Ascendis Submits Marketing Authorisation Application to the European Medicines Agency for TransCon® CNP for Treatment of Children with Achondroplasia
Ascendis (Nasdaq: ASND) submitted a Marketing Authorisation Application (MAA) to the European Medicines Agency for TransCon CNP (navepegritide) as a once‑weekly treatment for children with achondroplasia.
The MAA is supported by data from three randomized, double‑blind, placebo‑controlled trials and up to three years of open‑label extension data, including the pivotal ApproaCH Trial. Ascendis reported TransCon CNP was generally well tolerated with no discontinuations related to the study drug. Ascendis said it will engage with the EMA during review. In the U.S., TransCon CNP is under FDA priority review with a PDUFA target date of November 30, 2025.
Ascendis (Nasdaq: ASND) ha presentato una DOMANDA DI AUTORIZZAZIONE ALLA COMMERCIO (MAA) all'Agenzia Europea per i Medicinali per TransCon CNP (navepegritide) come trattamento una volta alla settimana per i bambini con achondroplasia.
La MAA è supportata da dati provenienti da tre studi randomizzati, in doppio cieco e controllati con placebo e fino a tre anni di dati di estensione in aperta età, inclusa il fondamentale Studio ApproaCH. Ascendis ha riportato che TransCon CNP è stato generalmente ben tollerato con nessuna interruzione legata al farmaco in trial. Ascendis ha dichiarato che terrà contatti con l'EMA durante la revisione. Negli Stati Uniti, TransCon CNP è sotto una revisione prioritaria della FDA con una data obiettivo PDUFA del 30 novembre 2025.
Ascendis (Nasdaq: ASND) presentó una Solicitud de Autorización de Comercialización (MAA) a la Agencia Europea de Medicamentos para TransCon CNP (navepegritide) como tratamiento semanal para niños con acondroplasia.
La MAA se respalda con datos de tres ensayos aleatorizados, doble ciego y controles con placebo y hasta tres años de datos de extensión en abierto, incluyendo el ensayo clave ApproaCH. Ascendis informó que TransCon CNP fue generalmente bien tolerado sin discontinuaciones relacionadas con el fármaco del estudio. Ascendis dijo que se comunicará con la EMA durante la revisión. En EE. UU., TransCon CNP está bajo revisión prioritaria de la FDA con una fecha objetivo de PDUFA del 30 de noviembre de 2025.
Ascendis(나스닥: ASND)가 유럽의약청(EMA)에 마케팅 승인 신청(MAA)을 제출했습니다. TransCon CNP(navepegritide)를 매주 투여하는 치료제로 소아의 achondroplasia를 대상으로 합니다.
MAA는 3건의 무작위, 이중 맹검, 위약 대조 시험과 최대 3년의 공개 라벨 확장 데이터, 주요한 ApproaCH 시험를 포함하고 있습니다. Ascendis는 TransCon CNP가 일반적으로 잘 견뎌지는 편이며 연구 약물과 관련된 중단은 없었다고 밝혔습니다. Ascendis는 평가 중 EMA와의 소통을 진행할 것이라고 말했습니다. 미국에서는 TransCon CNP가 FDA 우선 심사를 받고 있으며 PDUFA 목표일 2025년 11월 30일입니다.
Ascendis (Nasdaq: ASND) a soumis une Demande d'autorisation de mise sur le marché (AMM) à l'Agence européenne des médicaments pour TransCon CNP (navepegritide) en tant que traitement hebdomadaire pour les enfants atteints d’achondroplasie.
La MAA est étayée par des données de trois essais randomisés, en double aveugle, contrôlés par placebo et jusqu’à trois ans de données d’extension en ouvert, y compris l’essai pivot ApproaCH. Ascendis a déclaré que TransCon CNP était généralement bien toléré avec aucune interruption liée au médicament d’étude. Ascendis a dit qu’il dialoguerait avec l'EMA pendant l’évaluation. Aux États-Unis, TransCon CNP est sous révision prioritaire par la FDA avec une date cible PDUFA du 30 novembre 2025.
Ascendis (Nasdaq: ASND) hat eine Marketing-Autorisations-Antrag (MAA) bei der Europäischen Arzneimittelbehörde für TransCon CNP (navepegritide) als wöchentliche Behandlung für Kinder mit Achondroplasie eingereicht.
Die MAA wird durch Daten aus drei randomisierten, doppelblinden, placebokontrollierten Studien und bis zu drei Jahren offener verlängernder Daten unterstützt, einschließlich der wesentlichen ApproaCH-Studie. Ascendis berichtete, dass TransCon CNP im Allgemeinen gut verträglich war und keine Abbrüche aufgrund des Studienmedikaments verzeichnet wurden. Ascendis sagte, es werde während der Prüfung mit der EMA in Kontakt treten. In den USA befindet sich TransCon CNP in FDA Priority Review mit einem PDUFA-Zieltermin vom 30. November 2025.
Ascendis (نقطة البيع: ASND) قدمت إلى وكالة الأدوية الأوروبية طلب ترخيص تسويق (MAA) لـ TransCon CNP (navepegritide) كعلاج أسبوعي للأطفال المصابين بالتقزم الغضروفي.
يستند الـ MAA إلى بيانات من ثلاث تجارب عشوائية، مزدوجة التعمية، ومقارنات مع دواء placebo وحتى ثلاث سنوات من بيانات التمديد المفتوحة، بما في ذلك الدراسة المحورية ApproaCH. ذكرت Ascendis أن TransCon CNP كان عادةً جيد التحمل دون توقفات مرتبطة بالعقار الدوائي في الدراسة. قالت Ascendis إنها ستتواصل مع EMA أثناء المراجعة. في الولايات المتحدة، يخضع TransCon CNP لـ مراجعة أولوية من FDA مع تاريخ هدف PDUFA في 30 نوفمبر 2025.
Ascendis（纳斯达克：ASND）已向欧洲药品管理局提交了一份 上市许可申请（MAA），用于 TransCon CNP（navepegritide）作为每周一次的治疗，针对患有软骨发育不良的儿童。
MAA 得到来自 三项随机、双盲、安慰剂对照试验以及长达 三年的开放标签延伸数据的支持，包括关键的 ApproaCH 试验。Ascendis 表示 TransCon CNP 的 总体耐受性良好，未有因研究药物导致的中止。Ascendis 还表示将在评审过程中与 EMA 进行沟通。在美国，TransCon CNP 处于 FDA 优先审查，PDUFA 目标日期为 2025 年 11 月 30 日。
- MAA submitted to EMA for TransCon CNP
- Clinical package includes 3 randomized placebo‑controlled trials
- Up to 3 years of open‑label extension data
- Reported no drug‑related discontinuations in trials
- Once‑weekly dosing designed for continuous CNP exposure
- Approval pending — EMA review outcome and timing are uncertain
- U.S. regulatory outcome uncertain despite PDUFA Nov 30, 2025
Insights
Submission to EMA and concurrent FDA priority review mark a major regulatory milestone for Ascendis.
Ascendis Pharma has filed a Marketing Authorisation Application with the European Medicines Agency for TransCon CNP, supported by three randomized, double-blind, placebo-controlled trials plus up to three years of open-label data, including the pivotal ApproaCH trial. The program targets continuous inhibition of FGFR3 via a once-weekly prodrug to address growth and functional impacts in children with achondroplasia.
The regulatory path now depends on the EMA review and the outcome of the U.S. FDA priority review with a
Watch for the EMA acceptance/validation decision and review timeline following the
COPENHAGEN, Denmark, Oct. 08, 2025 (GLOBE NEWSWIRE) -- Ascendis Pharma A/S (Nasdaq: ASND) today announced it has submitted a Marketing Authorisation Application (MAA) to the European Medicines Agency (EMA) for TransCon CNP (navepegritide) as a treatment for children with achondroplasia, a rare genetic condition that causes skeletal dysplasia and, for many affected individuals, significant health, physical functioning, and quality of life impacts. TransCon CNP is an investigational prodrug of C-type natriuretic peptide (CNP) administered once weekly, designed for continuous inhibition of the overactive FGFR3 pathway in achondroplasia by providing continuous exposure of active CNP to receptors on tissues throughout the body, including growth plates and skeletal muscle.
The MAA is based on data from three randomized, double-blind, placebo-controlled clinical trials and up to three years of open-label extension data, including results from the pivotal ApproaCH Trial of children with achondroplasia. In these trials, TransCon CNP has been generally well tolerated, with no discontinuations related to the study drug.
“The achondroplasia community has been clear about its need for early pharmacological interventions that improve the medical, functional, and quality of life complications,” said Aimee Shu, M.D., Executive Vice President of Endocrine & Rare Disease Medical Sciences and Chief Medical Officer at Ascendis Pharma. “We look forward to engaging with the EMA during its review to bring TransCon CNP to patients in the European Union as soon as possible.”
In the United States, TransCon CNP as a treatment for children with achondroplasia is under priority review by the U.S. Food and Drug Administration, with a Prescription Drug User Fee Act (PDUFA) target date of November 30, 2025.
About Achondroplasia
Achondroplasia is a rare genetic condition arising from a systemic fibroblast growth factor receptor 3 (FGFR3) variant that leads to an imbalance in the effects of the FGFR3 and CNP signaling pathways, estimated to affect more than 250,000 individuals worldwide. While historically considered a bone growth disorder, the FGFR3 variant seen in achondroplasia is expressed in tissues throughout the body, causing serious muscular, neurological, and cardiorespiratory complications in addition to skeletal dysplasia. Medical complications of achondroplasia vary across different stages of life. Throughout infancy and childhood, observed complications include spinal abnormalities, enlarged brain ventricles, impaired muscle strength and stamina, hearing deficits and chronic ear infections, upper airway obstructions, sleep-disordered breathing, hip problems, leg bowing, and chronic pain; many of these persist or worsen in adulthood. These medical complications can have detrimental effects on quality of life, physical functioning, and psychosocial function. Individuals with achondroplasia often require multiple surgeries and procedures to alleviate the condition’s many complications.
About Ascendis Pharma A/S
Ascendis Pharma is a global biopharmaceutical company focused on applying our innovative TransCon technology platform to make a meaningful difference for patients. Guided by our core values of Patients, Science, and Passion, and following our algorithm for product innovation, we apply TransCon to develop new therapies that demonstrate best-in-class potential to address unmet medical needs. Ascendis is headquartered in Copenhagen, Denmark, and has additional facilities in Europe and the United States. Please visit ascendispharma.com to learn more.
Forward-Looking Statements
This press release contains forward-looking statements that involve substantial risks and uncertainties. All statements, other than statements of historical facts, included in this press release regarding Ascendis’ future operations, plans and objectives of management are forward-looking statements. Examples of such statements include, but are not limited to, statements relating to (i) TransCon CNP’s potential as a new treatment option for children with achondroplasia, (ii) Ascendis’ plans to work with the EMA to make TransCon CNP available as quickly as possible, (iii) the PDUFA target date for the U.S. Food and Drug Administration’s review of TransCon CNP for the treatment of children with achondroplasia, (iv) Ascendis’ ability to apply its TransCon technology platform to make a meaningful difference for patients, and (v) Ascendis’ application of its TransCon technologies to develop new therapies that demonstrate best-in-class potential to address unmet medical needs. Ascendis may not actually achieve the plans, carry out the intentions or meet the expectations or projections disclosed in the forward-looking statements and you should not place undue reliance on these forward-looking statements. Actual results or events could differ materially from the plans, intentions, expectations and projections disclosed in the forward-looking statements. Various important factors could cause actual results or events to differ materially from the forward-looking statements that Ascendis makes, including the following: dependence on third party manufacturers, distributors and service providers for Ascendis’ products and product candidates; unforeseen safety or efficacy results in Ascendis’ development programs or on-market products; unforeseen expenses related to commercialization of any approved Ascendis products; unforeseen expenses related to Ascendis’ development programs; unforeseen selling, general and administrative expenses, other research and development expenses and Ascendis’ business generally; delays in the development of its programs related to manufacturing, regulatory requirements, speed of patient recruitment or other unforeseen delays; Ascendis’ ability to obtain additional funding, if needed, to support its business activities; the impact of international economic, political, legal, compliance, social and business factors, including tariffs and trade policies. For a further description of the risks and uncertainties that could cause actual results to differ from those expressed in these forward-looking statements, as well as risks relating to Ascendis’ business in general, see Ascendis’ Annual Report on Form 20-F filed with the U.S. Securities and Exchange Commission (SEC) on February 12, 2025, and Ascendis’ other future reports filed with, or submitted to, the SEC. Forward-looking statements do not reflect the potential impact of any future licensing, collaborations, acquisitions, mergers, dispositions, joint ventures, or investments that Ascendis may enter into or make. Ascendis does not assume any obligation to update any forward-looking statements, except as required by law.
Ascendis, Ascendis Pharma, the Ascendis Pharma logo, the company logo and TransCon are trademarks owned by the Ascendis Pharma group.© October 2025 Ascendis Pharma A/S.
| Investor Contacts: | Media Contact: |
| Chad Fugere | Melinda Baker |
| Ascendis Pharma | Ascendis Pharma |
| ir@ascendispharma.com | media@ascendispharma.com |
| Patti Bank | |
| ICR Healthcare | |
| +1 (415) 513-1284 | |
| patti.bank@icrhealthcare.com | |
FAQ
What did Ascendis (ASND) file with the EMA on October 8, 2025?
What clinical data support Ascendis's MAA for TransCon CNP (ASND)?
What is the safety profile reported for TransCon CNP in Ascendis trials?
When is the FDA decision date for TransCon CNP in the U.S. (ASND)?
How is TransCon CNP administered and what is its mechanism?
