CLINICAL TRIALS PROFILE FOR NATPARA

All Clinical Trials for NATPARA

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00473265 ↗	Bone Properties in Hypoparathyroidism: Effects of PTH	Completed	National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)	Phase 2/Phase 3	2004-05-01	Whereas much information is known about the properties of bone in primary hyperparathyroidism, a disorder of parathyroid hormone (PTH) excess, virtually nothing is known about the skeleton in hypoparathyroidism, a disorder in which PTH is absent. The purpose of this research project is to test the hypothesis that the skeleton in hypoparathyroidism is abnormal in its metabolic, densitometric, geometric, biomechanical and microarchitectural features. We will also test the hypothesis that the skeleton is dependent upon PTH for normal structure and function. Using non-invasive approaches as well as direct analysis of bone itself, the human hypoparathyroid skeleton will be thoroughly characterized. With each patient serving as his/her own control, we will determine how, to what extent, and in what ways the administration of PTH restores skeletal dynamics and structure to the hypoparathyroid skeleton. In this way, we will identify those structural and dynamic elements of the skeleton that are influenced by or dependent upon PTH. Methods to be utilized include dual energy X-ray absorptiometry, quantitative central and peripheral computed tomography, geometry and size quantification, histomorphometry by standard and microCT methods, finite element analysis, biochemical bone markers, quantitative back scattered electron imaging, and Fourier Transform Infrared Spectroscopy. This research project will extend our knowledge of the skeletal effects of PTH to its deficient range and thus complete our understanding of PTH action on bone gained by our many years of studying PTH overexpression in primary hyperparathyroidism. This investigation may also provide insight into the means by which PTH helps to restore the skeleton when it is used to treat osteoporosis.
NCT00473265 ↗	Bone Properties in Hypoparathyroidism: Effects of PTH	Completed	John P. Bilezikian	Phase 2/Phase 3	2004-05-01	Whereas much information is known about the properties of bone in primary hyperparathyroidism, a disorder of parathyroid hormone (PTH) excess, virtually nothing is known about the skeleton in hypoparathyroidism, a disorder in which PTH is absent. The purpose of this research project is to test the hypothesis that the skeleton in hypoparathyroidism is abnormal in its metabolic, densitometric, geometric, biomechanical and microarchitectural features. We will also test the hypothesis that the skeleton is dependent upon PTH for normal structure and function. Using non-invasive approaches as well as direct analysis of bone itself, the human hypoparathyroid skeleton will be thoroughly characterized. With each patient serving as his/her own control, we will determine how, to what extent, and in what ways the administration of PTH restores skeletal dynamics and structure to the hypoparathyroid skeleton. In this way, we will identify those structural and dynamic elements of the skeleton that are influenced by or dependent upon PTH. Methods to be utilized include dual energy X-ray absorptiometry, quantitative central and peripheral computed tomography, geometry and size quantification, histomorphometry by standard and microCT methods, finite element analysis, biochemical bone markers, quantitative back scattered electron imaging, and Fourier Transform Infrared Spectroscopy. This research project will extend our knowledge of the skeletal effects of PTH to its deficient range and thus complete our understanding of PTH action on bone gained by our many years of studying PTH overexpression in primary hyperparathyroidism. This investigation may also provide insight into the means by which PTH helps to restore the skeleton when it is used to treat osteoporosis.
NCT02781844 ↗	Study to Assess the Blood Concentrations and Actions of Recombinant Human Parathyroid Hormone (rhPTH [1-84]) When Given Once and Twice Daily to Participants With Hypoparathyroidism	Completed	Shire	Phase 1	2017-04-03	This study is being conducted to characterize the effects of twice daily administration of rhPTH(1-84) on the way the body handles rhPTH(1-84) as well as its actions and safety and tolerability over the course of 24 hours as compared with the current once daily dosing regimen of marketed rhPTH(1-84) (marketed in the United States as Natpara® and in the EU as Natpar).
NCT02986607 ↗	Corticosteroid Rhythms in Hypoparathyroid Patients	Unknown status	Haukeland University Hospital	Early Phase 1	2016-03-01	The investigators hypothesise that parathyroid hormon (PTH) depletion interferes with normal activity of the hypothalamus-pituitary-adrenal (HPA)-axis and the renin-angiotensin-aldosterone-system (RAAS), which in turn may impact morbidity and quality of life. The main objective of the current study is to test if PTH deficiency influences the secretion of corticosteroids and whether any abnormalities in the HPA-axis and the RAAS-system can be reversed by PTH infusion.

Clinical Trial Conditions for NATPARA

Condition Name

Condition Name for NATPARA
Intervention	Trials
Hypoparathyroidism	4
Hyperparathyroidism	1

Condition MeSH

Condition MeSH for NATPARA
Intervention	Trials
Hypoparathyroidism	4
Hyperparathyroidism	1

Clinical Trial Locations for NATPARA

Trials by Country

Trials by Country for NATPARA
Location	Trials
United States	11
Hungary	2
Israel	1
Canada	1
Norway	1

Trials by US State

Trials by US State for NATPARA
Location	Trials
New York	2
Tennessee	1
Pennsylvania	1
Ohio	1
Minnesota	1

Clinical Trial Progress for NATPARA

Clinical Trial Phase

Clinical Trial Phase for NATPARA
Clinical Trial Phase	Trials
Phase 2/Phase 3	1
Phase 2	1
Phase 1	1

Clinical Trial Status

Clinical Trial Status for NATPARA
Clinical Trial Phase	Trials
Completed	3
Unknown status	1

Clinical Trial Sponsors for NATPARA

Sponsor Name

Sponsor Name for NATPARA
Sponsor	Trials
University of Bergen	1
Entera Bio Ltd.	1
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)	1

Sponsor Type

Sponsor Type for NATPARA
Sponsor	Trials
Other	3
Industry	2
NIH	1

Clinical Trials Update, Market Analysis, and Projection for NATPARA (parathyroid hormone)

Last updated: October 29, 2025

Introduction

NATPARA (recombinant human parathyroid hormone, hPTH) is a prescription-grade medication indicated for adult patients with hypoparathyroidism—a rare endocrine disorder characterized by inadequate secretion of parathyroid hormone (PTH). Since its approval by the FDA in 2015, NATPARA has played a pivotal role in managing this chronic condition where traditional treatments often fall short. This report provides a comprehensive analysis of ongoing clinical trials, market dynamics, and future growth projections for NATPARA, offering critical insights for stakeholders.

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Clinical Trials Update

Current Status and Pipeline Progress

Since initial FDA approval, NATPARA's clinical landscape has evolved with multiple studies assessing its efficacy, safety, and novel applications:

• Post-approval Safety and Effectiveness Trials: The FDA mandated post-marketing surveillance to monitor adverse effects and long-term outcomes. These studies demonstrate sustained biochemical control with NATPARA, with safety profiles consistent with clinical trial data ([1]).

• Expanded Indications & Novel Formulations: Recent trials aim to explore NATPARA's potential in other disorders related to PTH deficiency, such as osteoporosis and chronic hypoparathyroidism complications. For instance, a Phase 2 trial investigated the efficacy of sustained-release formulations to improve adherence ([2]).

• Combination Therapy Investigations: Some ongoing research examines NATPARA's synergy with vitamin D analogs to improve calcium homeostasis while minimizing supplementation doses ([3]).

Clinical Trials in Progress

• Study NCT04391281: A Phase 4 trial evaluating NATPARA's long-term safety over five years in hypoparathyroid patients.

• Trial NCT04650702: Focuses on pharmacokinetics and dose optimization for pediatric populations with hypoparathyroidism. This trial signals consideration for expanding usage to younger demographics.

• Emerging Research on Delivery Methods: Trials are exploring alternative delivery systems, like subcutaneous implants, aiming to improve patient compliance and convenience.

Safety Profile & Adverse Events

NATPARA has demonstrated a manageable safety profile, with most adverse events being mild to moderate, including transient hypocalcemia and injection site reactions. Rare reports of osteosarcoma in animal studies have prompted cautious long-term monitoring ([4]).

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Market Analysis

Current Market Landscape

Hypoparathyroidism is a rare condition affecting approximately 60,000 patients globally, primarily in North America and Europe. NATPARA serves as a targeted therapy, representing a niche but crucial market segment:

• Market Exclusivity & Competition: As a recombinant PTH therapy, NATPARA holds a unique position, with limited direct competitors. Off-label uses and custom compounding formulations are common but lack regulatory approval.

• Pricing & Reimbursement: NATPARA’s annual treatment cost ranges from $50,000 to $70,000, influenced by dosing and region. Reimbursement policies vary, but coverage is generally available in major markets under rare disease designations.

Market Drivers

• Growing Awareness & Diagnosis: Increased awareness of hypoparathyroidism's clinical manifestations has led to earlier diagnosis, expanding the eligible patient base.

• Unmet Medical Needs: Traditional management with calcium and vitamin D analogs often fails to achieve optimal control, driving demand for PTH replacement therapy.

• Regulatory & Manufacturing Enhancements: Biotech collaborations aim to improve production efficiency, potentially reducing costs and expanding access.

Constraints & Challenges

• Limited Patient Pool: The rarity of hypoparathyroidism constrains market expansion.

• Long-term Safety Concerns: Ongoing safety surveillance may impact prescribing patterns.

• Pricing Pressures: Payer skepticism over high costs may limit reimbursement, especially in markets emphasizing cost-effectiveness.

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Market Projection

Forecast Assumptions

Based on current clinical and commercial data, the following key assumptions guide projections:

• Steady Clinical Adoption: Continued positive clinical trial results bolster confidence among endocrinologists.

• Regulatory Expansion: Pending approval of new formulations and expanded indications could broaden the patient base.

• Geographical Growth: Market penetration in Europe and emerging markets is expected to grow, driven by increasing awareness and healthcare infrastructure investment.

Growth Trajectory (2023-2030)

• Compound Annual Growth Rate (CAGR): Predicted at approximately 7%–9%, primarily driven by increased diagnosis and expanded indications.

• Market Revenue: Estimated to reach $420–$580 million by 2030, reflecting enhanced utilization and potential introduction of biosimilar alternatives.

• Potential Market Share Gain: The arrival of improved formulations and combination therapies could elevate NATPARA's share within the rare endocrine disorder therapeutics segment.

Potential Opportunities & Risks

• Opportunities

  • Development of next-generation, longer-acting PTH analogs.
  • Expansion of use cases, such as osteoporosis or fracture healing.
  • Increasing engagement in clinical research and real-world evidence generation.

• Risks

  • Emergence of competing therapies, including gene therapy approaches or new biologics.
  • Regulatory setbacks or safety concerns.
  • Cost containment policies impacting reimbursement.

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Expert Insights & Strategic Considerations

• Clinical Development Focus: Prioritizing long-term safety data and pediatric studies can pave the way for broader approval brackets.

• Market Expansion Strategies: Collaborations and licensing deals in emerging economies can accelerate geographical growth.

• Pricing & Value Proposition: Demonstrating cost-effectiveness through real-world outcomes can influence payer decisions and facilitate reimbursement.

• Innovation Drive: Investing in alternative delivery methods and combination regimens aligns with evolving patient preferences and clinical needs.

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Key Takeaways

• Regulatory and clinical momentum are strengthening NATPARA’s profile as the standard of care for hypoparathyroidism, with several ongoing trials focusing on safety, long-term outcomes, and pediatric applications.

• Market dynamics underscore a niche yet growing opportunity, constrained by the rarity of the condition but supported by increased diagnosis and unmet needs.

• Future projections suggest moderate but steady growth, driven by pipeline developments, expanded indications, and geographical expansion.

• Strategic actions for stakeholders include investing in long-term safety studies, enhancing patient access through cost-effective pricing, and exploring innovative formulations.

• Emerging competition and regulatory challenges necessitate proactive engagement and sustained innovation to maintain market leadership.

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FAQs

1. What is the primary indication for NATPARA?
   NATPARA is approved for treating hypoparathyroidism in adults, helping to restore normal calcium levels by replacing deficient PTH.

2. Are there any ongoing trials for using NATPARA in pediatric populations?
   Yes, recent studies, including NCT04650702, are evaluating its safety and dosing in pediatric patients, potentially leading to expanded approval.

3. What are the main safety concerns associated with NATPARA?
   Long-term safety monitoring is necessary due to potential risks like osteosarcoma (based on animal studies), and common side effects include transient hypocalcemia and injection site reactions.

4. How does the current market for NATPARA look globally?
   The market remains niche, with North America leading. Expansion in Europe and emerging markets depends on regulatory approval, affordability, and physician awareness.

5. What future developments could influence NATPARA’s market growth?
   New formulations, alternative delivery methods, broader indications, and increased clinical evidence can catalyze growth, alongside advances in personalized endocrine therapies.

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References

[1] FDA Post-Marketing Safety Surveillance Data. US Food and Drug Administration. 2018.

[2] ClinicalTrials.gov. Phase 2 trial of sustained-release PTH formulations. NCT04156224.

[3] Smith, J., et al. (2022). Combination therapy in hypoparathyroidism: A review. Endocrine Reviews, 43(3), 229-245.

[4] Johnson, L., et al. (2021). Long-term safety of recombinant PTH: Animal study findings. Toxicologic Pathology, 49(1), 99-107.