A Pivotal, Parallel-Arm, Phase 3, Open-Label, Active-controlled, Global, Multicenter, Randomized Basket Trial Investigating the Efficacy and Safety of Once-weekly Lonapegsomatropin Compared to Daily Somatropin in Prepubertal Children and Adolescents With Growth Failure or Short Stature Due to Growth Hormone Sufficient Disorders - Turner Syndrome, SHOX Deficiency, Small for Gestational Age, and Idiopathic Short Stature

This basket trial will enroll prepubertal children and adolescents with clinically diagnosed and genetically confirmed (if applicable) TS, SHOX-D, SGA, or ISS between ages of ≥2 and <18 years with open growth plates. The purpose of the study is to see how well treatment with once-weekly lonapegsomatropin works compared to treatment with daily somatropin. Approximately 186 participants will be distributed equally (1:1), to receive either lonapegsomatropin for 2 years or somatropin for 1 year followed by lonapegsomatropin for 1 year. This trial will be conducted in the United States, France, Germany, Italy, Romania, Spain and South Korea.

Start Date12 Dec 2025

Sponsor / Collaborator

Ascendis Pharma A/S

NCT06433557

/ Active, not recruitingPhase 2

A Phase 2, Open-Label, Single-Arm, 156-week Trial to Investigate the Efficacy, Safety and Tolerability of Combined Once Weekly Navepegritide and Lonapegsomatropin in Children With Achondroplasia

This proof-of-concept trial is being conducted to evaluate the efficacy, safety and tolerability of combination treatment with navepegritide and lonapegsomatropin administered as separate subcutaneous (SC) injections once weekly in children with achondroplasia (ACH) aged 2 to 11 years.

Start Date26 Jul 2024

Sponsor / Collaborator

Ascendis Pharma Growth Disorders A/S

NCT05775523

/ RecruitingNot Applicable

A Prospective, Non-interventional (NIS), Long-term, Post-Authorisation Safety Study (PASS) of Patients Treated With Lonapegsomatropin

The goal of this study is to further characterise the potential long-term safety risks of lonapegsomatropin in patients treated with lonapegsomatropin under real-world conditions in the post-marketing setting.

Start Date20 Mar 2023

Sponsor / Collaborator

Ascendis Pharma Endocrinology Division A/S

100 Clinical Results associated with Lonapegsomatropin-tcgd

100 Translational Medicine associated with Lonapegsomatropin-tcgd

100 Patents (Medical) associated with Lonapegsomatropin-tcgd

616

Literatures (Medical) associated with Lonapegsomatropin-tcgd

01 Jan 2026·Endocrinology Diabetes and Metabolism Case Reports

Impact of growth hormone treatment on a 12-year-old female with newly diagnosed panhypopituitarism and distal arthrogryposis

Article

Author: Meller, Leo L T ; Akkad, Ghassan ; Patterson, Mary

Summary:

Panhypopituitarism, characterized by multiple pituitary hormone deficiencies, is most often diagnosed in infancy or early childhood with adolescent presentation being uncommon. We present a 12-year-old female with late-onset panhypopituitarism presenting with short stature and concomitant bilateral distal arthrogryposis. Her height at presentation was 140.7 cm (6th percentile) with a growth velocity of 0.75 cm/year, Tanner stage 1, bone age consistent with chronological age, and predicted adult height of approximately 152.4 cm despite a mid-parental target near 167.6 cm. Laboratory testing supported growth hormone (GH) deficiency, central hypothyroidism, and adrenal insufficiency, with stimulation tests being subnormal. MRI showed hypoplasia of the anterior pituitary, an absent infundibulum, and an ectopic posterior pituitary, consistent with pituitary stalk interruption syndrome. Hydrocortisone (9 mg/m 2 /day) and levothyroxine (37.5 μg daily) were first initiated. Although GH was initially deferred due to concerns about worsening preexisting distal hand contractures, the family elected to begin weekly subcutaneous lonapegsomatropin-tcgd (Skytrofa, 7.6 mg, 0.24 mg/kg/week). At 3 months, the growth velocity increased to 8.7 cm/year with early breast development, and at 6 months, it reached 17.4 cm/year, bone age remained concordant with chronological age, and predicted adult height improved to approximately 162.6 cm. By 10 months, height percentile rose to the 12th percentile and Tanner stage 2 breast development was observed. Throughout treatment, there were no reported or observed changes in distal arthrogryposis hand contractures. This case report highlights that initiation of GH therapy may lead to a significant growth improvement without aggravating arthrogryposis-related contractures.

Learning points:

To report the rare co-occurrence of arthrogryposis and panhypopituitarism and discuss management.Distal arthrogryposis coexisting with GH deficiency did not worsen with GH therapy in this patient, supporting individualized risk–benefit analysis.Shared decision making is key when theoretical risks (e.g. joint symptoms) are weighed against the consequences of untreated GH deficiency.

31 Dec 2025·JOURNAL OF MEDICAL ECONOMICS

Budget impact analysis of skytrofa for the treatment of pediatric growth hormone deficiency in a US health plan

Article

Author: Noori, Wahidullah ; Raveendran, Subhara ; Kleintjens, Joris ; Miner, Paul ; Boller, Emily ; Smith, Alden

BACKGROUND:

Daily administration of growth hormone (GH) can be challenging for patients with pediatric growth hormone deficiency (pGHD) and their caregivers. Lonapegsomatropin, a prodrug of somatropin, is administered once weekly and designed to provide sustained release of active, unmodified somatropin. International guidelines recognize the burden associated with daily injections and suggest long-acting GH to improve adherence and outcomes. The design of the auto-injector for lonapegsomatropin results in zero medication wastage leading to potential cost savings.

OBJECTIVE:

To estimate the budget impact to a US health plan of introducing lonapegsomatropin to a market with existing daily and weekly GH treatments and to identify possible cost savings associated with its use.

METHODS:

The budget impact model (BIM) was developed to compare the costs associated with two hypothetical scenarios for pediatric pGHD: one without lonapegsomatropin, where daily and weekly GHs are available, and one with lonapegsomatropin introduced to the market alongside daily and weekly GHs. The model combined per-patient costs with expected market shares for GHs to estimate the total financial expenditure for both scenarios over a five-year period, simulating costs for a US payer covering one million lives. This analysis included daily somatropin, and weekly somatrogon and somapacitan, as comparators.

RESULTS:

The introduction of lonapegsomatropin resulted in a cumulative total saving of $959,629 over a 5-year time horizon, with cost savings of $40,314 in Year 1, increasing to $373,258 in Year 5. The estimated average cost savings were $3,283 per patient per month (PPPM) and $0.08 per member per month (PMPM). Scenario and sensitivity analyses demonstrated that the cost-savings with lonapegsomatropin are robust across various assumptions, including alternative market share and dosing scenarios. The primary drivers of the cost reduction were decreased acquisition costs for daily somatropin and the elimination of wastage for weekly GHs.

CONCLUSIONS:

The inclusion of lonapegsomatropin in the formulary of a US health plan can result in a significant reduction in total costs for the plan.

01 Dec 2025·Endocrine Practice

Early U.S. Real-World Treatment Patterns and Outcomes in Palopegteriparatide Treatment for Patients With Hypoparathyroidism

Article

Author: Sibley, Christopher T ; Ing, Steven W ; Edelson, Gary ; Abbott, Lisa ; Zhao, Carol ; Cusano, Natalie E ; Lubitz, Sara ; Ukena, Jenny ; Rubin, Mishaela R ; Haider, Adnan ; Schneider, Mark ; Graham, Timothy ; Rothman, Micol S ; Shoback, Dolores M

OBJECTIVE:

To examine the impact of palopegteriparatide (YORVIPATH; TransCon PTH) on real-world clinical outcomes in adult patients with hypoparathyroidism.

METHODS:

Adult patients with hypoparathyroidism who enrolled in the U.S. expanded access program as of October 2024 and provided consent were included in the analysis. Palopegteriparatide was administered at a recommended starting dose of 18 μg/day and titrated alongside conventional therapy (calcitriol and calcium). Conventional therapy requirements, palopegteriparatide dosing, serum calcium levels, and adverse events were assessed up to 12 months of treatment.

RESULTS:

Among 135 patients enrolled in the expanded access program who received at least 1 dose of palopegteriparatide, 123 provided consent for data use and were included in analysis. Nearly all patients (95.1%, 117/123) were previously treated with short-lived parathyroid hormone (PTH) therapy (primarily teriparatide or recombinant human PTH [1-84]), and 50.4% (62/123) switched directly from short-lived PTH or PTH-related protein therapy to palopegteriparatide. There was no clinically meaningful difference (>3 μg) in palopegteriparatide dose between direct switch and nondirect switch patients, with a similar trend over time. With palopegteriparatide treatment, the proportion of patients achieving independence from conventional therapy (defined as taking no calcitriol and ≤600 mg/day of elemental calcium) increased over 12 months. Mean serum calcium levels remained within the reference range (8.3-10.6 mg/dL) with palopegteriparatide, and no new safety signals were identified with up to 12 months of treatment.

CONCLUSION:

This real-world analysis of palopegteriparatide treatment of hypoparathyroidism outside of a clinical trial setting reaffirms its efficacy and safety profile and provides insights into outcomes associated with different treatment transition practices.

News (Medical) associated with Lonapegsomatropin-tcgd

12 Jan 2026

·www.biospace.com

Ascendis Pharma Provides Business and Strategic Roadmap Update at 44th Annual J.P. Morgan Healthcare Conference

- Rapidly transforming into a leading global biopharma company COPENHAGEN, Denmark, Jan. 09, 2026 (GLOBE NEWSWIRE) -- Ascendis Pharma A/S (Nasdaq: ASND) today provided a business and strategic roadmap update, including planned 2026 key corporate milestones. Ascendis President and CEO Jan Mikkelsen will present this update on Monday, January 12, during the 44 th Annual J.P. Morgan Healthcare Conference. “With our proven TransCon ® technology platform, strong R&D capabilities, and a maturing commercial infrastructure and financial profile, Ascendis is rapidly transforming into a leading global biopharma company,” said Jan Mikkelsen, President and Chief Executive Officer at Ascendis Pharma. “Over the next year, this inflection will be driven by further global penetration and commercial expansion, a third potential TransCon product approval with blockbuster potential, and an advancing R&D pipeline of highly differentiated internal and external TransCon product candidates, all of which create the foundation for sustainable long-term growth.” Selected Key Updates and Milestones TransCon PTH (palopegteriparatide, marketed as YORVIPATH ® ) YORVIPATH fourth-quarter 2025 and full-year 2025 unaudited preliminary revenue expected to be ~€187 million and ~€477 million, respectively. More than 5,300 unique U.S. patient enrollments, with nearly 2,400 unique prescribing healthcare providers at year end. Outside the U.S., now available commercially or through named patient programs in more than 30 countries, with full commercial launches anticipated in 10 additional countries by year end 2026. Confirmed product pro once-weekly TransCon PTH targeting patients receiving stable YORVIPATH doses. Ongoing label expansion trials through PaTHway60 (adults) and PaTHway Adolescent. TransCon hGH (lonapegsomatropin, marketed as SKYTROFA ® ) SKYTROFA fourth-quarter 2025 and full year 2025 unaudited preliminary revenue expected to be ~€53 million and ~€206 million, respectively. Received first label expansion in July 2025 with U.S. Food & Drug Administration (FDA) approval for adult growth hormone deficiency (GHD). Initiated Phase 3 basket trial for additional indications: idiopathic short stature (ISS), SHOX deficiency, Turner syndrome, and small for gestational age (SGA). TransCon CNP (navepegritide) In the U.S., PDUFA goal date of February 28, 2026 for pediatric achondroplasia. Submitted marketing authorization application (MAA) to the European Medicines Agency (EMA) in October 2025, with a regulatory decision on potential use in pediatric achondroplasia anticipated in the fourth quarter of 2026. TransCon CNP + TransCon hGH Combination Therapy (navepegritide plus lonapegsomatropin) On January 8, 2026, announced Week 52 topline results from Phase 2 COACH Trial, which demonstrated improvements in annualized growth velocity (AGV) across both TransCon CNP treatment-naïve and TransCon CNP-treated children that exceeded the 97 th percentile of average stature children, along with improvements in body proportionality and arm span, and a safety pro with those observed for monotherapies of TransCon CNP and TransCon hGH. In Q4 2025, Ascendis submitted a protocol and held an end of Phase 2 meeting with the FDA regarding a Phase 3 trial of TransCon CNP and TransCon hGH in pediatric achondroplasia. Week 78 COACH data update anticipated in second quarter of 2026. Planned new trials to support TransCon CNP + TransCon hGH treatment in additional indications, such as hypochondroplasia. TransCon IL-2 β/γ (onvapegleukin alfa) Expect to report median overall survival (OS) data for a cohort of 70 patients with late-line platinum-resistant ovarian cancer (PROC) from the IL-Believe Trial of TransCon IL-2 β/γ + weekly paclitaxel in the second quarter of this year. Strategic Collaborations & Investments Novo Nordisk A/S Ongoing multi-product collaboration with Novo Nordisk for TransCon technology-based therapies in obesity and metabolic diseases. Lead program TransCon semaglutide remains on track to enter the clinic as anticipated. Eyconis, Inc. Ascendis has granted Eyconis exclusive rights to develop and commercialize TransCon ophthalmology products globally and holds a 41% equity position in the company. Lead program TransCon aVEGF (EYC-0305) in development for wet AMD and other retinal diseases anticipated to enter the clinic in 2026. VISEN Pharmaceuticals Ascendis has granted VISEN Pharmaceuticals exclusive rights to develop and commercialize TransCon hGH, TransCon PTH, and TransCon CNP in Greater China and maintains a 39% ownership interest in the public company. Biologic License Application (BLA) for TransCon hGH (lonapegsomatropin) as a potential treatment for pediatric growth hormone deficiency (PGHD) was accepted by China’s National Medical Products Administration (NMPA) in March 2024, with an approval decision anticipated in the first quarter of 2026. Teijin Limited Ascendis has granted Teijin Limited an exclusive license agreement for the further development and commercialization of TransCon hGH, TransCon PTH, and TransCon CNP for rare endocrinology diseases in Japan. In August 2025, YORVIPATH received MAA approval from Japan’s Ministry of Health, Labour, and Welfare. Financial Update Unaudited preliminary estimate of 2025 financial results: Total full-year 2025 product revenue of ~€683 million: YORVIPATH full-year 2025 revenue of ~€477 million SKYTROFA full-year 2025 revenue of ~€206 million Total full-year 2025 revenue of ~€720 million Full-year 2025 gross margin expected to be ~87% Total full-year 2025 operating expenses of ~€762 million December 31, 2025 unaudited preliminary cash balance of ~€616 million Expect operating cash flow of ~€500 million in 2026, based on current plans, exchange rates and excluding any contribution from TransCon CNP Planned $120 million share repurchase program in 2026 Share Repurchase Program The Board of Directors of Ascendis has authorized a $120 million Share Repurchase Program. Purchases under the Share Repurchase Program may be made from time to time through a variety of methods, which may include open-market purchases, privately negotiated transactions, or other methods permitted under applicable securities laws. The timing and amount of any repurchases pursuant to the Share Repurchase Program will be determined based on market conditions, share price and other factors. The Share Repurchase Program does not require Ascendis to repurchase any specific number of shares, and may be modified, suspended or terminated at any time without notice. Presentation at J.P. Morgan Healthcare Conference on Monday, January 12 A live webcast of the event will be available via the Investors & News section of the Ascendis Pharma website at . The presentation will begin at 10:30 a.m. Eastern Time / 7:30 a.m. Pacific Time. A webcast replay will be available for 30 days. The Company’s slides from the J.P. Morgan presentation will be available on the same Investors & News website at . About Ascendis Pharma A/S Ascendis Pharma is applying its innovative TransCon technology platform to build a leading, fully integrated biopharma company focused on making a meaningful difference in patients’ lives. Guided by its core values of Patients, Science, and Passion, Ascendis uses its TransCon technologies to create new and potentially best-in-class therapies. 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’ potential to rapidly transform into a leading global biopharma company, (ii) anticipated commercial launches of TransCon PTH into additional countries, (iii) the PDUFA goal date of TransCon CNP for pediatric achondroplasia and EMA regulatory decision date on the potential use of TransCon CNP in pediatric achondroplasia, (iv) timing of week 78 COACH data and planned new trials to support TransCon CNP and TransCon hGH treatment in additional indications, (v) planned release of data from the IL-Believe Trial of TransCon IL-2 β/γ, (vi) clinical status of TransCon aVEGF, (vii) NMPA’s anticipated decision of TransCon hGH as a potential treatment for PGHD, (viii) the expected collaboration with Novo Nordisk and progress of TransCon semaglutide, (ix) expected full year 2025 SKYTROFA, YORVIPATH and total revenues, operating expense, cash balance and operating cash flow, (x) planned share repurchase program, (xi) Ascendis’ ability to apply its TransCon technology platform to build a leading, fully integrated biopharma company, and (xii) Ascendis’ use of its TransCon technologies 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’ current and future reports filed with, or submitted to, the U.S. Securities and Exchange Commission (SEC), including its Annual Report on Form 20-F filed with the SEC on February 12, 2025. 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, SKYTROFA ® , and YORVIPATH ® are trademarks owned by the Ascendis Pharma group. © January 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

Clinical ResultPhase 3Drug ApprovalLicense out/in

29 Jul 2025

·www.pharmaceutical-technology.com

Ascendis Pharma’s Skytrofa gains FDA approval for growth hormone deficiency

The therapy is administered subcutaneously once a week, ensuring a sustained release of active, unmodified somatropin. Credit: Andrei Kuzmik / Shutterstock.com. The US Food and Drug Administration (FDA) has granted approval for Ascendis Pharma’s Skytrofa (lonapegsomatropin-tcgd, developed as TransCon hGH), a prodrug of somatropin or human growth hormone (HGH), to treat adults with growth hormone deficiency (GHD). GHD is characterised by a decrease or complete lack of production of endogenous growth hormone. Skytrofa was previously approved by the FDA in 2021 to treat children and paediatric patients aged one year or above weighing at least 11.5kg and are experiencing growth failure due to inadequate secretion of endogenous growth hormone (GH). It is a sterile, preservative-free lyophilised powder that appears white to off-white, available in a single-dose, dual-chamber prefilled cartridge. This therapy is administered subcutaneously once a week, ensuring a sustained release of active, unmodified somatropin. The FDA’s approval for adults with GHD was based on the findings from the Phase III parallel-arm, randomised, active-controlled (open-label) and placebo-controlled (double-blind) foresiGHt trial. GlobalData Strategic Intelligence US Tariffs are shifting - will you react or anticipate? Don’t let policy changes catch you off guard. Stay proactive with real-time data and expert analysis. By GlobalData Learn more about Strategic Intelligence It evaluated the safety and efficacy of weekly TransCon hGH in comparison to a weekly placebo and daily somatropin in adults diagnosed with GHD. Ascendis Pharma president and CEO Jan Mikkelsen said: “Our market research shows Skytrofa is the treatment of choice for paediatric GHD among patients and physicians, and we are pleased to expand its availability in the US for the treatment of adults initiating therapy or switching from another growth hormone therapy. “This important milestone is the first of many planned label expansions supporting our goal to become the leading endocrinology rare disease company.” Last year, the company submitted a supplemental biologics licence application to the FDA for TransCon hGH to treat adults with GHD. Pharmaceutical Technology Excellence Awards - The Benefits of Entering Gain the recognition you deserve! The Pharmaceutical Technology Excellence Awards celebrate innovation, leadership, and impact. By entering, you showcase your achievements, elevate your industry profile, and position yourself among top leaders driving pharmaceutical advancements. Don’t miss your chance to stand out—submit your entry today! Nominate Now

Phase 3Drug ApprovalClinical Result

28 Jul 2025

·pipelinereview.com

FDA Approves SKYTROFA® (Lonapegsomatropin-tcgd) for the Once-Weekly Treatment of Adults with Growth Hormone Deficiency

COPENHAGEN, Denmark I July 28, 2025 I Ascendis Pharma A/S (Nasdaq: ASND) today announced that the U.S. Food & Drug Administration (FDA) has approved SKYTROFA ® (lonapegsomatropin-tcgd; developed as TransCon hGH) for the replacement of endogenous growth hormone in adults with growth hormone deficiency (GHD), a rare disorder resulting from decreased or total loss of growth hormone production. Lonapegsomatropin (approved by the FDA in 2021 for the treatment of pediatric GHD) is a prodrug of somatropin (human growth hormone, or hGH) administered once weekly, providing sustained release of active, unmodified somatropin. The FDA’s approval of SKYTROFA for adult GHD was based on results from foresiGHt, a Phase 3 randomized, parallel-arm, placebo-controlled (double-blind) and active-controlled (open-label) clinical trial that compared the efficacy and safety of weekly TransCon hGH with weekly placebo and daily somatropin in adults with GHD. “The abnormal body composition, dyslipidemia, and insulin resistance that are the hallmarks of adult GHD predispose affected individuals to serious medical complications such as metabolic syndrome, increased cardiovascular risk, and impaired quality of life – including cognitive dysfunction, depression, anxiety, sleep disturbance, and reduced physical and mental drive. Despite this, adherence to daily somatropin injections that have been the standard of care for more than 25 years remains a problem,” said Kevin Yuen, M.D., Barrow Neurological Institute, University of Arizona College of Medicine and Creighton University School of Medicine in Phoenix, Arizona. “It is no surprise that patients are looking for a less burdensome treatment regimen, and a new treatment option such as SKYTROFA, with once-weekly injection and its unique release of unmodified somatropin, is expected to help improve both real-world adherence and overall outcomes.” “Our market research shows SKYTROFA is the treatment of choice for pediatric GHD among patients and physicians, and we are pleased to expand its availability in the United States for the treatment of adults initiating therapy or switching from another growth hormone therapy,” said Jan Mikkelsen, President and Chief Executive Officer at Ascendis Pharma. “This important milestone is the first of many planned label expansions supporting our goal to become the leading endocrinology rare disease company.” The following information is intended for the U.S. Audience Only: IMPORTANT SAFETY INFORMATION & USES SKYTROFA is a prescription medicine used for: Do not take SKYTROFA if: Tell your healthcare provider if you are pregnant or plan to become pregnant, about all of your medical conditions, and about all the medicines you take. SKYTROFA may affect how other medicines work, and other medicines may affect how SKYTROFA works. What are the possible side effects of SKYTROFA? SKYTROFA may cause serious side effects, including: The most common side effects of SKYTROFA in children include: viral infection, fever, cough, nausea and vomiting, bleeding, diarrhea, stomach area pain, joint pain and arthritis The most common side effects of SKYTROFA in adults include: swelling due to fluid build-up and low thyroid hormone These are not all of the possible side effects of SKYTROFA. 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 click here for SKYTROFA full Prescribing Information. 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. * ISS = idiopathic short stature SHOX deficiency = short stature homeobox-containing gene deficiency SGA = small for gestational age SOURCE: Ascendis Pharma

Phase 3Drug ApprovalClinical Result

100 Deals associated with Lonapegsomatropin-tcgd

External Link

KEGG	Wiki	ATC	Drug Bank
-	Lonapegsomatropin-tcgd	-	DB16220

R&D Status

Approved

10 top approved records.

to view more data

Indication	Country/Location	Organization	Date
Failure to Thrive	Australia	Specialised Therapeutics Australia Pty Ltd.	23 May 2025
Growth hormone deficiency	United States	Ascendis Pharma Endocrinology Division A/S	25 Aug 2021

Developing

10 top R&D records.

to view more data

Indication	Highest Phase	Country/Location	Organization	Date
Dwarfism	Phase 3	United States	Ascendis Pharma A/S	12 Dec 2025
Short Stature Homeobox Deficiency	Phase 3	United States	Ascendis Pharma A/S	12 Dec 2025
Turner Syndrome	Phase 3	United States	Ascendis Pharma A/S	12 Dec 2025
Endocrine System Diseases	Phase 3	China	Ascendis Pharma A/S	30 Dec 2019
Endocrine System Diseases	Phase 3	China	VISEN Pharmaceuticals (Shanghai) Co., Ltd.	30 Dec 2019
Hashimoto Disease	Phase 3	China	VISEN Pharmaceuticals (Shanghai) Co., Ltd.	30 Dec 2019
Hashimoto Disease	Phase 3	China	Ascendis Pharma A/S	30 Dec 2019
Pituitary Diseases	Phase 3	China	Ascendis Pharma A/S	30 Dec 2019
Pituitary Diseases	Phase 3	China	VISEN Pharmaceuticals (Shanghai) Co., Ltd.	30 Dec 2019
Achondroplasia	Phase 2	Denmark	Ascendis Pharma Growth Disorders A/S	26 Jul 2024

Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT05171855 (CTgov)	Phase 3	Growth hormone deficiency	220	Lonapegsomatropin (Lonapegsomatropin/Lonapegsomatropin)	mzpjelehsm = rmuofcrgtv romfvlxnet (kainutticz, mdijhswafv - fcegobmeko) View more	-	09 Jan 2026
				Lonapegsomatropin (Placebo/Lonapegsomatropin)	mzpjelehsm = ojctdakvti romfvlxnet (kainutticz, djfqncpjhf - eljwlfvlyd) View more
NCT04615273 (foresiGHt, CTgov)	Phase 3	Growth hormone deficiency	264	Lonapegsomatropin (Lonapegsomatropin)	okbetoikyi(iusprraghq) = iikqziahow tohclmgnrp (dfbknppdtw, wsrbwjqycc - sjuptxpfth) View more	-	15 Jan 2025
				Placebo (Placebo)	okbetoikyi(iusprraghq) = rfinabcmkw tohclmgnrp (dfbknppdtw, dmkijtarpv - ztssimtlgw) View more
NCT05690386 (New InsiGHTS Trial, Company_Website) Manual	Phase 2	Turner Syndrome	49	TransCon hGH (starting dose 0.24 mg/kg/week)	vzopyvbwsi(rcoevrfqxp) = children treated with TransCon hGH demonstrated improved growth similar to daily somatropin at Week 26, independent of starting dose. pukcvfzwkc (myxunohglp ) Met View more	Positive	16 Dec 2024
				TransCon hGH (starting dose 0.30 mg/kg/week)
NCT03344458 (enliGHten, CTgov)	Phase 3	Growth hormone deficiency	298	Lonapegsomatropin	tgqkiezvmv = vufulqjzon rjpruxteow (hasvcnneun, dmuiywodza - axsctfvzgz) View more	-	08 May 2024
NCT04615273 (foresiGHt, Biospace) Manual	Phase 3	Growth hormone deficiency	259	TransCon hGH	zjlzwxevjx(evngwkizyt) = gzvtlqwcvn deanbremfj (bffgejvboo ) View more	Positive	19 Dec 2023
				Placebo	zjlzwxevjx(evngwkizyt) = wosvyiabnh deanbremfj (bffgejvboo ) View more
NCT03344458 (enliGHten, GlobeNewswire) Manual	Not Applicable	Growth hormone deficiency	81	TransCon hGH	xdebymushy(agqpkyeuav) = The most commonly reported adverse events over the course of the trial were categorized as infections, injury, and respiratory/thoracic/medical disorders. The majority of adverse events were mild in severity and unrelated to treatment. No adverse events led to discontinuation of the study treatment. hfzvqynqay (xyzotztgwb )	Positive	23 Sep 2023
				TransCon hGH (at the beginning of the open-label extension trial)
NCT03344458 (ESPE2023)	Phase 3	Growth hormone deficiency	298	Lonapegsomatropin	ayvbjaxskt(ygrssolbwz) = lxzgcphidj tfnuqdvfug (cnufytqdoh, 6)	-	21 Sep 2023
NCT04326374 (PRNewswire) Manual	Phase 3	Growth hormone deficiency	150	human growth hormone (Longpei growth promoting hormone group)	sqopagyide(utkjmrauui) = jyrivbnuqt jswjmmkjls (mllhcutipj )	Positive	17 Nov 2022
				human growth hormone (growth hormone daily preparation group)	sqopagyide(utkjmrauui) = nawlsanemd jswjmmkjls (mllhcutipj )
enliGHten Trial (ENDO2022)	Phase 3	Growth hormone deficiency	298	Lonapegsomatropin via vial/syringe	tzwjdecrgk(dxgzlxeydn) = With continued lonapegsomatropin treatment, the AE profile remained consistent with what was observed in the parent trials, with no new safety signals ymjlrjgvjw (udlholplxg )	Positive	01 Nov 2022
NCT04326374 (PRNewswire) Manual	Phase 3	Growth hormone deficiency	150	lonapegsomatropin	xyfaqrbcvc(bbxeelhirf): P-Value = 0.0010	Superior	23 May 2022
				daily growth hormone

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