FDA Approves Once-Weekly YUVIWEL® (navepegritide) for Children with Achondroplasia Aged 2 Years and Older

Rhea-AI Summary

Ascendis Pharma (Nasdaq: ASND) announced FDA accelerated approval of YUVIWEL (navepegritide), the first and only once-weekly therapy to provide continuous systemic exposure to CNP for children with achondroplasia aged 2 years and older with open epiphyses. Continued approval may require confirmatory trials.

Commercial availability is expected in early Q2 2026 and a Rare Pediatric Disease Priority Review Voucher was issued.

Positive

• FDA accelerated approval granted on Feb 27, 2026
• First and only once-weekly CNP therapy with continuous exposure
• Commercial availability expected early Q2 2026
• Rare Pediatric Disease Priority Review Voucher awarded

Negative

• Approval is accelerated and may require confirmatory trials
• Risk of low blood pressure reported as serious side effect
• Common injection site reactions (redness, itching, swelling)

Market Reaction – ASND

+4.75% $244.60

15m delay 5 alerts

+4.75% Since News

+2.8% Peak in 1 min

$244.60 Last Price

$224.21 $258.99 Day Range

+$656M Valuation Impact

$14.47B Market Cap

0.0x Rel. Volume

Following this news, ASND has gained 4.75%, reflecting a moderate positive market reaction. Argus tracked a peak move of +2.8% during the session. Our momentum scanner has triggered 5 alerts so far, indicating moderate trading interest and price volatility. The stock is currently trading at $244.60. This price movement has added approximately $656M to the company's valuation.

Data tracked by StockTitan Argus (15 min delayed). Upgrade to Silver for real-time data.

Key Figures

Indicated age: 2 years and older Dosing frequency: Once-weekly Pivotal trials: 3 trials +5 more

8 metrics

Indicated age 2 years and older Children with achondroplasia with open epiphyses

Dosing frequency Once-weekly YUVIWEL treatment schedule for pediatric achondroplasia

Pivotal trials 3 trials Randomized, double-blind, placebo-controlled studies supporting approval

Open-label follow-up Up to 3 years Extension safety and efficacy data for TransCon CNP

Priority Review Voucher Rare Pediatric Disease PRV Granted in connection with YUVIWEL FDA approval

Share price $233.50 Pre-news level on publication date

Daily price change 1.97% ASND move on the day of the article

Trading volume 766,074 shares Volume on day of article vs 20-day avg 759,687

Market Reality Check

Price: $228.99 Vol: Volume 766,074 is roughly...

normal vol

$228.99 Last Close

Volume Volume 766,074 is roughly in line with the 20-day average of 759,687. normal

Technical Shares at $233.50 are trading above the 200-day MA of $196.75 and about 3.51% below the 52-week high of $242.00, reflecting a strong pre-news uptrend.

Peers on Argus

ASND was up 1.97% while momentum-flagged peers BMRN and EXEL were down 4.91% and...

2 Down

ASND was up 1.97% while momentum-flagged peers BMRN and EXEL were down 4.91% and 2.32%, respectively, indicating stock-specific strength versus weaker biotech peers.

Previous Fda approval Reports

3 past events · Latest: Jul 28 (Positive)

Same Type Pattern 3 events

Date	Event	Sentiment	Move	Catalyst
Jul 28	FDA approval	Positive	-2.0%	FDA approval of once-weekly SKYTROFA for adults with growth hormone deficiency.
Sep 30	Regulatory submission	Positive	+3.2%	Submission of sBLA to expand TransCon hGH label for adults with GHD.
Aug 12	FDA approval	Positive	+1.2%	FDA approval of YORVIPATH as first treatment for adult hypoparathyroidism.

Pattern Detected

FDA-related catalysts have produced mixed but generally constructive reactions, with two prior same-tag events trading higher and one selling off despite positive outcomes.

Recent Company History

Over the past two years, Ascendis has steadily built an endocrinology and rare-disease franchise through FDA interactions. Prior fda approval-tagged milestones included approval of once-weekly SKYTROFA for adult growth hormone deficiency and approval of YORVIPATH for hypoparathyroidism, alongside an sBLA aimed at expanding SKYTROFA’s label in adults with GHD. Those events showed modest average moves of about 0.82%, with both up and down sessions, suggesting measured market reactions to even meaningful regulatory wins. Today’s YUVIWEL approval extends this pattern into achondroplasia.

Historical Comparison

+0.8% avg move · In the past 18 months, ASND logged 3 FDA-related milestones averaging a 0.82% move. This achondropla...

fda approval

+0.8%

Average Historical Move fda approval

In the past 18 months, ASND logged 3 FDA-related milestones averaging a 0.82% move. This achondroplasia approval fits an ongoing pattern of incremental, not explosive, reactions.

Regulatory wins have progressed from YORVIPATH for adult hypoparathyroidism and SKYTROFA for adult GHD toward broader TransCon-based indications, now including pediatric achondroplasia with YUVIWEL.

Market Pulse Summary

This announcement delivers FDA accelerated approval for once-weekly YUVIWEL in children with achondr...

Analysis

This announcement delivers FDA accelerated approval for once-weekly YUVIWEL in children with achondroplasia aged 2 years and older, supported by three randomized, placebo-controlled trials and up to 3 years of extension data. It also grants Ascendis a Rare Pediatric Disease Priority Review Voucher, adding strategic optionality. Historically, FDA milestones such as YORVIPATH and SKYTROFA approvals have steadily expanded Ascendis’s rare-disease footprint, while leaving execution, reimbursement, and confirmatory-trial outcomes as key future watchpoints.

Key Terms

achondroplasia, c-type natriuretic peptide, fgfr3, prodrug, +4 more

8 terms

achondroplasia medical

"children 2 years of age and older with achondroplasia with open epiphyses"

A genetic condition that causes the most common form of short stature, where a specific change in a growth-control gene makes the long bones grow more slowly, producing a distinct body proportion and sometimes breathing, spinal or joint issues. It matters to investors because it defines a clear patient population, predictable medical needs and regulatory pathways for drugs or devices—similar to a niche market with steady, long-term demand for effective treatments or supportive care.

c-type natriuretic peptide medical

"YUVIWEL is a prodrug of C-type natriuretic peptide (CNP) administered once weekly"

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.

fgfr3 medical

"to counteract the overactive FGFR3 signaling in achondroplasia"

FGFR3 is a gene that makes a protein sitting on the surface of cells which helps control growth and division, like a light switch that tells a cell when to grow. Changes in FGFR3 can drive certain cancers or other growth disorders, so tests and drugs that target it can be important for diagnosing disease, selecting patients for treatments, and creating new therapies — all of which affect the commercial prospects and risk profile of biotech investments.

prodrug medical

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

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.

accelerated approval regulatory

"granted approval under the FDA’s Accelerated Approval Program for YUVIWEL"

Accelerated approval is a process that allows new medical treatments to be approved more quickly than usual if they address serious or life-threatening conditions and show promising early results. For investors, it signals that a treatment may reach the market sooner, potentially boosting a company's prospects, but it also involves some uncertainty since full evidence of effectiveness is still being gathered.

new drug application regulatory

"submitted with the Company’s New Drug Application, which included safety and efficacy data"

A new drug application is a formal request submitted to government regulators seeking approval to market a new medicine. It is like a detailed proposal that shows the drug has been tested for safety and effectiveness. For investors, receiving approval signals that the drug may soon become available for sale, potentially leading to revenue growth and impacting the company's value.

annualized growth velocity medical

"approval for this indication, which was based on an improvement in annualized growth velocity (AGV)"

Annualized growth velocity is a way of expressing how fast a financial metric (such as revenue, earnings, or user base) would grow over a full year based on growth observed during a shorter period. Investors use it to compare performance across companies or timeframes and to project future size; think of timing how far a car travels in one minute and converting that to a miles‑per‑hour estimate to understand overall speed.

placebo-controlled medical

"data from three randomized, double-blind, placebo-controlled clinical trials"

"Placebo-controlled" describes a testing method where one group receives the actual treatment or intervention, while another group receives a harmless, inactive version called a placebo. This approach helps determine whether the real treatment has genuine effects beyond psychological expectations. For investors, understanding this ensures confidence that reported benefits are real and not influenced by bias or false perceptions.

AI-generated analysis. Not financial advice.

• The first and only approved achondroplasia therapy to provide continuous systemic exposure to CNP over the weekly dosing interval
• Commercial availability expected during early part of Q2 2026
• Rare Pediatric Disease Priority Review Voucher granted in connection with approval
• Ascendis to host investor conference call Monday, March 2, at 8:00 am ET

COPENHAGEN, Denmark, Feb. 27, 2026 (GLOBE NEWSWIRE) -- Ascendis Pharma A/S (Nasdaq: ASND) today announced that the U.S. Food & Drug Administration (FDA) has granted approval under the FDA’s Accelerated Approval Program for YUVIWEL^® (navepegritide; developed as TransCon^® CNP), the first and only once-weekly treatment indicated to increase linear growth in children 2 years of age and older with achondroplasia with open epiphyses and the only one to provide continuous systemic exposure to CNP over the weekly dosing interval. Continued approval for this indication, which was based on an improvement in annualized growth velocity (AGV), may be contingent upon verification and description of clinical benefit in confirmatory trial(s).

Achondroplasia is a rare genetic condition causing skeletal dysplasia and, for many affected individuals, an increased risk of muscular, neurological, and cardiorespiratory complications. YUVIWEL 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.

“The approval of once-weekly YUVIWEL is a major step forward in the treatment of children with achondroplasia, giving physicians for the first time the option of prescribing a once-weekly medicine backed by compelling efficacy and excellent tolerability data from three randomized, double-blind, placebo-controlled clinical trials,” said Carlos A. Bacino, MD, FACMG, Professor of Molecular and Human Genetics, Baylor College of Medicine and Texas Children’s Hospital. “My goal is to help children and parents develop care plans tailored to their individual needs and objectives, and I look forward to adding YUVIWEL to my discussions with them.”

“Little People of America, the largest national advocacy and support organization for people with dwarfism, is committed to ensuring that the voices of people with dwarfism remain central in conversations about research and medical options such as YUVIWEL,” said the Board of Directors of Little People of America. “We champion dwarf and disability pride, advocate for inclusion and respect, and foster open dialogue across diverse perspectives. Our goal is to empower individuals and families to make healthcare decisions that reflect their own values and experiences, while pushing for research efforts and new treatment options such as this that could have the potential to support outcomes that truly matter to our community.”

The FDA based its approval of YUVIWEL on their review of the clinical package for TransCon CNP submitted with the Company’s New Drug Application, which included safety and efficacy data from three randomized, double-blind, placebo-controlled clinical trials and up to three years of open-label extension data. The pivotal ApproaCH Trial data is available in JAMA Pediatrics.ⁱ

“We are confident in YUVIWEL’s potential to transform the treatment of achondroplasia and are deeply grateful to patients, clinicians, and advocates for their many contributions to this important milestone,” said Jan Mikkelsen, President and Chief Executive Officer at Ascendis Pharma. “We have listened to advocacy groups for people with dwarfism to ensure we address what the community actually cares about. This reflects our ongoing commitment to pursue outcomes that patient communities have told us are important to them, and gives the achondroplasia community a new way to look at the promise of pharmacological treatment options.”

Ascendis expects to make YUVIWEL available through prescribing physicians in the United States during the early part of the second quarter of 2026. Ascendis plans to offer a suite of patient services for YUVIWEL through its U.S. Ascendis Signature Access Program (A.S.A.P.), including support navigating the treatment journey and financial assistance programs for eligible patients.

With this approval, the FDA also issued a Rare Pediatric Disease Priority Review Voucher (PRV), which confers priority review to a subsequent drug application that would not otherwise qualify for priority review. This program is designed to encourage development of new drugs and biologics for the prevention or treatment of rare pediatric diseases.

Conference Call and Webcast Information
Ascendis will host a call to review the FDA approval of YUVIWEL on Monday, March 2, 2026, at 8:00 am ET. Those who would like to participate may access the live webcast here or register in advance here. The link to the live webcast and slides will also be available on the Investors & News section of the Ascendis Pharma website at https://investors.ascendispharma.com. A replay of the webcast will be available on this section of the Ascendis Pharma website shortly after conclusion of the event for 30 days.

The following information is intended for the U.S. audience only:
USE AND IMPORTANT SAFETY INFORMATION

What is YUVIWEL?
YUVIWEL is a prescription medicine used to increase linear growth in children 2 years and older with achondroplasia with open growth plates (epiphyses).

YUVIWEL is approved under accelerated approval based on an improvement in annualized growth velocity. Continued approval may be contingent upon verification and description of clinical benefit in confirmatory trials.

IMPORTANT SAFETY INFORMATION

What are the possible side effects of YUVIWEL?
YUVIWEL may cause serious side effects, including risk of low blood pressure. If your child experiences a decrease in blood pressure or symptoms of low blood pressure (dizziness, feeling tired, and/or nausea) while being treated with YUVIWEL, call your child’s healthcare provider.

The most common side effects of YUVIWEL include injection site reactions (redness, itching, skin discoloration, bleeding, swelling, bruising, pain, and blistering).

Before you give YUVIWEL to your child, tell the healthcare provider about all of your child’s medical conditions, including if they:

• have kidney problems
• are pregnant or plan to become pregnant. It is not known if YUVIWEL will harm the unborn baby
• are breastfeeding or plan to breastfeed. It is not known if YUVIWEL passes into breast milk
  
  

Tell the healthcare provider about all the medicines your child takes, including prescription and over-the-counter medicines, vitamins, and herbal supplements.

These are not all of the possible side effects of YUVIWEL. Call your doctor for medical advice about side effects. You are encouraged to report side effects to FDA at 1-800-FDA-1088 or at www.fda.gov/medwatch. You may also report side effects to Ascendis Pharma at 1-844-442-7236.

Please see full Prescribing Information for YUVIWEL.

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, and is associated with an increased risk of muscular, neurological, and cardiorespiratory complications in addition to skeletal dysplasia. Medical complications of achondroplasia can vary from individual to individual and across different stages of life. Throughout infancy and childhood, observed complications include spinal abnormalities, enlarged brain ventricles, impaired muscle strength and reduced stamina, hearing deficits and chronic ear infections, upper airway obstructions, sleep-disordered breathing, hip problems, leg bowing, and chronic pain; some of which persist or worsen in adulthood. These medical complications can affect quality of life, physical well-being, influenced by a range of clinical, social, and individual 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 expected timing for the commercial availability of YUVIWEL, (ii) the continued approval for the indication, (iii) the suite of patient services Ascendis plans to offer for YUVIWEL, (iv) YUVIWEL’s potential to transform the treatment of achondroplasia, (v) Ascendis’ ability to apply its TransCon technology platform to make a meaningful difference for patients’ lives and (vi) Ascendis’ use of TransCon to create new and potentially best-in-class therapies. 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, TransCon, and YUVIWEL are trademarks owned by the Ascendis Pharma group. © February 2026 Ascendis Pharma A/S.

Investor Contacts: Chad Fugere Ascendis Pharma ir@ascendispharma.com	Media Contact: Melinda Baker Ascendis Pharma media@ascendispharma.com
Patti Bank ICR Healthcare +1 (415) 513-1284 patti.bank@icrhealthcare.com

ⁱ Savarirayan R, McDonnell C, Bacino CA, et al. JAMA Pediatr 2026;180(1):18–25. Published online November 17, 2025. doi:10.1001/jamapediatrics.2025.4771

FAQ

What did Ascendis (ASND) announce about YUVIWEL on February 27, 2026?

Ascendis announced FDA accelerated approval of YUVIWEL (navepegritide) for children 2 years and older with achondroplasia. According to Ascendis, approval is based on improved annualized growth velocity and may require confirmatory trials for continued approval.

When will YUVIWEL (ASND) be available for prescribing in the U.S.?

Ascendis expects to make YUVIWEL commercially available in early Q2 2026. According to Ascendis, the company will offer patient support and financial assistance through its U.S. Ascendis Signature Access Program (A.S.A.P.).

What is unique about YUVIWEL's dosing and mechanism versus other achondroplasia treatments (ASND)?

YUVIWEL is once-weekly and delivers continuous systemic CNP exposure across the dosing interval. According to Ascendis, it is designed to counteract overactive FGFR3 signaling by providing sustained CNP to relevant tissues.

Does the FDA approval of YUVIWEL (ASND) carry any regulatory conditions?

Yes. Approval is accelerated and may be contingent on confirmatory trials to verify clinical benefit. According to Ascendis, the approval was granted based on improvement in annualized growth velocity.

What safety risks and common side effects did Ascendis highlight for YUVIWEL (ASND)?

Serious risk of low blood pressure and common injection site reactions were highlighted as key safety concerns. According to Ascendis, patients should monitor for dizziness, fatigue, nausea, and report side effects to healthcare providers and FDA.