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.

NCT04998500

/ Unknown statusNot ApplicableIIT

Growth Hormone as a Model for Reversible Activation of Adipose Tissue Fibrosis

Background: Adipose tissue fibrosis denotes excessive pathological accumulation of extracellular matrix (ECM) in adipose tissue and is a marker of dysfunction. Growth hormone (GH) activates adipose tissue lipolysis and stimulates collagen synthesis in lean tissues. Intriguingly, we have novel pilot data to suggest that GH excess (acromegaly) also induces reversible fibrosis in vivo and potently activates the expression of fibroblast activation protein alpha (FAPα).
Hypothesis: GH induces adipose tissue fibrosis by increased FAPα expression together with proliferation and fibrogenic differentiation of fibro-adipogenic progenitor (FAP) cells.
Aim: To unravel the mechanisms underlying GH-induced adipose tissue fibrosis with emphasis on FAPα expression and proliferation of FAP cells.
Subjects and methods: In a single blinded, randomized, double-dummy crossover design, 10 adult, moderately overweight individuals will be subjected to one week of GH and GH receptor blockade (Pegvisomant). We will use single-cell technologies, fluorescence-activated cell sorting (FACS), RNA sequencing, and cell culture studies on adipose tissue samples, combined with in vivo assessment of adipose tissue turnover and metabolism.
Perspectives: Understanding fibrosis formation in human models may identify new targets for treatment of obesity-associated disorders.

Start Date01 Aug 2021

Sponsor / Collaborator

University of Aarhus

[+1]

100 Clinical Results associated with Pegvisomant

100 Translational Medicine associated with Pegvisomant

100 Patents (Medical) associated with Pegvisomant

587

Literatures (Medical) associated with Pegvisomant

01 Sep 2025·ANNALES D ENDOCRINOLOGIE

Cost-utility analysis of second line acromegaly treatment in France

Article

Author: Ferriere, Amandine ; Chanson, Philippe ; Beillat, Maud ; Briet, Claire ; Delemer, Brigitte ; Drui, Delphine ; Cabout, Elise ; Brue, Thierry ; Raverot, Gérald ; Aissani, Wassila ; Ben Romdhane, Haifa

OBJECTIVE:

This updated French cost-utility analysis aimed to assess the efficacy of the second-line pharmacological treatment - pegvisomant, pasireotide or pegvisomant combined with first generation somatostatin analogues (FGSA) - considering first-line treatment (surgery, FGSA, cabergoline and combination), radiotherapy and the impact of treatment on tumor volume.

METHODS:

The original three-state Markov model was revised to include an additional health state, representing patients controlled without pharmacological treatment following successful radiotherapy. The model also accounted for the history of first-line treatments as additional costs upon patients' entry. A cohort of 1000 simulated patients was followed over a lifetime horizon from a collective perspective. Treatment efficacy is defined on normalization of insulin growth factor-1 and was determined through a network meta-analysis. Cost and utility data were sourced from French databases and literature.

RESULTS:

All the evaluated treatments were included in an efficiency frontier. The incremental cost-utility ratio (ICUR) of pegvisomant compared to pasireotide was 27,805€ per quality-adjusted life year (QALY) gained. The ICUR of pegvisomant combined with FGSA compared to pegvisomant was 253,854€/QALY. Sensitivity analyses showed the robustness of the results. The addition of radiotherapy has shown improved quality of life and lower costs for all second-line treatments.

CONCLUSION:

This model explores the cost-effectiveness of second-line pharmacological treatments of acromegaly accounting for the overall management recommended in France. Radiotherapy appears as a key therapeutic option allowing long-term remission of patients even after pharmacological treatment failure. This study is an additional tool for treatment choice including both a clinical and economic perspective.

SIGNIFICANCE STATEMENT:

The aim of this study is to assess the cost-utility of second-line treatments of acromegaly accounting for the overall management recommended in France, considering surgery, first-line treatments, radiotherapy and the impact of treatments on tumor volume, in addition to IGF-1 normalization. All evaluated treatments were included in an efficiency frontier. This addition has shown that radiotherapy and the impact on tumor volume are decisive in the management of second-line treatments for acromegaly. Indeed, radiotherapy enables patients to achieve complete remission, i.e. without the need for drug treatment. This is an important factor for patients, given that acromegaly treatments are chronic and usually lifelong. This study is an additional tool for treatment choice including both a clinical and economic perspective.

01 Aug 2025·PROTEIN SCIENCE

Growth hormone receptor antagonists, GHA2 and GHA3, with dual activity against the human and mouse receptor.

Article

Author: Perry, Jo K ; Harms, Julia ; Wang, Yue ; Jamieson, Stephen M F ; Langley, Ries J ; Portlock, Theo

Aberrant human growth hormone (GH) expression has been increasingly linked to poor outcomes in certain cancers and reduced health span. GH receptor (GHR) signal transduction activates signaling pathways that promote cell proliferation and survival, contributing to tumor progression. Consequently, there has been growing interest in creating antagonists that target GH signaling for anticancer applications. Pegvisomant is the only clinically available GHR antagonist (GHA). It is a protein antagonist that consists of a mutated GH protein (B2036) conjugated to polyethylene glycol (PEG). While pegvisomant is a potent human GHR antagonist, it has lower efficacy in mice, necessitating the use of higher doses in preclinical models. Here, we describe two antagonists, GHA2 and GHA3, based on an earlier reported antagonist, B2024, that have improved efficacy in mice. Recombinant GHA2 and GHA3 were produced in E. coli and then conjugated to multiple 5 kDa amine-reactive mPEG (GHA2-PEG5K) or site-specifically PEGylated with 40 kDa mPEG at amino acid site 144 (GHA3-PEG40K). When compared with B2036, GHA2 and GHA3 had significantly improved in vitro activity against the mouse GHR in a Ba/F3 cell viability assay, and GHA2 more effectively reduced GH-dependent signal transduction in the mouse melanoma cell line, B16-F10. A single dose of 30 mg/kg of amine-PEGylated GHA2-PEG5K or site-specific conjugate, GHA3-PEG40K, reduced serum IGF-1 in mice at 24 h by 40.7% and 45.8%, respectively. In contrast, amine-PEGylated B2036-PEG5K did not significantly reduce serum IGF-I levels. In conclusion, we demonstrate here that GHA2-PEG5K and GHA3-PEG40K are more potent inhibitors than B2036-PEG5K in mouse models.

18 Jul 2025·Endocrine Metabolic & Immune Disorders-Drug Targets

The Combination Therapy of Pasireotide LAR Plus Pegvisomant and Cabergoline Relieved Acromegaly-Related Headache in a Female Patient with AIP-Mutated Acromegaly: A Case Report

Article

Author: Doglietto, Francesco ; De Marinis, Laura ; Giampietro, Antonella ; Bianchi, Antonio ; Mattogno, Pier Paolo ; Chiloiro, Sabrina ; Giambò, Penelope ; Lauretti, Liverana ; Pontecorvi, Alfredo

Introduction::

Acromegaly is a rare condition characterized by excessive exposure to GH and IGF-1 due to a pituitary adenoma in most cases. The disease is associated with numerous symptoms, including headaches, which are frequently difficult to manage.

Case presentation::

We report the clinical history of an 11-year-old female patient with an AIP mutation and acromegaly, who was unresponsive to treatment with somatostatin receptor ligands and diagnosed with chronic, disabling headaches resistant to multiple treatments, such as indomethacin, cyclooxygenase-2 inhibitors, gabapentin, melatonin, topiramate, galcanezumab, and verapamil. The patient achieved biochemical control of acromegaly with a combination therapy using lanreotide autogel (Lanreotide ATG) and pegvisomant. However, due to persistent and disabling headaches, lanreotide ATG was discontinued, and combination therapy with pasireotide long-acting release (Pasireotide LAR) and pegvisomant was initiated, resulting in the resolution of the headache, with sustained improvement over time.

Conclusion::

This case report highlights the potential benefits of pasireotide in the treatment of refractory headaches, particularly when combined with pegvisomant.

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.

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Indication	Highest Phase	Country/Location	Organization	Date
Advanced Malignant Solid Neoplasm	Phase 1	United States	Pfizer Inc.	01 Nov 2009
Advanced Malignant Solid Neoplasm	Phase 1	Canada	Pfizer Inc.	01 Nov 2009
Advanced Malignant Solid Neoplasm	Phase 1	Finland	Pfizer Inc.	01 Nov 2009
Advanced Malignant Solid Neoplasm	Phase 1	Germany	Pfizer Inc.	01 Nov 2009
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

Clinical Result

Indication

Phase

Evaluation

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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)	ycvvbrckez(dfazyetyds) = zbtnikaisd aoxgujfobu (rrkavtpfov, 1,645.49) View more	-	04 Jan 2023
NCT01538966 (CTgov)	Not Applicable	Acromegaly	76	Octreotide LAR+Lanreotide+Pegvisomant (Low Dose SRL + Daily Pegvisomant)	ycvvbrckez(dfazyetyds) = zyfkrmrejm aoxgujfobu (rrkavtpfov, 11,158.49) View more	-	04 Jan 2023
MON-311 (ENDO2020)	Not Applicable	Acromegaly blood glucose \| serum IGF-I levels \| hemoglobin A1c	72	Pegvisomant	tgngdqivdy(npaiwerqhx) = wyxejfoywe gxvwgiepvk (dntuthtqlr ) View more	-	08 May 2020
MON-311 (ENDO2020)	Not Applicable		72	Pasireotide LAR	tgngdqivdy(npaiwerqhx) = huuazkzicw gxvwgiepvk (dntuthtqlr )	-	08 May 2020
SUN-LB079 (ENDO2019)	Not Applicable	Acromegaly	2,221	Pegvisomant	pokavwtatc(dplxvavzst) = airyjsufsx cxupybxcxc (wjysssvctd ) View more	-	30 Apr 2019
ACROSTUDY (ENDO2019)	Not Applicable	Acromegaly	110	Pegvisomant	rcqftghitf(tolezvpine) = lpgbbhbjby slfmgfegpo (zdfjbbgazw, 5.4 - 6.1)	-	30 Apr 2019
NCT01261000 (CTgov)	Not Applicable	Acromegaly	8	Pegvisomant	qrohgacifs(fdvjyrvzhd) = ppwhwrytlt tcmebogxfi (xdbuztnvds, 107.33) View more	-	22 Aug 2017
NCT02023918 (PEGIR, CTgov)	Phase 2	Metabolic Syndrome \| Prediabetic State	6	pegvisomant	vxkmymmlbt(xyzozaunej) = quhyqglqqd wsythhgqea (bdmkfmxqip, 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)	kbiveguzyb = xydaatiepx zngkjqcbog (fzuixwwysm, evwxqeqmyz - aleberjobf) View more	-	13 Dec 2013
NCT00976508 (CTgov)	Phase 1		24	Figitumumab+Pegvisomant (Figitumumab 20mg/kg + Pegvisomant 20 mg)	kbiveguzyb = uqaufyyugz zngkjqcbog (fzuixwwysm, ematqmbion - fjuamrdaeg) View more	-	13 Dec 2013
NCT00969644 (Pubmed \| Andrology)	Not Applicable	-	8	GH treatment	elqyfikblx(pimacezzwl) = revzjfkamt rewlvlwpux (vhtgiahkzb ) View more	-	01 Jul 2013
NCT01278342 (HOSCAR, CTgov)	Phase 4	Acromegaly	70	Sandostatin LAR (Sandostatin LAR High Dose Alone)	ioguyaabre = mtnzujjrun lstvumwbav (qgpvrkysjq, jrhwugqrsn - ykykrugruz) View more	-	17 Feb 2011
NCT01278342 (HOSCAR, CTgov)	Phase 4	Acromegaly	70	Sandostatin LAR+pegvisomant (Sandostatin LAR High Dose + Pegvisomat)	ioguyaabre = sxfwhnvirz lstvumwbav (qgpvrkysjq, fcypklagaf - opjuyurdup) View more	-	17 Feb 2011

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