Background:
Lipodystrophy (LD) syndromes are a group of rare disorders that affect how a person s body can store and use fat tissue. Many people with LDs become severely insulin resistant. Some people are insulin resistant because of a variant in the insulin receptor gene. Insulin resistance causes many health problems.
Objective:
To learn if blocking the effects of growth hormone in the body will help people with severe insulin resistance.
Eligibility:
Adults aged 18 to 65 years with either a known variant in the insulin receptor gene or with a diagnosis of partial LD.
Design:
Participants will have 2 hospital stays, about 1 month apart. Each stay will be 3 or 4 nights.
During each hospital stay, participants will have many tests. They will have a physical exam with blood tests. They will have all of their urine collected for a 24-hour period. They will have scans to measure their muscle, bone, and fat tissues. They will have tests to measure metabolism and insulin sensitivity. They may have an optional biopsy of fat tissue.
During the first hospital visit, participants will learn how to give themselves shots of a drug (pegvisomant) that blocks growth hormone. The drug is injected under the skin. Participants will continue to give themselves these shots once a day at home.
After the first hospital visit, participants will talk on the phone with members of the study team once each week. After 2 weeks they will have blood drawn for tests.
Participants will stop the shots after the second hospital visit.

Start Date23 Jan 2023

Sponsor / Collaborator

National Institute of Diabetes & Digestive & Kidney Diseases

NCT05131100

/ RecruitingNot Applicable

Korean Post-marketing Surveillance for Somavert

Non-interventional observational study, to identify safety and effectiveness of Somavert during the post-marketing period based on the Korean RMP as required by the regulations of Ministry of Food and Drug Safety (MFDS)

Start Date13 Jul 2022

Sponsor / Collaborator

Pfizer Inc.

CTIS2024-518214-11-01

/ RecruitingPhase 2IIT

InvestiGating the rOle of Growth Hormone in hepatic lipid metabolism in humans

Start Date01 Jan 2022

Sponsor / Collaborator

Medical University of Vienna

100 Clinical Results associated with Pegvisomant

100 Translational Medicine associated with Pegvisomant

100 Patents (Medical) associated with Pegvisomant

424

Literatures (Medical) associated with Pegvisomant

31 Dec 2026·JOURNAL OF MEDICAL ECONOMICS

Cost-effectiveness of pasireotide long-acting release in acromegaly: a systematic literature review and methodology assessment

Review

Author: Kossi, Sika Dédé ; Bronikowska, Małgorzata ; Schmidt, Fabian ; Gilis-Januszewska, Aleksandra ; Gueron, Beatrice ; Binowski, Grzegorz ; Jachimowicz, Michał

BACKGROUND:

Acromegaly is a rare and progressive condition caused by excessive secretion of growth hormone and insulin-like growth factor type 1. Pasireotide long-acting release (LAR) is indicated as a second-line therapy for adults with acromegaly who are unsuitable for or unresponsive to surgery and inadequately controlled with first-generation somatostatin receptor ligands (FGSRLs). Although its efficacy and safety have already been established, its cost-effectiveness remains unclear. The primary objective of this study was to systematically assess the cost-effectiveness of pasireotide LAR compared to other second-line medical options, particularly pegvisomant.

METHODS:

A systematic literature review was conducted in May 2024 in Medline, Medline In Process, Web of Science, and the Centre for Reviews and Dissemination (CRD), York. Studies were eligible if they were full or partial economic analyses of pasireotide LAR as a second-line pharmacological treatment for adult patients with acromegaly. Studies not available in English and publications prior to 2009 were excluded. Included articles were assessed for transparency using the appropriate checklists. Data extraction focused on costs, incremental cost-effectiveness ratios and quality-adjusted life years. Formal data synthesis of outcomes was not undertaken due to the small number of studies and notable heterogeneity between them. Methodological assessment involving the evaluation of model inputs, data sources, and their alignment with the evidence on the course of disease and clinical practice was performed to examine the main factors contributing to discrepancies in cost-effectiveness outcomes and assess the credibility of the results.

RESULTS:

Of 160 records identified, six publications met the inclusion criteria. Their findings demonstrated variability. The critical assessment highlighted considerable variability in methodological rigor among the included studies.

CONCLUSIONS:

Evidence on pasireotide LAR's cost-effectiveness versus pegvisomant regimens remains inconclusive. Although two studies indicated that pegvisomant is more cost-effective, the study with the highest methodological credibility found that pasireotide LAR is a cost-effective alternative.

08 Jan 2026·ENDOCRINOLOGY

Ghrelin-induced food intake in male mice requires peripheral extrahepatic GHR signaling

Article

Author: De Francesco, Pablo N ; dos Santos, Willian O ; Donato, Jose ; Wasinski, Frederick ; Kopchick, John J ; de Souza, Gabriel O ; Barrile, Franco ; Perelló, Mario ; Cornejo, María Paula

Abstract:

Ghrelin acts via the growth hormone secretagogue receptor (GHSR) and increases both food intake and growth hormone (GH) secretion. Studies in mice with genetic manipulations of GH receptor (GHR) revealed that GH action is required for ghrelin's orexigenic effects. However, the biological basis of this interdependence remains unclear. Here, we studied the mechanisms by which GHR contributes to ghrelin-induced hyperphagia in male mice. Transcriptomic analyses of single-cell datasets revealed that Ghr and Ghsr are co-expressed in a small subset of neurons, particularly within the hypothalamic arcuate nucleus (ARH). Systemic ghrelin administration increased food intake, circulating GH, and glycemia but did not induce GHR activation in the brain, as indicated by the absence of pSTAT5 immunoreactivity. Central GH administration failed to enhance ghrelin-induced food intake or glycemia. To evaluate the role of peripheral GHR signaling, we treated mice with the brain-impermeable GHR antagonist pegvisomant. Systemically injected pegvisomant impaired ghrelin's orexigenic effect without affecting its impact on glycemia or hypothalamic c-Fos activation, indicating that peripheral GHR signaling is required for ghrelin-induced hyperphagia. Pegvisomant did not alter refeeding-induced or AgRP neuron-mediated hyperphagia, suggesting a selective blockade of ghrelin's action. Moreover, ghrelin-induced food intake was preserved in hepatocyte-specific GHR knockout mice, despite disrupted hepatic GH signaling. Thus, peripheral, non-hepatic GHR signaling is selectively required for the orexigenic effects of ghrelin. This work reveals a critical GH-dependent, liver-independent mechanism underlying ghrelin-driven feeding, with potential implications for the neuroendocrine regulation of appetite and for therapeutic strategies targeting the ghrelin–GH axis in metabolic diseases.

01 Nov 2025·Nature Reviews Endocrinology

Consensus on acromegaly therapeutic outcomes: an update

Review

Author: Buchfelder, Michael ; Luque, Raúl M ; Melmed, Shlomo ; Luger, Anton ; Petersenn, Stephan ; Colao, Annamaria ; Marazuela, Mónica ; van der Lely, Aart Jan ; Esposito, Daniela ; Frara, Stefano ; Mortini, Pietro ; Ioachimescu, Adriana G ; Giustina, Andrea ; Brue, Thierry ; Beckers, Albert ; Webb, Susan M ; Fleseriu, Maria ; Valassi, Elena ; Shimon, Ilan ; Kadioglu, Pinar ; Tsagarakis, Stylianos ; Ferone, Diego ; Biermasz, Nienke ; Katznelson, Laurence ; Chanson, Philippe ; Salvatori, Roberto ; Chiloiro, Sabrina ; Pirchio, Rosa ; Mercado, Moisés ; Losa, Marco ; Gurnell, Mark ; Greenman, Yona ; Zatelli, Maria Chiara ; Maffei, Pietro ; Bollerslev, Jens ; Kaiser, Ursula B ; Strasburger, Christian J ; Ho, Ken K Y ; Biller, Beverly M K ; Pereira, Alberto M ; Schilbach, Katharina ; Boguszewski, Cesar L ; Casanueva, Felipe F ; Gadelha, Monica R ; Clemmons, David R ; Geer, Eliza B ; Lamberts, Steven W J ; Samson, Susan L ; Neggers, Sebastian ; Puig-Domingo, Manel ; Karavitaki, Niki ; di Filippo, Luigi

The 15th Acromegaly Consensus Conference in September 2023 updated recommendations on therapeutic outcomes for acromegaly. Since the publication of medical management guidelines in 2018, new pharmacological agents and new treatment approaches have been developed. Fifty-two experts in the management of acromegaly reviewed the current literature and assessed changes in drug approvals, clinical practice standards and management. Current outcome goals were considered, with a focus on the effect of current and emerging somatostatin receptor ligands, the growth hormone receptor antagonist pegvisomant and the dopamine agonist cabergoline on biochemical control, clinical control, adenoma mass and surgical outcomes. Participants assessed factors that determine pharmacological choices, as well as the proposed use of each agent. Here, we present consensus recommendations highlighting how an evidence-based acromegaly management algorithm could be optimized in clinical practice.

News (Medical) associated with Pegvisomant

14 Jul 2025

·pipelinereview.com

Marea Therapeutics Presents Preclinical Data on Novel Growth Hormone Receptor Antagonist Antibody, MAR002, for the Treatment of Acromegaly at ENDO 2025

SOUTH SAN FRANCISCO, CA, USA I July 14, 2025 I Marea Therapeutics, Inc., a clinical-stage biotechnology company harnessing the latest advances in human genetics to develop first-in-class, next-generation medicines for cardioendocrine diseases, today announced the presentation of preclinical data on MAR002, a novel growth hormone receptor (GHR) antagonist antibody for the treatment of acromegaly, at the 2025 Annual Meeting of the Endocrine Society (ENDO). The oral presentation, titled “Development of a Novel Growth Hormone Receptor Antagonist Antibody for the Treatment of Acromegaly,” highlights the significant potential of MAR002 to address critical unmet needs in patients suffering from this rare and highly morbid disease. Presentation highlights include: Marea Therapeutics has completed a GLP-toxicology study to assess MAR002’s safety. The preclinical pharmacology data strongly support advancing MAR002 into clinical development; a Phase 1 first-in-human study is expected to begin in the third quarter of 2025. Marea’s development strategy aims for rapid advancement into acromegaly patients after achieving proof of mechanism in healthy volunteers, with a clear path to approval guided by FDA guidance for IGF-1 normalization. “The preclinical data presented at ENDO 2025 underscore the potential of MAR002 to meaningfully transform the treatment landscape for acromegaly,” said Ethan Weiss, M.D., chief scientific officer of Marea Therapeutics. “With its strong potency, enhanced durability, and favorable developability profile, MAR002 is uniquely positioned to overcome the limitations of current therapies and offer a more effective, targeted approach for patients. We are excited to advance MAR002 into clinical development and bring forward a next generation and potential best-in-class GHR antagonist.” Marea Therapeutics believes MAR002 represents a significant opportunity to replace and expand the market for existing growth hormone receptor antagonists (GHRAs) and could also be used in combination with somatostatin receptor ligands (SRLs), offering a more effective and convenient treatment option for patients with acromegaly. About MAR002 MAR002 is a novel, potent and selective half-life-extended, allosteric, human monoclonal GHRA antibody being developed for the treatment of acromegaly. The PK and PD properties of MAR002 are predictable and typical of a half-life extended human antibody, showing a long duration of action compatible with infrequent subcutaneous dose administration in humans. These characteristics support its potential to offer an effective and convenient treatment for patients with acromegaly. About Acromegaly Acromegaly is an orphan disease characterized by the excess secretion of growth hormone (GH) from a benign pituitary adenoma. Acromegaly affects approximately 30,000 patients in the U.S. If left untreated, acromegaly is highly morbid, leading to significant comorbidities such as GH-induced insulin resistance and diabetes, and serious cardiovascular pathology. The median lifespan of patients can be shortened by 10 years without effective therapy, and incomplete IGF-1 normalization is associated with increased mortality. Despite its severity, acromegaly is often under or misdiagnosed, with an average time from symptom onset to diagnosis of approximately eight years. The current treatment paradigm for acromegaly often involves surgery, performed in over 90% of patients, which achieves remission in about 50% of cases, though this can degrade over time. Medical therapy is required for approximately 65% (around 20,000 in the U.S.) of patients during their disease journey, regardless of their surgical history. Current medical treatments include somatostatin receptor ligands (SRLs) and growth hormone receptor antagonists (GHRAs), such as pegvisomant. However, many patients do not achieve biochemical control with existing therapies. About Marea Therapeutics Marea Therapeutics is a clinical-stage biotechnology company harnessing the latest advances in human genetics to develop first-in-class, next-generation medicines for cardioendocrine diseases. The company’s lead therapy, MAR001, is in Phase 2 clinical development for adults with metabolic dysfunction and high risk for atherosclerotic cardiovascular disease. The company is also advancing MAR002 for the treatment of acromegaly. To learn more, please visit www.mareatx.com and follow us on LinkedIn and X . SOURCE: Marea Therapeutics

Phase 2Orphan DrugPhase 1

19 Mar 2024

·www.biospace.com

Crinetics Bags Second Phase III Win for Acromegaly, Heads for NDA Submission

Pictured: Blood sample for insulin-like growth factor-1 (IGF-1)/iStock, Jarun011 Crinetics Pharmaceuticals has secured another late-stage win as its oral, once-daily investigational drug paltusotine aced a second Phase III study for acromegaly, a rare hormonal disorder. The biotech’s stock shot up over 10% Tuesday morning following the announcement. In the late-stage trial, 56% of patients taking paltusotine achieved the primary endpoint for the insulin-like growth factor 1 (IGF-1) level compared to 5% on placebo. The treatment also helped reduce symptoms of the disease—a secondary endpoint of the study. No serious adverse events were reported. Acromegaly, sometimes referred to as gigantism, is a serious hormonal disorder caused by a benign tumor in the pituitary gland that secretes growth hormone. Over 25,000 patients in the U.S. suffer from the rare disease, which results in enlargement of face, hands and feet. Left untreated, it can cause type 2 diabetes, hypertension, heart disease and other serious complications. Surgical removal of the tumor is the preferred treatment but many patients are not candidates for surgery. Current pharmacological options include painful monthly injections or Pfizer’s subcutaneous daily injection of Somavert. The Phase III data are the second positive trial results for Crinetics in acromegaly. In September 2023, the biotech shared data for a study of paltusotine in patients who switched from their current injections to Crinetics’ pill. Among treated patients, 83% were able to maintain their IGF-1 levels within the upper limit of normal, compared to only 4% of those on placebo. With these data in hand, Crinetics plans to submit its New Drug Application to the FDA in the second half of this year, while prepping for a 2025 launch. Jefferies analysts believe the market opportunity could be around $300 million for the company. Crinetics is also testing the drug candidate in carcinoid syndrome, reporting positive Phase II topline results last week. Kate Goodwin is a freelance life science writer based in Des Moines, Iowa. She can be reached at kate.goodwin@biospace.com and on LinkedIn.

Phase 3Clinical ResultPhase 2NDA

19 Mar 2024

·www.biospace.com

Crinetics Bags Second Phase III Win for Acromegaly, Heads for NDA Submission

Pictured: Blood sample for insulin-like growth fac The biotech Tuesday reported that 56% of patients taking paltusotine in a late-stage trial achieved the primary endpoint of IGF-1 levels for their rare hormonal disorder. An NDA submission is slated for later this year with a launch anticipated in 2025. Pictured: Blood sample for insulin-like growth factor-1 (IGF-1)/iStock, Jarun011 Crinetics Pharmaceuticals has secured another late-stage win as its oral, once-daily investigational drug paltusotine aced a second Phase III study for acromegaly, a rare hormonal disorder. The biotech’s stock shot up over 10% Tuesday morning following the announcement . In the late-stage trial, 56% of patients taking paltusotine achieved the primary endpoint for the insulin-like growth factor 1 (IGF-1) level compared to 5% on placebo. The treatment also helped reduce symptoms of the disease—a secondary endpoint of the study. No serious adverse events were reported. Acromegaly, sometimes referred to as gigantism, is a serious hormonal disorder caused by a benign tumor in the pituitary gland that secretes growth hormone. Over 25,000 patients in the U.S. suffer from the rare disease, which results in enlargement of face, hands and feet. Left untreated, it can cause type 2 diabetes, hypertension, heart disease and other serious complications. Surgical removal of the tumor is the preferred treatment but many patients are not candidates for surgery. Current pharmacological options include painful monthly injections or Pfizer’s subcutaneous daily injection of Somavert. The Phase III data are the second positive trial results for Crinetics in acromegaly. In September 2023, the biotech shared data for a study of paltusotine in patients who switched from their current injections to Crinetics’ pill. Among treated patients, 83% were able to maintain their IGF-1 levels within the upper limit of normal, compared to only 4% of those on placebo. With these data in hand, Crinetics plans to submit its New Drug Application to the FDA in the second half of this year, while prepping for a 2025 launch. Jefferies analysts believe the market opportunity could be around $300 million for the company. Crinetics is also testing the drug candidate in carcinoid syndrome, reporting positive Phase II topline results last week. Kate Goodwin is a freelance life science writer based in Des Moines, Iowa. She can be reached at kate.goodwin@biospace.com and on LinkedIn .

Clinical ResultPhase 3Phase 2NDA

100 Deals associated with Pegvisomant

External Link

KEGG	Wiki	ATC	Drug Bank
D05394	Pegvisomant	H01AX01	DB00082

R&D Status

Approved

10 top approved records.

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Indication	Country/Location	Organization	Date
Acromegaly	European Union	Pfizer Europe MA EEIG	12 Nov 2002
Acromegaly	Iceland	Pfizer Europe MA EEIG	12 Nov 2002
Acromegaly	Liechtenstein	Pfizer Europe MA EEIG	12 Nov 2002
Acromegaly	Norway	Pfizer Europe MA EEIG	12 Nov 2002

Developing

10 top R&D records.

to view more data

Indication	Highest Phase	Country/Location	Organization	Date
Insulin Resistance	Phase 3	Italy	Pfizer SRL	04 Dec 2015
Albuminuria	Phase 2	United Kingdom	Pfizer Ltd. (India)	04 Sep 2004
Diabetic Nephropathies	Phase 2	United Kingdom	Pfizer Ltd. (India)	04 Sep 2004
Breast Cancer	Phase 1	United States	Pfizer Inc.	01 Nov 2009
Breast Cancer	Phase 1	Canada	Pfizer Inc.	01 Nov 2009
Breast Cancer	Phase 1	Finland	Pfizer Inc.	01 Nov 2009
Breast Cancer	Phase 1	Germany	Pfizer Inc.	01 Nov 2009
Colorectal Cancer	Phase 1	United States	Pfizer Inc.	01 Nov 2009
Colorectal Cancer	Phase 1	Canada	Pfizer Inc.	01 Nov 2009
Colorectal Cancer	Phase 1	Finland	Pfizer Inc.	01 Nov 2009

Clinical Result

Indication

Phase

Evaluation

View All Results

Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT01538966 (CTgov)	Not Applicable	Acromegaly	76	Octreotide LAR+Lanreotide+Pegvisomant (High Dose SRL + Weekly Pegvisomant)	zgswqmgwyf(ypinbunerg) = aiybikbzqu zplxgceynq (cbknzximek, 1,645.49) View more	-	04 Jan 2023
NCT01538966 (CTgov)	Not Applicable	Acromegaly	76	Octreotide LAR+Lanreotide+Pegvisomant (Low Dose SRL + Daily Pegvisomant)	zgswqmgwyf(ypinbunerg) = riqdoyalba zplxgceynq (cbknzximek, 11,158.49) View more	-	04 Jan 2023
ENDO2020	Not Applicable	Acromegaly blood glucose \| serum IGF-I levels \| hemoglobin A1c	72	Pegvisomant	rksrzdmaoz(ubqqnfrgiv) = vltwibucxk qaeaehgtdt (rgrdhtltzi ) View more	-	08 May 2020
ENDO2020	Not Applicable		72	Pasireotide LAR	rksrzdmaoz(ubqqnfrgiv) = iomlqhqwgk qaeaehgtdt (rgrdhtltzi )	-	08 May 2020
SUN-LB079 (ENDO2019)	Not Applicable	Acromegaly	2,221	Pegvisomant	awgacrwvom(cekvfxlcdo) = vrhqnyaohe kzestkttom (paybjlqjsn ) View more	-	30 Apr 2019
ACROSTUDY (ENDO2019)	Not Applicable	Acromegaly	110	Pegvisomant	ztrygecaqw(uxmgydlncy) = hiushwbbap hbfavkxznb (owmpifcyqi, 5.4 - 6.1)	-	30 Apr 2019
NCT01261000 (CTgov)	Not Applicable	Acromegaly	8	Pegvisomant	uuilmnaegl(jpaimjsgqz) = fmwydwzust wnurkgyfuj (fpjvrqinrc, 107.33) View more	-	22 Aug 2017
NCT02023918 (PEGIR, CTgov)	Phase 2	Metabolic Syndrome \| Prediabetic State	6	pegvisomant	sgculkjpcm(pvfojnnpld) = xrocrfzxqt gvemilqhoh (jeyblrszml, 1.46) View more	-	13 Mar 2017
NCT00976508 (CTgov)	Phase 1	Sarcoma \| Lung Cancer \| Advanced Malignant Solid Neoplasm ... View more	24	Figitumumab+Pegvisomant (Figitumumab 20mg/kg + Pegvisomant 10 mg)	diggqejlzj = dtuslxmygl vvwcjlhzav (xsdyfonzas, ybnekjmljg - qrlelftxce) View more	-	13 Dec 2013
NCT00976508 (CTgov)	Phase 1		24	Figitumumab+Pegvisomant (Figitumumab 20mg/kg + Pegvisomant 20 mg)	diggqejlzj = igqzmlnzek vvwcjlhzav (xsdyfonzas, mnvupypcad - ncuyqfcflo) View more	-	13 Dec 2013
NCT00969644 (Pubmed \| Andrology)	Not Applicable	-	8	GH treatment	xriedhfeaa(oyeecqpzum) = rwmtgyehzw znvmhqotzz (uoioizsugq ) View more	-	01 Jul 2013
NCT01278342 (HOSCAR, CTgov)	Phase 4	Acromegaly	70	Sandostatin LAR (Sandostatin LAR High Dose Alone)	geihhfdach = ohusxgvyzd ztiprhpasg (kvznnlqdxj, wltdcnriiy - agrtuqlwwl) View more	-	17 Feb 2011
NCT01278342 (HOSCAR, CTgov)	Phase 4	Acromegaly	70	Sandostatin LAR+pegvisomant (Sandostatin LAR High Dose + Pegvisomat)	geihhfdach = wiprlfopab ztiprhpasg (kvznnlqdxj, khmynwuxtt - qvanszglsr) View more	-	17 Feb 2011

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