ASND Ascendis Pharma A/S Sponsored ADR

Ascendis Submits Marketing Authorisation Application to the European Medicines Agency for TransCon® CNP for Treatment of Children with Achondroplasia

Ascendis Submits Marketing Authorisation Application to the European Medicines Agency for TransCon® CNP for Treatment of Children with Achondroplasia

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 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 FugereMelinda Baker
Ascendis PharmaAscendis Pharma
 
  
Patti Bank 
ICR Healthcare 
+1 (415) 513-1284 
  
  





EN
08/10/2025

Underlying

To request access to management, click here to engage with our
partner Phoenix-IR's CorporateAccessNetwork.com

Reports on Ascendis Pharma A/S Sponsored ADR

Dave Nicoski ... (+2)
  • Dave Nicoski
  • Ross LaDuke

Vermilion Compass: Weekly Equity Strategy

Downgrading Financials to Underweight We remain bullish since our 4/22/25 Compass, and we will maintain our bullish outlook as long as market dynamics remain healthy and the S&P 500 (SPX) is above 6028-6059. We would need to see a break below 6569 in order to have confidence that a consolidation period has finally begun. For now, we continue to expect support to show up at the 4-month uptrend, which generally coincides with the 20-day MA. Short-term supports to buy include 6640 (the 20-day MA),...

 PRESS RELEASE

Ascendis Submits Marketing Authorisation Application to the European M...

Ascendis Submits Marketing Authorisation Application to the European Medicines Agency for TransCon® CNP for Treatment of Children with Achondroplasia 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 imp...

Wedbush Research
  • Wedbush Research
UBI UBISOFT ENTERTAINMENT SA
XNCR XENCOR INC.
XENE XENON PHARMACEUTICALS INC
WDC WESTERN DIGITAL CORPORATION
VYGR VOYAGER THERAPEUTICS INC.
TTWO TAKE-TWO INTERACTIVE SOFTWARE INC.
TSLA TESLA INC
SIMON SILICON MOTION TECHNOLOGY CORPORATION SPONSORED ADR
RARE ULTRAGENYX PHARMACEUTICAL INC.
PEGA PEGASYSTEMS INC.
PANW PALO ALTO NETWORKS INC.
NVCR NOVOCURE LTD.
NBIX NEUROCRINE BIOSCIENCES INC.
KURA KURA ONCOLOGY INC.
IMAX IMAX CORPORATION
GERN GERON CORP.
FATE FATE THERAPEUTICS INC
CTMX CYTOMX THERAPEUTICS INC.
CHKP CHECK POINT SOFTWARE TECHNOLOGIES LTD.
BMRN BIOMARIN PHARMACEUTICAL INC.
BIIB BIOGEN INC.
ASND ASCENDIS PHARMA A/S SPONSORED ADR
ARDX ARDELYX INC
CDR CD PROJEKT S.A.
FRX_CN FENNEC PHARMACEUTICALS
7974 NINTENDO CO. LTD.
EA ELECTRONIC ARTS INC.
ANAB ANAPTYSBIO INC.
ARGX ARGENX ADS
MRSN MERSANA THERAPEUTICS
ISR ISORAY
AMZN AMAZON.COM INC.
AAPL APPLE INC.
MSFT MICROSOFT CORPORATION
IBM INTERNATIONAL BUSINESS MACHINES CORPORATION
APLS APELLIS PHARMACEUTICALS
ZG ZILLOW GROUP INC. CLASS A
DNLI DENALI THERAPEUTICS INC
RCUS ARCUS BIOSCIENCES
SRRK SCHOLAR ROCK HOLDING CORPORATION
KNSA KINIKSA PHARMACEUTICALS
REPL REPLIMUNE GROUP
ARVN ARVINAS HOLDING
STRO SUTRO BIOPHARMA
CRWD CROWDSTRIKE HOLDINGS INC. CLASS A
STOK STOKE THERAPEUTICS
IDYA IDEAYA BIOSCIENCES
PASG PASSAGE BIO
BDTX BLACK DIAMOND THERAPEUTICS
ACET ADICET BIO INC
KROS KEROS THERAPEUTICS
ZNTL ZENTALIS PHARMACEUTICALS
BEAM BEAM THERAPEUTICS
LRMR LARIMAR THERAPEUTICS INC
PRAX PRAXIS PRECISION MEDICINES
U UNITY SOFTWARE
TVTX TRAVERE THERAPEUTICS INC
VRDN VIRIDIAN THERAPEUTICS INC
PLTK PLAYTIKA HOLDING
RBLX ROBLOX
DAWN DAY ONE BIOPHARMACEUTICALS INC
APP APPLOVIN CORP
ATXS ASTRIA THERAPEUTICS INC
GMTX GEMINI THERAPEUTICS INC
TYRA TYRA BIOSCIENCES INC
EWTX EDGEWISE THERAPEUTICS INC
APGE APOGEE THERAPEUTICS INC
EMBRAC B EMBRACER GROUP AB
DNTH DIANTHUS THERAPEUTICS INC
LAC. LITHIUM AMERICAS CORP NEW
ORKA ORUKA THERAPEUTICS INC
ZBIO ZENAS BIOPHARMA INC.
TLX TELIX PHARMACEUTICALS LIMITED
MAZE MAZE THERAPEUTICS INC
David Nierengarten ... (+8)
  • David Nierengarten
  • Dennis Pak
  • Geoffrey Von Der Ahe
  • Laura Chico
  • Martin Fan
  • Ritika Das
  • Robert Driscoll
  • Yun Zhong

ResearchPool Subscriptions

Get the most out of your insights

Get in touch