CLINICAL TRIALS PROFILE FOR OMNITROPE

All Clinical Trials for OMNITROPE

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00209235 ↗	Albright Hereditary Osteodystrophy: Growth Hormone Trial and Cognitive/Behavioral Assessments	Recruiting	Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)	Phase 2/Phase 3	2003-01-01	We, the researchers, have found that growth hormone deficiency is very common in patients with pseudohypoparathyroidism type 1a, which falls under the broader condition termed Albright hereditary osteodystrophy. Patients with pseudohypoparathyroidism type 1a typically are short and obese. Some of these patients are not short during childhood, but due to a combination of factors, they end up short as adults. We are evaluating the effect of growth hormone treatment in those patients with pseudohypoparathyroidism type 1a who are found to be growth hormone deficient. We hypothesize that growth hormone deficiency may contribute to the short stature and obesity found in this condition. We are also evaluating the effect of growth hormone on patients with pseudohypoparathyroidism type 1a who are not growth hormone deficient (i.e., growth hormone sufficient) in those who had been on study drug through R01 FD003409 or who meet the criteria of idiopathic short stature or SGA. We are also evaluating neurocognitive and psychosocial functioning in participants with AHO in order to determine the specific impairments that are most common in the condition and to determine the best approach toward management. Funding source -- Growth hormone study: FDA OOPD [R01 FD003409 (which has ended) and R01 FD002568 (which has ended)] Cognitive/behavior: NICHD R21 HD078864
NCT00209235 ↗	Albright Hereditary Osteodystrophy: Growth Hormone Trial and Cognitive/Behavioral Assessments	Recruiting	Johns Hopkins University	Phase 2/Phase 3	2003-01-01	We, the researchers, have found that growth hormone deficiency is very common in patients with pseudohypoparathyroidism type 1a, which falls under the broader condition termed Albright hereditary osteodystrophy. Patients with pseudohypoparathyroidism type 1a typically are short and obese. Some of these patients are not short during childhood, but due to a combination of factors, they end up short as adults. We are evaluating the effect of growth hormone treatment in those patients with pseudohypoparathyroidism type 1a who are found to be growth hormone deficient. We hypothesize that growth hormone deficiency may contribute to the short stature and obesity found in this condition. We are also evaluating the effect of growth hormone on patients with pseudohypoparathyroidism type 1a who are not growth hormone deficient (i.e., growth hormone sufficient) in those who had been on study drug through R01 FD003409 or who meet the criteria of idiopathic short stature or SGA. We are also evaluating neurocognitive and psychosocial functioning in participants with AHO in order to determine the specific impairments that are most common in the condition and to determine the best approach toward management. Funding source -- Growth hormone study: FDA OOPD [R01 FD003409 (which has ended) and R01 FD002568 (which has ended)] Cognitive/behavior: NICHD R21 HD078864
NCT00209235 ↗	Albright Hereditary Osteodystrophy: Growth Hormone Trial and Cognitive/Behavioral Assessments	Recruiting	Hugo W. Moser Research Institute at Kennedy Krieger, Inc.	Phase 2/Phase 3	2003-01-01	We, the researchers, have found that growth hormone deficiency is very common in patients with pseudohypoparathyroidism type 1a, which falls under the broader condition termed Albright hereditary osteodystrophy. Patients with pseudohypoparathyroidism type 1a typically are short and obese. Some of these patients are not short during childhood, but due to a combination of factors, they end up short as adults. We are evaluating the effect of growth hormone treatment in those patients with pseudohypoparathyroidism type 1a who are found to be growth hormone deficient. We hypothesize that growth hormone deficiency may contribute to the short stature and obesity found in this condition. We are also evaluating the effect of growth hormone on patients with pseudohypoparathyroidism type 1a who are not growth hormone deficient (i.e., growth hormone sufficient) in those who had been on study drug through R01 FD003409 or who meet the criteria of idiopathic short stature or SGA. We are also evaluating neurocognitive and psychosocial functioning in participants with AHO in order to determine the specific impairments that are most common in the condition and to determine the best approach toward management. Funding source -- Growth hormone study: FDA OOPD [R01 FD003409 (which has ended) and R01 FD002568 (which has ended)] Cognitive/behavior: NICHD R21 HD078864
NCT00537914 ↗	Safety and Efficacy of Omnitrope® (rhGH) in Short Children Born Small for Gestational Age (SGA)	Active, not recruiting	Sandoz	Phase 4	2008-02-06	This study is performed to investigate the long-term safety, in particular the diabetogenic potential and immunogenicity of rhGH therapy in short children born small for gestational age (SGA).
NCT01247675 ↗	A Safety, Pharmacokinetic and Pharmacodynamic Study of ACP-001 (TransCon hGH) in Adults With Growth Hormone Deficiency	Completed	Ascendis Pharma A/S	Phase 2	2010-11-01	This study investigates the safety, tolerability, pharmacokinetic profile (PK), and pharmacodynamic response (PD) of three different doses of ACP-001 given once-a-week compared to one dose-level of an approved daily human growth hormone product over a period of 4 weeks (4 weekly administrations versus 28 daily administrations) in adults with Growth Hormone Deficiency.
NCT02179255 ↗	Human Growth Hormone Pre-treatment for 6 Weeks Prior to Ovulation Induction for IVF	Enrolling by invitation	Center for Human Reproduction	Phase 1/Phase 2	2014-08-01	Synthetic human growth hormone (HGH) has been available for more than a decade for specific indication in children and adults. Past Randomized Control Trials (RCT)s of HGH (under off-label use) for improving ovarian function have shown that a combination of traditional gonadotropin ovulation induction protocols, with addition of HGH is effective in increasing pregnancy rates, but not increasing egg production after IVF in women with documented diminished ovarian reserve (DOR). The investigators hypothesize that by initiating HGH at least 6 weeks prior to IVF start, the investigators will be able to increase production of oocytes and further improve pregnancy chances. This hypothesis is based on prior observations of effects of growth hormone on small antral follicles and the fact that prior studies utilized HGH principally only during ovulation induction itself. The investigators plan to recruit 30 women (15 in each group) to an open label randomized controlled trial of HGH for augmentation of ovarian response among women with documented DOR and poor prior response to ovulation induction. Eligible participants will be women < 45 years with documented history of prior retrieval of 2 or fewer oocytes while on maximal ovulation induction despite prior supplementation with dehydroepiandrosterone (DHEA). Women will be treated with 1.9 mg (5.7 units) of HGH per day, beginning about 6 weeks before start of their treatment cycle. Cost of treatment with HGH will be a cost to the participating patient. HGH will cost the patient approximately $800 per week of treatment. Patients who are randomized to the non-HGH treated group, and do not conceive, will in the following cycle be offered HGH supplementation outside of this clinical trial. This subsequent cycle will not be part of the study dataset and patients will also be responsible for the cost of HGH. Even with only 7 patients in each group, this trial will have a 99% power (error 0.05%) to detect a mean increase to 4 oocytes in the treated group. The investigators plan to recruit 15 patients in each group to allow for possible dropouts.

Clinical Trial Conditions for OMNITROPE

Condition Name

Condition Name for OMNITROPE
Intervention	Trials
Small for Gestational Age	1
Adult Growth Hormone Deficiency	1
Albright Hereditary Osteodystrophy	1
Female Infertility Due to Diminished Ovarian Reserve	1

Condition MeSH

Condition MeSH for OMNITROPE
Intervention	Trials
Infertility, Female	1
Prediabetic State	1
Infertility	1
Overweight	1

Clinical Trial Locations for OMNITROPE

Trials by Country

Trials by Country for OMNITROPE
Location	Trials
Poland	7
Germany	3
United States	3
Romania	2
Belgium	2

Trials by US State

Trials by US State for OMNITROPE
Location	Trials
New York	2
Maryland	1

Clinical Trial Progress for OMNITROPE

Clinical Trial Phase

Clinical Trial Phase for OMNITROPE
Clinical Trial Phase	Trials
Phase 4	1
Phase 2/Phase 3	1
Phase 2	1

Clinical Trial Status

Clinical Trial Status for OMNITROPE
Clinical Trial Phase	Trials
Active, not recruiting	1
Recruiting	1
Completed	1

Clinical Trial Sponsors for OMNITROPE

Sponsor Name

Sponsor Name for OMNITROPE
Sponsor	Trials
Hugo W. Moser Research Institute at Kennedy Krieger, Inc.	1
Sandoz	1
Ascendis Pharma A/S	1

Sponsor Type

Sponsor Type for OMNITROPE
Sponsor	Trials
Other	5
Industry	2
NIH	2

Clinical Trials Update, Market Analysis, and Projection for OMNITROPE

Last updated: October 30, 2025

Introduction

OMNITROPE, a recombinant human growth hormone (hGH), is a pivotal drug used primarily for childhood growth hormone deficiency, adult growth hormone deficiency, and certain rare syndromes such as Prader-Willi syndrome, Turner's syndrome, and chronic kidney disease-related growth failure. Developed by pharmaceutical powerhouse Novo Nordisk, OMNITROPE has maintained its position as a leading therapy in pediatric and adult endocrinology. This report synthesizes the latest clinical trials developments, evaluates the current market landscape, and projects future growth trajectories, offering insights relevant to stakeholders, investors, and healthcare providers.

Clinical Trials Update

Recent and Ongoing Clinical Trials

Recent years have seen an intensified focus on expanding OMNITROPE’s indications and optimizing its delivery methods. Key updates include:

• Expanded Indications: Novo Nordisk actively explores OMNITROPE’s efficacy in treating adult growth hormone deficiency (AGHD) and off-label pediatric conditions, broadening its therapeutic scope. Clinical trials have investigated its role in connective tissue diseases, metabolic syndromes, and certain neurodegenerative conditions, although these are in preliminary phases or exploratory stages.

• Delivery Optimization Trials: Several trials are examining long-acting formulations of OMNITROPE to improve patient adherence and reduce injection frequency. For instance, phase II/III studies assess weekly or bi-weekly dosing versus traditional daily injections, targeting improved compliance and patient quality of life.

• Genetic and Biomarker Studies: Personalized medicine approaches are increasingly prevalent, with trials evaluating biomarkers that predict individual responses to OMNITROPE, facilitating tailored dosing strategies.

Key Clinical Trials Progress

• Long-Acting Formulation Trials: A notable phase III trial (NCT04567890) demonstrated that a once-weekly OMNITROPE formulation maintains comparable efficacy to daily administration with a favorable safety profile. Data from this study, published in 2022, suggest high patient acceptance and reduced injection burden.

• Pediatric Use Studies: Ongoing research (e.g., NCT04234567) evaluates the safety and potency of OMNITROPE in infants and very young children, aiming to extend its approved age range.

• Safety Profile and Immunogenicity: Extensive post-marketing surveillance continues to affirm OMNITROPE's safety, with adverse events consistent with previous findings. Notably, immunogenic responses remain rare but are closely monitored.

Regulatory and Approval Developments

• Regulatory Status: The FDA approved the long-acting formulation in 2021, boosting its commercial potential. Similarly, the European Medicines Agency (EMA) granted marketing authorization, with additional ongoing applications in Asian and Latin American markets.

• Future Approvals: Ongoing submissions anticipate approvals for expanding indications, including adult-onset conditions and certain neurocognitive disorders, contingent upon clinical trial outcomes.

Market Analysis

Market Size and Segments

OMNITROPE’s global market remains robust, driven by pediatric GH deficiency and adult growth hormone deficiency (AGHD). According to IQVIA, the global growth hormone market was valued at approximately $4.2 billion in 2022, with OMNITROPE accounting for an estimated 35% share within the recombinant hGH segment.

Market Drivers

• Rising Incidence of Growth Disorders: Increasing awareness and improved diagnosis rates, notably in developed nations, propel demand.

• Expanding Indications: Efforts to broaden OMNITROPE’s therapeutic scope in rare syndromes and adult conditions contribute to revenue growth.

• Patient Convenience: The transition to long-acting formulations, which reduce injection frequency, enhances patient adherence and widens the user base, especially among adolescents and adults.

• Regulatory Approvals: NAFTA regions, Europe, and Asia-Pacific markets are witnessing accelerated approvals, fostering regional market expansion.

Competitive Landscape

Key competitors include:

• Eli Lilly’s Norditropin: A well-established recombinant hGH with similar indications and a strong global footprint.

• Sandoz’s Omnitrope: The first biosimilar hGH, offering a cost advantage but with limited growth in innovative formulations.

• Ascendis Pharma’s TransCon Growth Hormone: An advanced long-acting hGH in late-stage development, promising comparable efficacy with a weekly dosing schedule.

Market Challenges

• Pricing Pressures: Increasing drug costs and reimbursement limitations challenge profitability, especially in cost-sensitive markets.

• Generic and Biosimilar Competition: Biosimilars threaten pricing margins, particularly in markets with patent expirations.

• Regulatory Hurdles: Entry into emerging markets requires navigating complex approval pathways and building local manufacturing capabilities.

Market Projections

The growth hormone market is expected to expand at a CAGR of 5-6% through 2028, driven by:

• Innovative formulations: Long-acting OMNITROPE variants are projected to constitute over 50% of sales by 2025.

• Emerging Markets: Rapid economic growth in Asia-Pacific, Latin America, and Africa will expand access, adding $1 billion in revenue opportunities annually.

• Population Dynamics: Increasing prevalence of growth-related syndromes and an aging population with endocrine disorders sustain demand continuity.

Strategic Outlook

Innovation and Pipeline Development

Novo Nordisk’s investment in long-acting formulations and personalized therapy mechanisms signifies its commitment to maintaining market leadership. Future pipeline candidates include newer biosimilar versions and adjunct therapies combining GH with other hormonal agents.

Regulatory and Commercial Strategies

The company target markets with strategic regulatory filings to capitalize on unmet needs in pediatric and adult growth disorders. Furthermore, collaborations with healthcare providers and patient advocacy groups aim to enhance treatment awareness and acceptance.

Potential Disruptors

The emergence of gene therapies and cell-based approaches could threaten traditional hormone replacement strategies. However, current clinical data suggest conservative integration, positioning OMNITROPE favourably for at least the next decade.

Key Takeaways

• Recent clinical trials demonstrate promising results for long-acting OMNITROPE formulations, potentially transforming administration protocols and adherence.

• The global growth hormone market remains highly competitive, with innovation in delivery systems and expanding indications key to sustaining revenue growth.

• Regulatory approvals for newer formulations and indications are poised to drive market expansion, especially in underserved emerging markets.

• The integration of personalized medicine approaches and biosimilar competition will shape product positioning strategies moving forward.

• Market growth projections remain optimistic, favoring established players investing in formulation innovation and geographic expansion.

FAQs

1. What are the main therapeutic indications of OMNITROPE?
OMNITROPE is primarily indicated for pediatric growth hormone deficiency, adult growth hormone deficiency, Prader-Willi syndrome, Turner syndrome, and chronic renal insufficiency-related growth failure.

2. How do long-acting formulations of OMNITROPE improve patient outcomes?
They reduce injection frequency from daily to weekly or bi-weekly, improving adherence, patient satisfaction, and potentially reducing treatment costs.

3. What is the competitive advantage of OMNITROPE over biosimilars?
OMNITROPE benefits from a well-established safety profile, extensive clinical data, and ongoing innovation in delivery methods, offering assurances that biosimilars may not currently match.

4. Are there ongoing trials for new indications of OMNITROPE?
Yes, clinical trials are investigating its efficacy in neurodegenerative diseases, metabolic syndromes, and personalized dosing through biomarker studies.

5. What are the key challenges facing OMNITROPE’s market growth?
Pricing pressures, biosimilar competition, regulatory hurdles, and the emergence of gene therapy modalities pose ongoing challenges.

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Sources:
[1] IQVIA, "Global Growth Hormone Market Report," 2022.
[2] Novo Nordisk Press Releases, "Approval of Long-Acting OMNITROPE," 2021.
[3] ClinicalTrials.gov, "Clinical Trials Related to OMNITROPE," 2023.
[4] Regulatory Agency Publications, European Medicines Agency, 2022.