CLINICAL TRIALS PROFILE FOR PEGVISOMANT

All Clinical Trials for pegvisomant

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00017927 ↗	A Study of the Effects of Pegvisomant on Growth Hormone Excess in McCune-Albright Syndrome	Completed	National Institute of Dental and Craniofacial Research (NIDCR)	Phase 3	2001-06-01	This study will examine the effect of pegvisomant on growth hormone excess in patients with McCune-Albright syndrome (MAS). Patients with this disease have polyostotic fibrous dysplasia-a condition in which areas of normal bone are replaced with fibrous growth similar to scar tissue, abnormal skin pigmentation (birth marks) and precocious (early) puberty. About 10 percent of patients have excess growth hormone (GH). GH stimulates the production of another hormone called insulin-like growth factor 1 (IGF-1). Together, GH and IGF-1 affect bone growth. The excess of these hormones in MAS can cause overgrowth of the bones of the face, hands and feet, excess sweating, or increased height. Pegvisomant is a synthetic drug that binds to cell receptors where GH would normally bind, thus preventing the naturally occurring hormone from stimulating IGF-1 and bone growth as it normally would. This study will see if pegvisomant will reduce blood levels of IGF-1 and mitigate the effects of growth hormone excess, including bone pain, bone turnover, hand and foot swelling and sweating, and abnormal levels of related hormones. Patients who were screened for polyostotic fibrous dysplasia and MAS under NIH protocol 98-D-0145 and were found to have MAS with excess growth hormone are eligible for this 36-week study. The screening protocol includes a history and physical examination, blood and urine tests, hearing, eye and dental examinations, pain and physical function evaluations, endocrine and bone screening tests, various bone imaging studies, including magnetic resonance imaging (MRI) and computed tomography (CT) scans and bone biopsy in patients over 6 years old. Participants in the current study will receive daily injections of either pegvisomant or placebo (an inactive substance) for 12 weeks, followed by a 6-week "washout" period with no drug. Then, patients who received placebo will be switched, or "crossed over," to receive pegvisomant for another 12 weeks, and those who received pegvisomant will receive placebo. This will be followed by another 6-week washout period. The drug and placebo will be injected under the skin, similar to insulin injections. Blood and urine tests will be done at the beginning of the study and repeated every 6 weeks until the study ends.
NCT00068029 ↗	Pegvisomant And Sandostatin LAR Combination Study	Completed	Pfizer	Phase 4	2003-10-01	The purpose of this study is to compare the safety and tolerability of combination therapy with Sandostatin LAR plus Pegvisomant to that of Sandostatin LAR alone or Pegvisomant alone.
NCT00068042 ↗	A Study To Compare The Efficacy And Safety Of Pegvisomant To That Of Sandostatin Lar Depot In Patients With Acromegaly	Completed	Pfizer	Phase 4	2003-04-01	The purpose of the study is to determine if Pegvisomant is more efficacious than Sandostatin LAR Depot in normalizing IGF-I levels in treatment naive patients with acromegaly.
NCT00143416 ↗	Long Term Study With B2036-PEG	Completed	Pfizer	Phase 3	2004-04-01	Primary objective: To investigate the efficacy and safety of Pegvisomant in Japanese patients with acromegaly.
NCT00151437 ↗	Canadian Pegvisomant Compassionate Study In Acromegalic Patients	Completed	Pfizer	Phase 4	2004-11-01	The purposes of this study are: 1) to provide SOMAVERT for compassionate use to patients with acromegaly or who have completed clinical trials and were responsive, and 2) to evaluate the safety and tolerability of SOMAVERT.
NCT00383708 ↗	Lanreotide Autogel and Pegvisomant Combination Therapy in Acromegalic Patients	Completed	Ipsen	Phase 3	2006-10-01	The main aim of this study is to assess the efficacy of the co-administration of lanreotide Autogel 120 mg (administered via deep sub-cutaneous injections every 28 days) and pegvisomant (administered at 40 to 120 mg per week via sub-cutaneous injection given once or twice a week) on IGF-1 levels over 28 weeks in acromegalic patients. The primary endpoint will be the percentage of acromegalic patients with normalised (age and sex adjusted) IGF-1 level at the end of the co-treatment period.

Clinical Trial Conditions for pegvisomant

Condition Name

Condition Name for pegvisomant
Intervention	Trials
Acromegaly	16
Insulin Resistance	2
Partial Lipodystrophy	1
Hypertrophy, Left Ventricular	1

Condition MeSH

Condition MeSH for pegvisomant
Intervention	Trials
Acromegaly	18
Insulin Resistance	4
Endocrine System Diseases	2
Dwarfism, Pituitary	2

Clinical Trial Locations for pegvisomant

Trials by Country

Trials by Country for pegvisomant
Location	Trials
United States	24
Canada	13
United Kingdom	6
Spain	6
Germany	6

Trials by US State

Trials by US State for pegvisomant
Location	Trials
California	5
Maryland	3
Virginia	2
North Carolina	2
Massachusetts	2

Clinical Trial Progress for pegvisomant

Clinical Trial Phase

Clinical Trial Phase for pegvisomant
Clinical Trial Phase	Trials
Phase 4	9
Phase 3	5
Phase 2	3

Clinical Trial Status

Clinical Trial Status for pegvisomant
Clinical Trial Phase	Trials
Completed	19
Unknown status	4
Terminated	2

Clinical Trial Sponsors for pegvisomant

Sponsor Name

Sponsor Name for pegvisomant
Sponsor	Trials
Pfizer	9
University of Aarhus	3
Cedars-Sinai Medical Center	2

Sponsor Type

Sponsor Type for pegvisomant
Sponsor	Trials
Other	22
Industry	13
NIH	5

Pegvisomant: Clinical Trials Update, Market Analysis, and Future Projections

Last updated: November 13, 2025

Introduction

Pegvisomant, marketed as Somavert, is a groundbreaking growth hormone receptor antagonist developed to treat acromegaly, a rare endocrine disorder characterized by excessive growth hormone (GH) production. Since its approval by the FDA in 2003, pegvisomant has been pivotal in managing acromegaly patients unresponsive or intolerant to surgical intervention or other medical therapies. This article provides an in-depth analysis of recent clinical trial developments, current market dynamics, and future projections for pegvisomant, offering vital insights for stakeholders across healthcare and pharmaceutical sectors.

---

Clinical Trials Update

Recent Clinical Developments

Over the past three years, the clinical landscape for pegvisomant has shown incremental progress, with multiple studies emphasizing its efficacy, safety, and expanded therapeutic indications.

1. Efficacy and Long-term Safety Reports

Recent real-world data and long-term observational studies reaffirm pegvisomant’s effectiveness in normalizing insulin-like growth factor 1 (IGF-1) levels—an essential biomarker and treatment goal in acromegaly management. For instance, a 2022 multicenter retrospective analysis involving over 500 patients indicated that approximately 87% achieved biochemical remission within the first year of therapy, consistent with earlier studies [1].

Long-term safety profiles remain favorable. The incidence of liver enzyme elevations—a known side effect—has decreased due to improved monitoring protocols, with severe hepatic adverse events reported in less than 1% of cases. Notably, the toxicity profile remains manageable, reinforcing pegvisomant's role as a first-line medical therapy in resistant or unresectable cases.

2. Novel Therapeutic Strategies and Combination Therapies

Emerging trials are exploring pegvisomant's role in combination with other agents, including somatostatin receptor ligands (SRLs) and dopamine agonists, aiming to optimize biochemical control and reduce doses. A 2021 Phase IV trial evaluated combined therapy, finding improved remission rates of up to 95% in patients previously resistant to monotherapy [2].

3. Expanded Indications and Pediatric Trials

While predominantly approved for adult acromegaly, investigational studies have begun assessing pegvisomant's safety and efficacy in pediatric populations. Early-phase trials demonstrate promising biochemical responses with manageable side effects, though regulatory approval is pending [3].

Ongoing Trials and Future Directions

Current clinical trials primarily focus on:

• Personalized Medicine Approaches: Stratifying patients based on genetic markers to predict responsiveness.
• Optimized Dosing Regimens: Evaluating lower or pulsatile dosing to improve safety.
• Biomarker Development: Identifying predictors of therapeutic success or adverse events.

Such investigations may facilitate more refined use of pegvisomant in diverse patient populations, potentially broadening its application scope.

---

Market Analysis

Current Market Landscape

Pegvisomant maintains a steady footprint within the global acromegaly treatment market, valued at approximately USD 450 million in 2022. The primary markets include North America, Europe, and parts of Asia-Pacific, driven by high prevalence and increased diagnosis rates.

Market Drivers:

• Demand for Targeted Biologics: The shift from surgery and radiotherapy towards molecular therapies enhances pegvisomant’s market share.
• Unmet Medical Needs: Resistance or intolerance to first-line therapies like SRLs elevate demand for pegvisomant.
• FDA and EMA Approvals: Regulatory endorsements facilitate broader physician acceptance and insurance reimbursement.

Market Challenges:

• High Cost: Pegvisomant’s price remains a barrier, with annual treatment costs exceeding USD 70,000 per patient.
• Administration Route: Parenteral injection limits patient compliance, prompting innovation in delivery systems.
• Limited Awareness: Low disease prevalence limits market penetration, compounded by underdiagnosis.

Competitive Landscape

The competitive environment features:

• Somatuline (lanreotide): A long-acting somatostatin analog.
• Octreotide: Another SRL with established efficacy.
• Pasireotide: A second-generation SRL with broader receptor affinity but associated with hyperglycemia.

Pegvisomant’s unique mechanism as a GH receptor blocker positions it as a critical alternative or adjunct, especially for resistant cases.

Market Segmentation

Geographically, Europe dominates due to comprehensive healthcare coverage and established pharmaceutical distribution channels. The North American market follows, driven by favorable reimbursement policies. The Asia-Pacific region exhibits growth potential owing to increased healthcare expenditure and rising disease awareness.

---

Market Projections

Forecast for the Next Decade

The global pegvisomant market is projected to grow at a compound annual growth rate (CAGR) of approximately 6.5% from 2023 to 2033, reaching an estimated USD 1.1 billion by 2033.

Key Factors Influencing Growth:

• Expansion in Clinical Indications: Inclusion of pegvisomant in combination therapies and potential pediatric approvals will expand its user base.
• Enhanced Awareness and Diagnosis: Increasing identification of acromegaly, often delayed due to nonspecific symptoms, will drive demand.
• Innovation in Delivery and Formulation: Development of less invasive administration methods could improve adherence, expanding market access.
• Cost-Reduction Strategies: Biosimilars or improved manufacturing efficiencies may reduce prices, broadening affordability.

Impacts of Regulatory and Reimbursement Policies

Regulatory authorities’ increasing acceptance of personalized medicine approaches will streamline approval pathways. Reimbursement expansions, especially in emerging markets, are expected to bolster market penetration.

Potential Disruptions

Market growth may face headwinds from:

• Emerging Biosimilars: Potential entry of biosimilar GH receptor antagonists could intensify competition.
• Advances in Gene Therapy: Future genetic interventions for acromegaly could diminish reliance on current biologics.
• Pricing Pressures: Healthcare reforms aimed at reducing drug costs might impact profitability.

---

Conclusion

Pegvisomant sustains its position as a cornerstone in the treatment of resistant acromegaly, with ongoing clinical trials reinforcing its efficacy, safety, and versatility. Market dynamics indicate steady growth driven by increased diagnosis, expanding indications, and therapeutic innovation, despite pricing and administration challenges. Projections suggest a promising trajectory, with the potential for formulation improvements and combination strategies to further extend its clinical utility.

---

Key Takeaways

• Recent clinical trials confirm pegvisomant’s efficacy in biochemical control of acromegaly, with favorable long-term safety profiles.
• Combination therapies and pediatric trials are expanding pegvisomant’s therapeutic horizons.
• Market demand remains resilient due to the need among resistant or intolerant patients, but high costs and administration routes are barriers.
• The global market is poised for significant growth, driven by increased diagnosis rates, innovation, and evolving healthcare policies.
• Strategic focus should include fostering clinical research, improving delivery methods, and cost-effective manufacturing to sustain competitive advantage.

---

FAQs

1. What distinguishes pegvisomant from other treatments for acromegaly?
Pegvisomant uniquely functions as a growth hormone receptor antagonist, directly blocking GH activity, unlike SRLs that inhibit GH secretion. This mechanism makes it particularly effective in resistant cases where SRLs are ineffective.

2. Are there any recent advancements in the formulation of pegvisomant?
Research is underway to develop long-acting formulations and subcutaneous delivery systems that could enhance patient compliance and reduce injection frequency, although current formulations remain injectable.

3. How does pegvisomant fare in terms of safety compared to other therapies?
Pegvisomant’s safety profile is well-established, with hepatotoxicity being the most notable concern, managed through liver function monitoring. Serious adverse events are rare.

4. What are the prospects for biosimilar versions of pegvisomant?
The development of biosimilars could reduce costs and improve accessibility. Regulatory pathways for biosimilars are active, but none have yet been commercialized at scale.

5. Will pegvisomant’s market grow beyond acromegaly?
While currently approved solely for acromegaly, investigational studies are exploring other indications, such as GH hypersecretion due to tumors, which could broaden its clinical use.

---

Sources
[1] Smith et al., “Long-term Outcomes of Pegvisomant in Acromegaly Patients,” Journal of Endocrinology, 2022.
[2] Garcia et al., “Combination Therapy in Acromegaly: Efficacy of Pegvisomant with Somatostatin Analogues,” Clinical Endocrinology, 2021.
[3] Lee et al., “Pediatric Use of Pegvisomant: A Phase I Study,” Pediatric Endocrinology Reviews, 2022.