Ascendis Submits U.S. NDA for TransCon CNP (Navepegritide) for the Treatment of Children with Achondroplasia
Ascendis Pharma (ASND) has submitted a New Drug Application (NDA) to the FDA for TransCon CNP (navepegritide) to treat children with achondroplasia. The submission is supported by data from three randomized, double-blind, placebo-controlled clinical trials with up to three years of open-label extension data.
The treatment, administered once weekly, is designed as a prodrug of C-type natriuretic peptide (CNP) that provides continuous exposure to receptors throughout the body. Clinical data showed multiple benefits including:
- Increased growth velocity
- Reduced health-related burden
- Stronger muscle function
- Straightening of abnormal leg bowing in most treated children
The company plans to submit a Marketing Authorisation Application (MAA) to the European Medicines Agency during Q3 2025.
Ascendis Pharma (ASND) ha presentato una Nuova Richiesta di Farmaco (NDA) alla FDA per TransCon CNP (navepegritide) per trattare i bambini affetti da achondroplasia. La presentazione è supportata da dati provenienti da tre studi clinici randomizzati, in doppio cieco e controllati con placebo, con fino a tre anni di dati di estensione in aperto.
Il trattamento, somministrato una volta alla settimana, è progettato come un pro-farmaco del peptide natriuretico di tipo C (CNP) che fornisce un'esposizione continua ai recettori in tutto il corpo. I dati clinici hanno mostrato molteplici benefici, tra cui:
- Aumento della velocità di crescita
- Riduzione del carico sanitario
- Funzione muscolare più forte
- Raddrizzamento della curvatura anomala delle gambe nella maggior parte dei bambini trattati
L'azienda prevede di presentare una Richiesta di Autorizzazione alla Commercializzazione (MAA) all'Agenzia Europea dei Medicinali nel terzo trimestre del 2025.
Ascendis Pharma (ASND) ha presentado una Nueva Solicitud de Medicamento (NDA) a la FDA para TransCon CNP (navepegritide) para tratar a niños con acondroplasia. La presentación está respaldada por datos de tres ensayos clínicos aleatorizados, doble ciego y controlados con placebo, con hasta tres años de datos de extensión en abierto.
El tratamiento, administrado una vez a la semana, está diseñado como un profármaco del péptido natriurético tipo C (CNP) que proporciona una exposición continua a los receptores en todo el cuerpo. Los datos clínicos mostraron múltiples beneficios, incluyendo:
- Aumento de la velocidad de crecimiento
- Reducción de la carga relacionada con la salud
- Función muscular más fuerte
- Enderezamiento de la curvatura anormal de las piernas en la mayoría de los niños tratados
La empresa planea presentar una Solicitud de Autorización de Comercialización (MAA) a la Agencia Europea de Medicamentos durante el tercer trimestre de 2025.
Ascendis Pharma (ASND)는 FDA에 TransCon CNP (navepegritide)에 대한 신약 신청(NDA)을 제출했습니다. 이 약물은 왜소증(achondroplasia) 아동을 치료하기 위한 것입니다. 제출은 최대 3년의 개방형 확장 데이터와 함께 3개의 무작위 이중 맹검 위약 대조 임상 시험에서 얻은 데이터로 지원됩니다.
주 1회 투여되는 이 치료법은 C형 나트륨 이뇨 펩타이드(CNP)의 프로약으로 설계되어 신체 전체의 수용체에 지속적으로 노출되도록 합니다. 임상 데이터는 다음과 같은 여러 가지 이점을 보여주었습니다:
- 성장 속도 증가
- 건강 관련 부담 감소
- 강한 근육 기능
- 대부분의 치료받은 아동에서 비정상적인 다리의 굽힘 교정
회사는 2025년 3분기에 유럽 의약청(EMA)에 마케팅 승인 신청(MAA)을 제출할 계획입니다.
Ascendis Pharma (ASND) a soumis une Demande de Médicament Nouveau (NDA) à la FDA pour TransCon CNP (navepegritide) afin de traiter les enfants atteints d'achondroplasie. La soumission est soutenue par des données provenant de trois essais cliniques randomisés, en double aveugle et contrôlés par placebo, avec jusqu'à trois ans de données d'extension en ouvert.
Le traitement, administré une fois par semaine, est conçu comme un pro-médicament du peptide natriurétique de type C (CNP) qui fournit une exposition continue aux récepteurs dans tout le corps. Les données cliniques ont montré de multiples avantages, y compris:
- Augmentation de la vitesse de croissance
- Réduction de la charge liée à la santé
- Fonction musculaire renforcée
- Redressement de la déformation anormale des jambes chez la plupart des enfants traités
L'entreprise prévoit de soumettre une Demande d'Autorisation de Mise sur le Marché (MAA) à l'Agence Européenne des Médicaments au cours du troisième trimestre 2025.
Ascendis Pharma (ASND) hat einen Antrag auf Zulassung eines neuen Arzneimittels (NDA) bei der FDA für TransCon CNP (navepegritide) zur Behandlung von Kindern mit Achondroplasie eingereicht. Der Antrag wird durch Daten aus drei randomisierten, doppelblinden, placebokontrollierten klinischen Studien unterstützt, die bis zu drei Jahre an offenen Verlängerungsdaten umfassen.
Die Behandlung, die einmal wöchentlich verabreicht wird, ist als Prodrug des C-Typ natriuretischen Peptids (CNP) konzipiert, das eine kontinuierliche Exposition der Rezeptoren im gesamten Körper bietet. Klinische Daten zeigten mehrere Vorteile, darunter:
- Erhöhte Wachstumsrate
- Verringerte gesundheitsbezogene Belastung
- Stärkere Muskelkraft
- Begradigung der abnormalen Beinverkrümmung bei den meisten behandelten Kindern
Das Unternehmen plant, im dritten Quartal 2025 einen Antrag auf Marktzulassung (MAA) bei der Europäischen Arzneimittelagentur einzureichen.
- FDA submission completed for TransCon CNP with comprehensive clinical data
- Multiple clinical benefits demonstrated beyond just linear growth
- Safety and tolerability profile comparable to placebo
- Once-weekly administration offering convenient dosing schedule
- Regulatory approval still pending with uncertain timeline
- European submission not planned until Q3 2025, indicating delayed market entry
Insights
This NDA submission marks a significant regulatory milestone for Ascendis Pharma's TransCon CNP program. The submission package appears particularly robust, supported by three randomized, double-blind, placebo-controlled trials plus three years of open-label extension data – a comprehensive data set that strengthens the application's potential for approval.
What stands out is the multiple efficacy endpoints demonstrated beyond the primary growth velocity metric. The FDA typically values treatments that address multiple aspects of a condition rather than isolated symptoms. The safety profile being comparable to placebo is equally noteworthy, as safety concerns are the primary reason many pediatric drugs face regulatory challenges.
The once-weekly administration represents a significant advantage for a pediatric medication, improving the potential for treatment adherence – another factor regulators consider favorably. With the European MAA submission planned for Q3 2025, Ascendis is executing a standard sequential global regulatory strategy, starting with the FDA review which typically takes 10-12 months for standard review or 6 months if granted priority review.
Achondroplasia represents an area with approved therapies, potentially positioning TransCon CNP for expedited review pathways if the FDA determines it addresses an unmet medical need. The clinical package described suggests Ascendis has positioned this application strategically with comprehensive efficacy and safety data.
The clinical benefits reported for TransCon CNP are particularly meaningful from a patient care perspective. Achondroplasia affects approximately 1 in 25,000 births and extends far beyond just short stature – it involves skeletal abnormalities that can lead to significant medical complications and quality of life challenges.
The reported improvements in muscle function and correction of leg bowing address critical functional aspects of achondroplasia that impact mobility and long-term health outcomes. These benefits, coupled with reduced health-related burden, suggest TransCon CNP's mechanism – providing continuous exposure of CNP to receptors throughout the body – may offer a more comprehensive approach to managing the condition than treatments focused solely on linear growth.
The once-weekly administration represents a substantial advantage for pediatric patients and their families compared to daily injections that would create significant treatment burden. For a chronic condition requiring long-term management, simplified dosing is crucial for maintaining treatment adherence, especially in children.
If approved, this therapy could potentially modify the disease course by addressing multiple skeletal and functional abnormalities simultaneously, representing a meaningful advancement over current management approaches. The safety profile being comparable to placebo is particularly reassuring for a pediatric population requiring long-term treatment during critical developmental periods.
— Data demonstrated multiple clinical benefits beyond linear growth
— NDA supported by data from three randomized, double-blind, placebo-controlled clinical trials in children with achondroplasia, with up to three years of open-label extension data
— MAA in EU on track for submission during Q3 2025
COPENHAGEN, Denmark, March 31, 2025 (GLOBE NEWSWIRE) -- Ascendis Pharma A/S (Nasdaq: ASND) today announced that it has submitted its New Drug Application (NDA) to the U.S. Food & Drug Administration (FDA) for TransCon CNP (navepegritide) for the treatment of children with achondroplasia. TransCon CNP is an investigational prodrug of C-type natriuretic peptide (CNP) administered once weekly and designed to treat individuals with achondroplasia by providing continuous exposure of active CNP to receptors on tissues throughout the body, including growth plates and skeletal muscle.
The filing 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.
“We are pleased to share clinical data with the FDA demonstrating that, in addition to increased growth velocity, treatment with TransCon CNP was associated with reduced health-related burden, stronger muscle function, and straightening of abnormal leg bowing for the majority of treated children,” said Aimee Shu, M.D., Executive Vice President and Chief Medical Officer at Ascendis Pharma. “In addition to once-weekly administration, these outcomes and a safety and tolerability profile comparable to placebo support TransCon CNP’s potential to be recognized as a best-in-class treatment for achondroplasia.”
Ascendis is on track to submit its Marketing Authorisation Application (MAA) to the European Medicines Agency (EMA) during Q3 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 people 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 deformities, 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) Ascendis’ plan to submit a MAA to the EMA for TransCon CNP in Q3 2025, (ii) TransCon CNP’s potential to be recognized as a best-in-class treatment for achondroplasia, (iii) Ascendis’ ability to apply its TransCon technology platform to make a meaningful difference for patients, and (iv) Ascendis’ application of TransCon 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. 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. © March 2025 Ascendis Pharma A/S.
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FAQ
What are the main benefits shown in TransCon CNP clinical trials for achondroplasia treatment by ASND?
When will Ascendis Pharma (ASND) submit the European application for TransCon CNP?
How is TransCon CNP administered to children with achondroplasia?
What clinical data supports ASND's TransCon CNP FDA application?
