Once-Weekly TransCon® CNP (Navepegritide) to Receive 10 Years of Market Exclusivity Upon Approval in the European Union

The planned first EU launch in Germany remains conditional on European Commission authorization.

Rhea-AI Summary

Ascendis Pharma (ASND) received a positive European regulatory opinion recommending continued orphan designation for once-weekly TransCon CNP for children with achondroplasia. Final European Commission adoption would provide 10 years of orphan market exclusivity.

The recommendation follows a positive opinion supporting EU marketing authorization for children aged two years and older whose growth plates remain open. Ascendis expects the Commission’s final marketing authorization decision in Q4 2026. If authorized, the company expects its first EU launch, under the name YUVIWEL, in Germany during Q1 2027. The FDA approved the treatment in February 2026 to increase linear growth in children aged two years and older with achondroplasia and open growth plates.

5 points · 1 major

PositiveiHow this balance works

How this balance works

Rhea-AI gives every point it takes from this document a weight. Minor counts 1, Moderate 3 and Major 9, so one Major point outweighs several Minor ones. The bar adds up the weights on each side, and when neither side holds more than 65% of the total the balance reads Mixed.

It reads the document as published, with the same rules for every company, and it does not look at what the market expected or at how the stock traded, so a point can be objectively good on a day the stock falls.

Rhea-AI Sentiment measures something else, the tone of the wording.

How the balance worksHow Sentiment works

0 major · 1 point

Hollow bars mark forward-looking points. How the balance works

Positive

• Major pointFDA approval in February 2026 covers increased linear growth in children two years and older with open growth plates.
• Moderate point. Forward-looking: it has not happened yet and may not happen.Maintained orphan designation would provide 10 years of orphan market exclusivity upon final European Commission adoption.
• Moderate pointPositive CHMP opinion recommends EU authorization for children two years and older with open growth plates.
• Minor pointPositive COMP opinion recommends maintaining orphan designation for TransCon CNP in children with achondroplasia.
• Minor point. Forward-looking: it has not happened yet and may not happen.First EU launch is expected in Germany during Q1 2027, if authorized.

Negative

• Minor point. Forward-looking: it has not happened yet and may not happen.European Commission decisions remain pending; Ascendis expects the final marketing authorization decision during Q4 2026.

Key Figures

Orphan market exclusivity: 10 years European Commission decision: Q4 2026 First EU launch: Q1 2027

Orphan market exclusivity

10 years

Upon final adoption of the COMP opinion by the European Commission

European Commission decision

Q4 2026

Expected final decision on the Marketing Authorisation Application

First EU launch

Q1 2027

Germany; conditional on European Commission authorization

Historical Context

1 past event · Latest: Oct 16

1 event

1. Oct 16

   CHMP opinion

   24h Move

   -0.6%

   CHMP recommended EU marketing authorization for the same treatment in eligible children.

24h Move is the share-price change in the day after each event; other market factors may also have contributed.

Key Terms

orphan medicinal product designation, marketing authorisation application, epiphyses, prodrug, +1 more

5 terms

orphan medicinal product designation regulatory

"recommending maintenance of orphan medicinal product designation"

A regulatory designation granted to a medicine aimed at treating a rare disease, giving the developer special incentives such as fee waivers, development support and a limited period of market protection once approved. For investors, it matters because these benefits can lower development costs, shorten timelines and reduce competition—think of it as a government-backed boost and temporary safety net that can increase the drug’s commercial potential and make an investment less risky.

marketing authorisation application regulatory

"Ascendis Pharma’s Marketing Authorisation Application (MAA)"

A marketing authorisation application is the formal package a drug or medical-device maker submits to a health regulator to get permission to sell a product. Think of it as an application for a sales license: regulators review safety, effectiveness and manufacturing quality before granting permission. Investors watch these submissions because approval unlocks revenue and reduces development risk, while rejection or delays can materially affect a company’s value and timeline.

epiphyses medical

"whose epiphyses are not closed"

The epiphyses are the rounded ends of long bones where bone growth occurs in children and adolescents, acting like caps on a pipe that lengthen as a person grows and later fuse into solid bone in adulthood. Investors should care because diseases, treatments, implants or imaging that affect these regions influence pediatric and orthopedic drug or device markets, regulatory reviews and long‑term safety and efficacy profiles.

prodrug technical

"TransCon CNP is a prodrug of C-type natriuretic peptide"

A prodrug is an inactive or less-active compound that is designed to be converted into an active drug inside the body, like a packaged meal that needs heating before it's ready to eat. For investors, prodrugs matter because this design can improve how a medicine is absorbed, reduce side effects, extend patent protection, or enable new dosing forms — all factors that can affect a drug's regulatory path, marketability, and commercial value.

C-type natriuretic peptide medical

"a prodrug of C-type natriuretic peptide (CNP)"

C-type natriuretic peptide is a small, naturally occurring protein the body uses to relax blood vessels and regulate cell growth, especially in blood vessel walls and bone. Think of it as a local traffic cop that calms traffic and tells cells when to slow down dividing. For investors, it matters because drugs or tests that mimic or measure this peptide can become treatments or diagnostics for heart, vascular and growth disorders, affecting market value and regulatory risk.

AI-generated analysis. How Rhea-AI works. Not financial advice.

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– Final European Commission decision expected during Q4 2026

COPENHAGEN, Denmark, Nov. 06, 2026 (GLOBE NEWSWIRE) -- Ascendis Pharma A/S (Nasdaq: ASND) today announced that the Committee for Orphan Medicinal Products (COMP) of the European Medicines Agency (EMA) has adopted a positive opinion recommending maintenance of orphan medicinal product designation for once-weekly TransCon CNP (navepegritide) as a treatment for achondroplasia in children. The designation would provide Ascendis benefits that include 10 years of orphan market exclusivity upon final adoption of the COMP opinion by the European Commission (EC).

The opinion follows the EMA’s Committee for Medicinal Products for Human Use (CHMP) positive opinion recommending marketing authorization for once-weekly TransCon CNP in the European Union for the treatment of achondroplasia in children two years of age and older whose epiphyses are not closed. The final EC decision on Ascendis Pharma’s Marketing Authorisation Application (MAA) is expected during Q4 2026.

“We are pleased to see TransCon CNP recognized as a differentiated treatment able to make a major contribution to patient care for children with achondroplasia,” said Jan Mikkelsen, President and Chief Executive Officer at Ascendis Pharma. “If authorized, we are ready to make this novel medicine available to families and healthcare providers in the European Union seeking to address the medical complications of achondroplasia.”

If authorized by the EC, Ascendis expects its first EU launch of TransCon CNP under the brand name YUVIWEL^® to be in Germany during Q1 2027.

About TransCon CNP
TransCon CNP is a prodrug of C-type natriuretic peptide (CNP) administered once weekly, designed to provide continuous exposure of active CNP to receptors on tissues throughout the body to counteract the overactive FGFR3 signaling in achondroplasia. It is the only approved therapy to provide continuous systemic exposure to active CNP over the weekly dosing interval. In February 2026, TransCon CNP was approved by the U.S. Food & Drug Administration (FDA) under the trade name YUVIWEL^® to increase linear growth in pediatric patients 2 years of age and older with achondroplasia with open epiphyses. A final decision on Ascendis Pharma’s MAA for TransCon CNP by the European Commission is anticipated in the fourth quarter of 2026.

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 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 affect physical well-being and quality of life, and may be impacted by a range of individual, clinical, and social factors. Some individuals with achondroplasia require multiple procedures and surgeries to address specific functional or anatomical concerns.

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 within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. Examples of such statements include, but are not limited to, statements relating to (i) the expectation that the orphan medicinal products designation will provide Ascendis benefits that include 10 years of orphan market exclusivity upon marketing authorization in the European Union, (ii) the anticipated timing of the European Commission’s final decision on Ascendis’ Marketing Authorisation Application for TransCon CNP (navepegritide), expected during the fourth quarter of 2026, (iii) the potential for the European Commission to grant marketing authorization for TransCon CNP, (iv) Ascendis’ expectation and anticipated timing for its first European Union launch of TransCon CNP under the brand name YUVIWEL in Germany in the first quarter of 2027, (v) the potential for TransCon CNP to make a major contribution to patient care for children with achondroplasia, (vi) Ascendis’ ability to apply its TransCon technology platform to make a meaningful difference for patients, and (vii) Ascendis’ use of TransCon to develop new and potentially best-in-class therapies 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, without limitation: dependence on third-party manufacturers, distributors, and service providers for Ascendis’ products and product candidates; risks related to regulatory review and approval, including the possibility of delays, requests for additional data or analyses, restrictions or limitations on use, approval with labeling that is more limited than expected, or failure to obtain approval in the United States, European Union, or other jurisdictions; clinical development risks, including that results from ongoing or future trials may not confirm earlier data; unforeseen safety or efficacy findings in development programs or on-market products; manufacturing, supply chain, quality, or logistics issues that could delay development or commercialization; unforeseen expenses related to commercialization of any approved Ascendis products; unforeseen research and development or selling, general and administrative expenses and other costs impacting Ascendis’ business generally; market acceptance, pricing, and reimbursement challenges, including payer coverage decisions and health technology assessments; competitive developments, including new or improved therapies; intellectual property protection, freedom-to-operate, and litigation risks; Ascendis’ ability to obtain additional funding, if needed, to support its business activities; cybersecurity, data privacy, and information technology disruptions; and the impact of international economic, political, legal, compliance, public health, and business factors, including tariffs, trade policies, currency fluctuations, and geopolitical events. 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 11, 2026, 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, TransCon, and YUVIWEL^® are trademarks owned by the Ascendis Pharma group. © November 2026 Ascendis Pharma A/S. 

Investor Contact:	Media Contact:
Chad Fugere	Melinda Baker
Ascendis Pharma	Ascendis Pharma
+1 (650) 519-7494	+1 (650) 709-8875

FAQ

AI-generated questions and answers. How Rhea-AI works. Not financial advice.

How much EU market exclusivity could Ascendis Pharma receive for TransCon CNP?

TransCon CNP would receive 10 years of orphan market exclusivity upon final European Commission adoption of the recommendation to maintain its orphan designation. The EMA’s Committee for Orphan Medicinal Products has issued a positive opinion recommending that maintenance.

When does Ascendis Pharma expect an EU decision and launch for TransCon CNP?

Ascendis expects the final European Commission marketing authorization decision during Q4 2026 and, if authorized, its first EU launch in Germany during Q1 2027. The planned EU brand name is YUVIWEL.