Eton Pharmaceuticals Announces Acquisition of U.S. Rights to Amglidia (Glyburide Oral Suspension)
Eton Pharmaceuticals has acquired U.S. rights to Amglidia (glyburide oral suspension) from AMMTeK for treating neonatal diabetes mellitus. The product, already approved by the European Medicines Agency since 2018, has received Orphan Drug Designation from the FDA. Amglidia is a patented liquid formulation targeting approximately 300 U.S. patients with neonatal diabetes mellitus, for which no FDA-approved oral treatments currently exist. The company plans to meet with FDA in Q1 2025 and aims to submit a New Drug Application in 2026, supported by five years of real-world safety and efficacy data from European patients.
Eton Pharmaceuticals ha acquisito i diritti statunitensi per Amglidia (sospensione orale di gliburide) da AMMTeK per il trattamento del diabete mellito neonatale. Il prodotto, già approvato dall'Agenzia Europea dei Medicinali dal 2018, ha ricevuto la designazione di farmaco orfano dalla FDA. Amglidia è una formulazione liquida brevettata che mira a circa 300 pazienti negli Stati Uniti affetti da diabete mellito neonatale, per i quali attualmente non esistono trattamenti orali approvati dalla FDA. L'azienda prevede di incontrare la FDA nel primo trimestre del 2025 e intende presentare una Nuova Richiesta di Farmaco nel 2026, supportata da cinque anni di dati reali sulla sicurezza e sull'efficacia ottenuti da pazienti europei.
Eton Pharmaceuticals ha adquirido los derechos en EE. UU. para Amglidia (suspensión oral de gliburida) de AMMTeK para el tratamiento de la diabetes mellitus neonatal. El producto, ya aprobado por la Agencia Europea de Medicamentos desde 2018, ha recibido la designación de medicamento huérfano de la FDA. Amglidia es una formulación líquida patentada que tiene como objetivo a aproximadamente 300 pacientes en EE. UU. con diabetes mellitus neonatal, para los cuales actualmente no existen tratamientos orales aprobados por la FDA. La empresa planea reunirse con la FDA en el primer trimestre de 2025 y tiene como objetivo presentar una Nueva Solicitud de Medicamento en 2026, respaldada por cinco años de datos de seguridad y eficacia del mundo real de pacientes europeos.
Eton Pharmaceuticals는 AMMTeK로부터 신생아 당뇨병 치료를 위한 Amglidia (글리부리드 경구 현탁액)의 미국 권리를 인수했습니다. 이 제품은 2018년부터 유럽 의약품청의 승인을 받았으며, FDA로부터 희귀 약물 지정을 받았습니다. Amglidia는 신생아 당뇨병 환자 약 300명을 대상으로 하는 특허된 액상 제형으로, 현재 FDA가 승인한 경구 치료제가 없습니다. 회사는 2025년 1분기에 FDA와 회의를 가질 계획이며, 2026년에 유럽 환자들로부터의 5년간의 실생활 안전성 및 효능 데이터를 근거로 새로운 약물 신청서를 제출할 예정입니다.
Eton Pharmaceuticals a acquis les droits aux États-Unis pour Amglidia (suspension orale de gliburide) auprès de AMMTeK pour le traitement du diabète sucré néonatal. Le produit, déjà approuvé par l'Agence européenne des médicaments depuis 2018, a reçu la désignation de médicament orphelin de la FDA. Amglidia est une formulation liquide brevetée ciblant environ 300 patients aux États-Unis atteints de diabète sucré néonatal, pour lesquels il n'existe actuellement aucun traitement oral approuvé par la FDA. La société prévoit de rencontrer la FDA au premier trimestre de 2025 et envisage de soumettre une demande de nouveau médicament en 2026, soutenue par cinq ans de données sur la sécurité et l'efficacité de patients européens.
Eton Pharmaceuticals hat die US-Rechte an Amglidia (glyburid orale Suspension) von AMMTeK zur Behandlung von neonatalem Diabetes mellitus erworben. Das Produkt, das seit 2018 bereits von der Europäischen Arzneimittel-Agentur genehmigt wurde, hat die Orphan Drug Designation von der FDA erhalten. Amglidia ist eine patentierte Flüssigkeitsformulierung, die sich an etwa 300 US-Patienten mit neonatalem Diabetes mellitus richtet, für die derzeit keine von der FDA genehmigten oralen Behandlungen existieren. Das Unternehmen plant, sich im ersten Quartal 2025 mit der FDA zu treffen, und beabsichtigt, 2026 einen Antrag auf neue Arzneimittelzulassung zu stellen, unterstützt durch fünf Jahre an Daten zu Sicherheit und Wirksamkeit aus Europa.
- Product already approved and commercialized in Europe, reducing regulatory uncertainty
- Granted FDA Orphan Drug Designation, providing market exclusivity benefits
- Addresses unmet medical need with no FDA-approved oral alternatives
- Five years of real-world safety and efficacy data available to support FDA submission
- Small patient population of only 300 patients in the US, limiting market size
- NDA submission not expected until 2026, indicating long timeline to potential revenue
- Will face competition from existing off-label and compounded treatments
Insights
The acquisition of Amglidia's U.S. rights represents a strategic expansion of Eton's rare disease portfolio, particularly in pediatric endocrinology. With no FDA-approved oral treatments currently available for neonatal diabetes mellitus in the U.S., this creates a significant market opportunity despite the small patient population of approximately 300 patients.
The existing EMA approval and five-year real-world data from Europe significantly de-risks the regulatory pathway. The Orphan Drug Designation provides additional benefits including market exclusivity and potential tax credits. The 2026 timeline for NDA submission suggests potential commercialization by 2027, assuming standard review times.
For investors, this acquisition aligns with Eton's core competencies in liquid formulations and rare diseases. The orphan drug status and lack of approved alternatives could support premium pricing, though the small patient population may limit revenue potential. The established European track record provides validation of both safety and market demand.
The regulatory pathway for Amglidia appears well-positioned with several key advantages. The existing European approval and substantial post-market safety data significantly strengthen the potential U.S. application. The planned FDA meeting in Q1 2025 will be important for determining whether additional studies beyond the European real-world evidence will be required.
The Orphan Drug Designation is particularly valuable, offering 7 years of market exclusivity upon approval, plus potential tax credits and waived user fees. The current reliance on compounded or off-label products creates a compelling regulatory case for approval, as the FDA generally favors approved formulations over compounded alternatives for safety and standardization reasons.
- Amglidia® has been approved by the European Medicines Agency (EMA) and is commercially available in Europe
- Strong strategic fit with Eton’s existing pediatric endocrinology focus
- Amglidia has been granted Orphan Drug Designation by the U.S. Food and Drug Administration (FDA)
- Potential New Drug Application (NDA) submission in 2026
DEER PARK, Ill., Nov. 25, 2024 (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 that it has acquired the U.S. rights to Amglidia (glyburide oral suspension, known as glibenclamide in Europe) for the treatment of neonatal diabetes mellitus from AMMTeK.
“This exciting transaction adds another attractive, patented product candidate to our growing pediatric endocrinology portfolio. In addition, the product aligns with Eton’s expertise and wealth of experience in bringing to market liquid and precision dose formulations for pediatric patients,” said Sean Brynjelsen, CEO of Eton Pharmaceuticals.
“Currently, there are no FDA-approved oral treatments for neonatal diabetes mellitus. Amglidia has been used successfully to treat European patients for years, and families and pediatric endocrinologists have expressed to us the significant need for this treatment in the United States. We look forward to working with AMMTeK to bring the product to U.S. patients as quickly as possible,” concluded Brynjelsen.
Amglidia is a proprietary, patented liquid product that was developed for the treatment of neonatal diabetes mellitus by AMMTeK, a French biotechnology company. The product was approved by the EMA in 2018. The product has already been granted Orphan Drug Designation by the U.S. FDA. Neonatal diabetes mellitus is a rare condition estimated to impact approximately 300 patients in the United States. Currently, there are no FDA-approved oral treatment options and as a result, U.S. patients frequently either rely on compounded products that are not FDA-approved or administer products off-label by making homemade suspensions.
AMMTeK has conducted a post-approval study tracking five years of real-world safety and efficacy in European patients, which will be used to support Eton’s NDA submission. Eton plans to hold a meeting with the FDA in the first quarter of 2025 and anticipates submitting an NDA for the product in 2026.
About Eton Pharmaceuticals
Eton is an innovative pharmaceutical company focused on developing and commercializing treatments for rare diseases. The Company currently has five commercial rare disease products: ALKINDI SPRINKLE®, PKU GOLIKE®, Carglumic Acid, Betaine Anhydrous, and Nitisinone. The Company has three additional product candidates in late-stage development: ET-400, ET-600, 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
FAQ
When does Eton Pharmaceuticals (ETON) plan to submit NDA for Amglidia?
How many patients in the US could benefit from Amglidia according to Eton (ETON)?
When was Amglidia approved by the European Medicines Agency?
What is the current treatment status for neonatal diabetes mellitus in the US?
