Eton Pharmaceuticals Announces Submission of NDA for ET-600 (Desmopressin Oral Solution)
Eton Pharmaceuticals (ETON) has submitted a New Drug Application (NDA) to the FDA for ET-600, a proprietary oral solution of desmopressin designed to treat central diabetes insipidus. The company anticipates a 10-month FDA review period, targeting potential approval and launch in Q1 2026.
ET-600 would be the first FDA-approved oral liquid formulation of desmopressin, specifically developed to provide precise, titratable doses for pediatric patients. The drug has demonstrated pharmacokinetic equivalence to the FDA-approved reference product in a bioequivalence study involving 75 subjects.
Key highlights:
- Patent protection extends through 2044
- Target market: approximately 3,000 pediatric patients with central diabetes insipidus in the US
- Pre-launch commercial activities are already in progress
Eton Pharmaceuticals (ETON) ha presentato una New Drug Application (NDA) alla FDA per ET-600, una soluzione orale proprietaria di desmopressina progettata per trattare il diabete insipido centrale. L'azienda prevede un periodo di revisione della FDA di 10 mesi, puntando a un'eventuale approvazione e lancio nel primo trimestre del 2026.
ET-600 sarebbe la prima formulazione liquida orale di desmopressina approvata dalla FDA, sviluppata specificamente per fornire dosi precise e titolabili per pazienti pediatrici. Il farmaco ha dimostrato equivalenza farmacocinetica rispetto al prodotto di riferimento approvato dalla FDA in uno studio di bioequivalenza condotto su 75 soggetti.
Punti chiave:
- La protezione brevettuale si estende fino al 2044
- Mercato target: circa 3.000 pazienti pediatrici con diabete insipido centrale negli Stati Uniti
- Le attività commerciali pre-lancio sono già in corso
Eton Pharmaceuticals (ETON) ha presentado una Solicitud de Nuevo Fármaco (NDA) a la FDA para ET-600, una solución oral patentada de desmopresina diseñada para tratar la diabetes insípida central. La compañía anticipa un período de revisión de la FDA de 10 meses, con el objetivo de una posible aprobación y lanzamiento en el primer trimestre de 2026.
ET-600 sería la primera formulación líquida oral de desmopresina aprobada por la FDA, desarrollada específicamente para proporcionar dosis precisas y ajustables para pacientes pediátricos. El medicamento ha demostrado equivalencia farmacocinética con el producto de referencia aprobado por la FDA en un estudio de bioequivalencia con 75 sujetos.
Puntos clave:
- La protección de la patente se extiende hasta 2044
- Mercado objetivo: aproximadamente 3,000 pacientes pediátricos con diabetes insípida central en EE. UU.
- Las actividades comerciales previas al lanzamiento ya están en marcha
Eton Pharmaceuticals (ETON)은 중추성 요붕증 치료를 위해 설계된 독점 구강용 데스모프레신 용액 ET-600에 대해 FDA에 신약 신청서(NDA)를 제출했습니다. 회사는 FDA의 심사 기간을 10개월로 예상하며, 2026년 1분기 승인 및 출시를 목표로 하고 있습니다.
ET-600은 FDA 승인 받은 데스모프레신의 최초 구강 액상 제형으로, 소아 환자에게 정확하고 조절 가능한 용량을 제공하도록 특별히 개발되었습니다. 해당 약물은 75명을 대상으로 한 생물학적 동등성 연구에서 FDA 승인 기준 제품과 약동학적 동등성을 입증했습니다.
주요 내용:
- 특허 보호 기간은 2044년까지 연장됨
- 목표 시장: 미국 내 중추성 요붕증 소아 환자 약 3,000명
- 출시 전 상업 활동이 이미 진행 중
Eton Pharmaceuticals (ETON) a soumis une demande d'autorisation de mise sur le marché (NDA) à la FDA pour ET-600, une solution orale propriétaire de desmopressine conçue pour traiter le diabète insipide central. La société prévoit une période d'examen par la FDA de 10 mois, visant une approbation et un lancement potentiels au premier trimestre 2026.
ET-600 serait la première formulation liquide orale de desmopressine approuvée par la FDA, spécialement développée pour fournir des doses précises et ajustables aux patients pédiatriques. Le médicament a démontré une équivalence pharmacocinétique avec le produit de référence approuvé par la FDA lors d'une étude de bioéquivalence menée auprès de 75 sujets.
Points clés :
- La protection par brevet s'étend jusqu'en 2044
- Marché cible : environ 3 000 patients pédiatriques atteints de diabète insipide central aux États-Unis
- Les activités commerciales pré-lancement sont déjà en cours
Eton Pharmaceuticals (ETON) hat einen Antrag auf Zulassung eines neuen Medikaments (NDA) bei der FDA für ET-600 eingereicht, eine proprietäre orale Lösung von Desmopressin zur Behandlung des zentralen Diabetes insipidus. Das Unternehmen rechnet mit einer 10-monatigen Prüfungsdauer durch die FDA und strebt eine mögliche Zulassung und Markteinführung im ersten Quartal 2026 an.
ET-600 wäre die erste von der FDA zugelassene orale flüssige Desmopressin-Formulierung, die speziell entwickelt wurde, um präzise, titrierbare Dosierungen für pädiatrische Patienten bereitzustellen. Das Medikament hat in einer Bioäquivalenzstudie mit 75 Probanden eine pharmakokinetische Äquivalenz zum von der FDA zugelassenen Referenzprodukt nachgewiesen.
Wichtige Punkte:
- Patentschutz gilt bis 2044
- Zielmarkt: ca. 3.000 pädiatrische Patienten mit zentralem Diabetes insipidus in den USA
- Vorbereitende kommerzielle Aktivitäten laufen bereits
- First-to-market potential as the only FDA-approved oral liquid desmopressin formulation
- Patent protection secured until 2044
- Successful bioequivalence study completed with 75 subjects
- Addresses unmet medical need in pediatric patient population
- Small target market of only 3,000 pediatric patients in the US
- Extended timeline to potential revenue with launch not expected until Q1 2026
Insights
Eton's NDA submission for patented desmopressin oral solution targets unmet pediatric need with 2044 patent protection, positioning for Q1 2026 launch.
Eton's NDA submission for ET-600 represents a significant regulatory milestone in their rare disease strategy. This proprietary oral desmopressin solution addresses a critical treatment gap for central diabetes insipidus in pediatric patients. The company has secured patent protection until 2044, creating a substantial competitive advantage for nearly two decades if approved.
The clinical foundation appears solid, with bioequivalence demonstrated in a 75-subject human study showing pharmacokinetic equivalence to the FDA-approved reference product. With the standard 10-month review timeline, Eton is actively preparing commercial infrastructure for a potential Q1 2026 launch.
The target population of approximately 3,000 pediatric patients in the US characterizes this as a classic orphan disease opportunity. This specialized approach aligns with Eton's strategic focus on addressing rare diseases with innovative formulations where traditional options fall short.
If approved, ET-600 would become the only FDA-approved oral liquid formulation of desmopressin, solving the precise dosing challenges that have frustrated pediatric endocrinologists. The submission advances Eton's specialized portfolio while addressing a genuine medical need with long-term market exclusivity.
ET-600 potentially solves critical dosing challenges for pediatric diabetes insipidus patients with precision liquid formulation currently unavailable in US market.
The NDA submission for ET-600 addresses a significant clinical challenge in pediatric endocrinology practice. Central diabetes insipidus in children requires extraordinarily precise dosing that current tablet formulations cannot reliably provide. For the estimated 3,000 pediatric patients affected in the US, treatment options have been to adult formulations that require modification.
Eton's desmopressin oral solution would be the first FDA-approved liquid formulation in the US market, enabling the small, precise, and titratable doses essential for pediatric patients. This directly responds to documented needs expressed by leading pediatric endocrinologists who have struggled with existing formulation limitations.
Current workarounds like tablet splitting or compounded formulations introduce inconsistency in both dosing and bioavailability. These inconsistencies can lead to dangerous fluctuations in fluid balance, serum sodium levels, and overall disease management in these vulnerable patients.
The bioequivalence demonstrated in Eton's study suggests clinicians would gain a more flexible dosing tool without compromising established efficacy. This represents a meaningful advancement in pediatric endocrine care, potentially improving treatment precision while maintaining the pharmacological properties that specialists trust for this challenging condition.
- NDA submitted for the treatment of central diabetes insipidus -
- Expected 10-month review; commercial preparations underway for a potential Q1 2026 launch –
DEER PARK, Ill., April 28, 2025 (GLOBE NEWSWIRE) -- Eton Pharmaceuticals, Inc (“Eton” or the “Company”) (Nasdaq: ETON), an innovative pharmaceutical company focused on developing and commercializing treatments for rare diseases, today announced the submission of a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for ET-600, Eton’s proprietary, patented oral solution of desmopressin under development for the treatment of central diabetes insipidus. The Company expects the application to be assigned a 10-month FDA review, allowing for potential approval and launch in the first quarter of 2026.
“The NDA submission for ET-600 is another important milestone for Eton, and for the thousands of children impacted by diabetes insipidus. If approved, ET-600 will be the only FDA-approved oral liquid formulation of desmopressin, providing the small, precise, and titratable doses required to treat pediatric patients,” said Sean Brynjelsen, CEO of Eton Pharmaceuticals. “Leading pediatric endocrinologists have long expressed a need for this product, and we’re pleased to be one step closer to bringing it to patients. Pre-launch commercial activities are already underway as we plan for a potential approval in the first quarter of 2026.”
Eton has been issued a patent by the U.S. Patent and Trademark Office on ET-600, which expires in 2044 and has additional patent applications under review. In a bioequivalence study conducted in 75 human subjects, ET-600 demonstrated pharmacokinetic equivalence to the FDA-approved reference product of the same active ingredient. The company estimates that central diabetes insipidus impacts approximately 3,000 pediatric patients in the United States.
About Eton Pharmaceuticals
Eton is an innovative pharmaceutical company focused on developing and commercializing treatments for rare diseases. The Company currently has seven commercial rare disease products: INCRELEX®, ALKINDI SPRINKLE®, GALZIN®, PKU GOLIKE®, Carglumic Acid, Betaine Anhydrous, and Nitisinone. The Company has six additional product candidates in late-stage development: ET-400, ET-600, Amglidia®, ET-700, ET-800 and ZENEO® hydrocortisone autoinjector. For more information, please visit our website at www.etonpharma.com.
Forward-Looking Statements
Statements contained in this press release regarding matters that are not historical facts are “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995, including statements associated with the expected ability of Eton to undertake certain activities and accomplish certain goals and objectives. These statements include but are not limited to statements regarding Eton’s business strategy, Eton’s plans to develop and commercialize its product candidates, the safety and efficacy of Eton’s product candidates, Eton’s plans and expected timing with respect to regulatory filings and approvals, and the size and growth potential of the markets for Eton’s product candidates. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. Words such as “believes,” “anticipates,” “plans,” “expects,” “intends,” “will,” “goal,” “potential” and similar expressions are intended to identify forward-looking statements. These forward-looking statements are based upon Eton’s current expectations and involve assumptions that may never materialize or may prove to be incorrect. Actual results and the timing of events could differ materially from those anticipated in such forward-looking statements as a result of various risks and uncertainties, which include, without limitation, risks associated with the process of discovering, developing and commercializing drugs that are safe and effective for use as human therapeutics, and in the endeavor of building a business around such drugs. These and other risks concerning Eton’s development programs and financial position are described in additional detail in Eton’s filings with the Securities and Exchange Commission. All forward-looking statements contained in this press release speak only as of the date on which they were made. Eton undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made.
Investor Relations:
Lisa M. Wilson, In-Site Communications, Inc.
T: 212-452-2793
E: lwilson@insitecony.com
Source: Eton Pharmaceuticals, Inc.
FAQ
When is the expected FDA approval date for Eton's ET-600 desmopressin oral solution?
How many pediatric patients could benefit from ETON's ET-600 treatment in the United States?
What makes Eton's ET-600 unique for treating central diabetes insipidus?
How long does Eton's patent protection for ET-600 last?
What clinical evidence supports ETON's ET-600 effectiveness?
