin receptor type 2 agonist gives it a potential competitive advantage over existing injectable treatments, which could drive adoption if efficacy and safety profiles prove comparable or superior. The regulatory progress across both FDA and EMA jurisdiction indicates Crinetics has built a harmonized global development program - a sophisticated approach that maximizes efficiency in bringing this treatment to market worldwide.
Acromegaly represents a classic rare disease opportunity with significant unmet need. As a progressive endocrine disorder affecting roughly 3-4 people per 100,000, it fits squarely in the orphan disease category while providing sufficient patient population to support a commercially viable product.
Paltusotine's differentiated profile as the first once-daily oral treatment option addresses a critical gap in the current treatment landscape. Existing somatostatin analog therapies typically require monthly intramuscular injections, creating burden for patients requiring lifelong treatment. The oral route potentially improves adherence and quality of life for a chronic condition requiring consistent biochemical control.
The statistically significant results in both biochemical control and patient-reported symptom improvements suggest paltusotine could deliver meaningful clinical benefits matching or exceeding current standards of care. For rare diseases, demonstrating symptomatic improvement alongside biomarker control substantially strengthens the value proposition for payers.
The ODD designation reinforces the unmet need recognition by regulatory authorities and provides Crinetics additional leverage in market access negotiations. Crinetics' dual-track US/European regulatory strategy positions them well for coordinated global commercialization, critical for maximizing return on investment in rare disease spaces where each territory contributes meaningful revenue.
With both regulatory submissions accepted, Crinetics must now shift focus toward launch preparation, including market access strategy, pricing considerations, and patient identification initiatives. The company's advancement from development-stage to commercial-stage represents a fundamental transition that will reshape its valuation framework toward revenue-based metrics rather than purely clinical milestone achievements.
SAN DIEGO, March 27, 2025 (GLOBE NEWSWIRE) -- Crinetics Pharmaceuticals, Inc. (Nasdaq: CRNX) today announced that the European Medicines Agency (EMA) has validated the Marketing Authorization Application (MAA) for paltusotine, the first once-daily, oral, selectively-targeted somatostatin receptor type 2 nonpeptide agonist, for the proposed treatment and long-term maintenance therapy of acromegaly, a serious, rare and progressive endocrine disorder characterized by consistently elevated levels of growth hormone. The MAA will now be reviewed by the Committee for Medicinal Products for Human Use (CHMP). Additionally, the EMA on February 27, 2025 granted paltusotine Orphan Drug Designation (ODD) for the treatment of acromegaly.
“The submission of our MAA for paltusotine to the EMA is a significant milestone and underscores our commitment to making our therapies accessible worldwide,” said Scott Struthers, Ph.D., Founder and Chief Executive Officer of Crinetics. “As we focus on our anticipated U.S. launch, this submission demonstrates our global capabilities and sets the stage for the continued growth of Crinetics.”
“Orphan designation from the EMA further highlights the unmet need in acromegaly and the potential benefit paltusotine can bring for patients,” said Dana Pizzuti, MD, Chief Medical and Development Officer of Crinetics. “We look forward to continued collaboration with the European regulatory authorities throughout their review process.”
The MAA submission is supported by data from 18 clinical trials, including two Phase 3 trials that evaluated paltusotine for the treatment of acromegaly in medically untreated and treated patients. All primary and secondary endpoints were met in both Phase 3 studies. Treatment with paltusotine was well-tolerated and resulted in statistically significant biochemical control and patient reported symptom control compared to placebo.
MAA validation follows the acceptance by the U.S. Food and Drug Administration (FDA) of the New Drug Application (NDA) for paltusotine for the treatment and long-term maintenance of acromegaly. The FDA assigned a Prescription Drug User Fee Act (PDUFA) target action date of September 25, 2025.
The EMA grants orphan designation to medicines intended for the treatment, prevention, or diagnosis of life-threatening or chronically debilitating diseases affecting no more than 5 in 10,000 people in the European Union, among other criteria. The medicine must also provide significant benefit to those affected by the condition. Orphan designation provides certain benefits, including reduction in regulatory fees and potential for 10 years of market exclusivity.
About Paltusotine
Crinetics’ lead development candidate, paltusotine, is the first investigational once-daily, oral, selectively-targeted somatostatin receptor type 2 (SST2) nonpeptide agonist that has completed Phase 3 clinical development for acromegaly and is initiating Phase 3 clinical development for carcinoid syndrome associated with neuroendocrine tumors. It was designed to be a once daily oral option for the control of acromegaly and the symptoms related to carcinoid syndrome. In Phase 3 studies, once-daily, oral paltusotine maintained IGF-1 levels and symptom control in patients with acromegaly who were switched from monthly injectable medications (PATHFNDR-1) and rapidly decreased IGF-1 levels and symptom burden in medically untreated acromegaly patients (PATHFNDR-2). IGF-1 is the primary biomarker endocrinologists use to manage acromegaly patients. Results from a Phase 2 study in carcinoid syndrome demonstrated rapid and sustained reductions in flushing episodes and bowel movement frequency, which are the most common symptoms of carcinoid syndrome. Crinetics is preparing to initiate a global Phase 3 trial for control of symptoms associated with carcinoid syndrome in patients with neuroendocrine tumors.
About Acromegaly
Acromegaly is a serious rare disease generally caused by a benign pituitary adenoma (tumor) that secretes excess growth hormone (GH). Excess GH secretion causes excess secretion of insulin-like growth factor-1 (IGF-1) from the liver. Prolonged exposure to increased levels of IGF-1 and GH leads to progressive and serious systemic complications, often resulting in bone, joint, cardiovascular, metabolic, cerebrovascular, or respiratory disease. Acromegaly symptoms include headache, joint aches, fatigue, sleep apnea, severe sweating, hyperhidrosis/oily skin, bone and cartilage overgrowth, abnormal growth of hands and feet, enlargement of heart, liver and other organs and alteration of facial features. Uncontrolled acromegaly results in increased mortality and has a debilitating impact on daily functioning and quality of life.
Monthly depot injections of peptide somatostatin receptor ligands are the most common pharmacologic treatment for people suffering with acromegaly. However, these depots typically require many months to achieve the correct dose level. People suffering with acromegaly often experience a return of symptoms towards the end of the monthly injection cycle and many must adjust their injection frequency to more often than monthly. Further, these depots are difficult to administer and employ large gauge needles that are commonly associated with pain, injection site reactions and an increased burden on the lives of patients.
About Crinetics Pharmaceuticals
Crinetics Pharmaceuticals is a clinical stage pharmaceutical company focused on the discovery, development, and commercialization of novel therapeutics for endocrine diseases and endocrine-related tumors. Crinetics’ lead development candidate, paltusotine, is the first investigational once-daily, oral, selective somatostatin receptor type 2 (SST2) nonpeptide agonist that is in clinical development for acromegaly and carcinoid syndrome associated with neuroendocrine tumors. Atumelnant is currently in development for congenital adrenal hyperplasia and ACTH-dependent Cushing’s syndrome. All of the company’s drug candidates are orally delivered, small molecule, new chemical entities resulting from in-house drug discovery efforts, including additional discovery programs addressing a variety of endocrine conditions such as hyperparathyroidism, polycystic kidney disease, Graves’ disease (including thyroid eye disease), diabetes, obesity and GPCR-targeted oncology indications.
Forward-Looking Statements
This press release contains forward-looking statements within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. All statements other than statements of historical facts contained in this press release are forward-looking statements, including statements about the expected MAA review process, the potential global direction and growth of Crinetics, the plans and timelines for the commercial launch of paltusotine for acromegaly, if approved, the plans for initiating a Phase 3 program of paltusotine for carcinoid syndrome, and the potential of our other research, discovery, and clinical trial programs. In some cases, you can identify forward-looking statements by terms such as “may,” “will,” “should,” “expect,” “plan,” “anticipate,” “could,” “intend,” “target,” “project,” “contemplates,” “believes,” “estimates,” “predicts,” “potential,” “upcoming” or “continue” or the negative of these terms or other similar expressions. These forward-looking statements speak only as of the date of this press release and are subject to a number of risks, uncertainties and assumptions, including, without limitation, potential delays in the commencement, enrollment and completion of clinical trials and the reporting of data therefrom; clinical studies and preclinical studies may not proceed at the time or in the manner expected, or at all; regulatory developments in the United States and foreign countries; the timing and outcome of research, development and regulatory review is uncertain, and Crinetics’ drug candidates may not advance in development or be approved for marketing; and the other risks and uncertainties described in the company’s periodic filings with the Securities and Exchange Commission (SEC). The events and circumstances reflected in the company’s forward-looking statements may not be achieved or occur and actual results could differ materially from those projected in the forward-looking statements. Additional information on risks facing Crinetics can be found under the heading “Risk Factors” in Crinetics’ periodic filings with the SEC, including its annual report on Form 10-K for the year ended December 31, 2024. You are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof. Except as required by applicable law, Crinetics does not plan to publicly update or revise any forward-looking statements contained herein, whether as a result of any new information, future events, changed circumstances or otherwise.
Investors:
Gayathri Diwakar
Head of Investor Relations
gdiwakar@crinetics.com
(858) 345-6340
Media:
Natalie Badillo
Head of Corporate Communications
nbadillo@crinetics.com
(858) 345-6075
FAQ
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