Somavert (pegvisomant) / Pfizer - LARVOL DELTA

Case Report: Should IGF-1R targeted therapy be revisited in Ewing sarcoma? a report of long-term complete response and review of the literature. (PubMed, Front Oncol) - P1, P1/2 | "We present the case of a 42-year-old female with recurrent ES with pulmonary metastases who, after progressing on anti-IGF-1R monotherapy with figitumumab (CP-751,871, NCT00560235), achieved complete remission in a phase I clinical trial (NCT00976508) that combined figitumumab IGF-1R-inhibition with growth hormone receptor antagonist pegvisomant. The impressive response observed highlights the clinical synergy of this combination which warrants further clinical exploration as well as the potential of IGF-1R inhibition for ES. Additionally, this case suggests that targeted therapy discontinuation might be an option for select patients with long-term complete remission."

Journal • Ewing Sarcoma • Oncology • Sarcoma • Solid Tumor • IGF1

Indirect Treatment Comparison of Biochemical Control Between Pasireotide Long-Acting Release and Pegvisomant as Second-Line Therapies for Acromegaly, Based on Real-World Evidence (ISPOR-EU 2025) - "This meta-analysis of RWE suggests similar efficacy in normalizing IGF-1 levels between PAS LAR and PEG in the second-line treatment of acromegaly. Further ITCs are planned to assess other comparative outcomes in real-world clinical practice, including tumour response, quality of life, treatment safety and discontinuation."

Clinical • HEOR • Real-world • Real-world evidence • Acromegaly • Endocrine Disorders • IGF1 • SSTR

Acromegaly treatment and bone: a bidirectional relationship. (PubMed, Pituitary) - "Recent studies have shown that first- and second-generation somatostatin receptor ligands (SRLs) can reduce the incidence of vertebral fractures (i-VFs). However, a direct effect of these molecules on bone metabolism has not yet been reported. Aims: This review summarizes the results of studies investigating the frequency of i-VFs according to different GH/IGF-I-lowering drugs and the potential effects of these treatments on bone metabolism, as well as preclinical data on potential molecular pathways that interact between GH/IGF-I-lowering drugs and bone metabolism."

Journal • Review • Acromegaly • Endocrine Disorders • Musculoskeletal Diseases • Neuroendocrine Tumor • Oncology • Orthopedics • Rare Diseases • Solid Tumor • IGF1 • SSTR

Pegvisomant in managing comorbidities of acromegaly: a review. (PubMed, Rev Endocr Metab Disord) - No abstract available

Journal • Review • Acromegaly • Endocrine Disorders

Medical treatment in acromegaly: a network meta-analysis. (PubMed, Eur J Endocrinol) - "Our findings suggest pasireotide LAR and pegvisomant as the most effective treatments for IGF-1 normalization. Pasireotide LAR was the best treatment for tumor shrinkage, though the evidence base was limited, requiring cautious interpretation. Their potential role as first-line options after surgery requires further research. Clinical decisions should consider cost, safety and patient-specific parameters to optimize outcomes."

Journal • Retrospective data • Acromegaly • Endocrine Disorders • Oncology • Pituitary Gland Carcinoma • Rare Diseases • IGF1

Safety and Efficacy of Pegvisomant in Children With Growth Hormone Excess (clinicaltrials.gov) - P3 | N=12 | Completed | Sponsor: Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) | Active, not recruiting ➔ Completed | Trial completion date: Dec 2026 ➔ Sep 2025 | Trial primary completion date: Dec 2026 ➔ Sep 2025

Trial completion • Trial completion date • Trial primary completion date • Endocrine Disorders • IGF1

Personalized medicine in acromegaly: insights from the ACROFAST clinical trial. (PubMed, Expert Rev Endocrinol Metab) - "ACROFAST has laid critical groundwork by demonstrating how the combination of predictive biomarkers enhances therapeutic outcomes, improving the rate of biochemical remission and in a shorter follow-up period. In addition, the application of artificial intelligence and machine learning algorithms trained on multiomic and clinical datasets may be useful to improve outcomes and reduce the trial-and-error nature of current pharmacologic management of acromegaly."

Journal • Review • Acromegaly • Endocrine Disorders • Oncology • SSTR

Disease-Specific Mortality in Patients With Acromegaly Treated With Pegvisomant: An ACROSTUDY Analysis. (PubMed, Endocr Connect) - "Younger age (35-50 vs >50 years) at diagnosis (4.50 [1.08-18.83], P=.0394) and sleep apnea (4.98 [1.34-18.53], P=.0168) were associated with higher SMR ratios for respiratory causes. Younger age, higher on-therapy IGF-I and radiotherapy were associated with higher SMR for cardiovascular/cerebrovascular causes, highlighting the importance of achieving IGF-I normalization."

Journal • Acromegaly • Cardiovascular • Endocrine Disorders • Obstructive Sleep Apnea • Oncology • Respiratory Diseases • Sleep Apnea • Sleep Disorder • IGF1

Study of Growth Hormone Inhibition Using Pegvisomant in Severe Insulin Resistance (clinicaltrials.gov) - P2 | N=25 | Recruiting | Sponsor: National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) | Trial completion date: Jan 2026 ➔ Jan 2028 | Trial primary completion date: Jan 2026 ➔ Jan 2028

Trial completion date • Trial primary completion date • Lipodystrophy • Metabolic Disorders • IR

Adherence, duration and healthcare costs in a real-world population of patients with acromegaly. (PubMed, J Comp Eff Res) - "Among these patients, 46.1% (n = 206) were treated with cabergoline as their index treatment, 24.5% (n = 111) with injectable octreotide, 15.0% (n = 68) with lanreotide, 5.5% (n = 25) with bromocriptine, 4.9% (n = 22) with pegvisomant, 2.2% (n = 10) with pasireotide, 1.1% (n = 5) with oral octreotide, 0.4% (n = 2) with cabergoline + octreotide, and 0.2% (n = 1) with cabergoline + lanreotide. However, treatment switching and add-on therapy represent ongoing efforts to optimize patient care toward more effective and tolerable treatments. Expanded treatment options may serve an unmet need in this patient population."

HEOR • Journal • Real-world evidence • Retrospective data • Acromegaly • Endocrine Disorders

Consensus on acromegaly therapeutic outcomes: an update. (PubMed, Nat Rev Endocrinol) - "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."

Journal • Review • Acromegaly • Endocrine Disorders • SSTR

Peptide-based antagonists for the human growth hormone receptor (ACS-Fall 2025) - "In addition, one stapled S1H derivative, SS1HB, showed better GHR antagonism than Pegvisomant. These results indicate that helical S1H peptides may serve as chemical genetics agents to better understand the molecular basis of GH-GHR interactions and offer potential lead compounds for developing next-generation therapeutics to treat GH-related disorders."

Diabetes • Metabolic Disorders

Helical peptide antagonist of the human growth hormone receptor (ACS-Fall 2025) - "One SS1H derivative, SS1HB, inhibited hGH-mediated phosphorylation of STAT5 in cultured UMUC3 cells more effectively than Pegvisomant (Peg), the only FDA-approved antagonist of the hGHR. Taken together, the results from this study show that S1H peptides can be used as powerful chemical genetics agents to explore the molecular mechanisms through which S1H (and its structured derivatives) affect hGH-mediated signaling. Furthermore, we anticipate that structured S1H peptides will serve as potent lead compounds for developing next-generation therapeutics designed to treat hGH-mediated disorders."

Acromegaly • Diabetes • Endocrine Disorders • Metabolic Disorders • STAT5

Growth Hormone Signaling in Bladder Cancer: Transcriptomic Profiling of Patient Samples and In Vitro Evidence of Therapy Resistance via ABC Transporters and EMT Activation. (PubMed, Int J Mol Sci) - "Importantly, these effects were abrogated by Pegvisomant, a GHR antagonist, indicating the functional relevance of GH/GHR signaling in the mediation of these phenotypes. Collectively, our findings support a mechanistic role for GH signaling in driving therapy resistance and tumor aggressiveness in bladder cancer and suggest GHR antagonism as a potential therapeutic strategy to improve treatment outcomes."

Journal • Preclinical • Bladder Cancer • Genito-urinary Cancer • Oncology • Solid Tumor • Urothelial Cancer

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. (PubMed, Endocr Metab Immune Disord Drug Targets) - "This case report highlights the potential benefits of pasireotide in the treatment of refractory headaches, particularly when combined with pegvisomant.  ."

Journal • Acromegaly • Endocrine Disorders • Pain • Pituitary Gland Carcinoma • IGF1 • SSTR

Impact of Medical Therapies on Cardiovascular Risk Factors in Acromegaly. (PubMed, Cureus) - "The growth hormone receptor blocker pegvisomant was associated with a significant post-treatment reduction in both fasting plasma glucose and HbA1c. The long-acting somatostatin receptor ligand pasireotide, on the other hand, was associated with an increase in these glucose parameters...A significant reduction in lipid parameters was also observed in patients treated with somatostatin receptor ligands. However, the impact of these agents on blood pressure control was confounded by concurrent antihypertensive therapies."

Journal • Review • Acromegaly • Cardiovascular • Endocrine Disorders • SSTR

Growth hormone receptor antagonists, GHA2 and GHA3, with dual activity against the human and mouse receptor. (PubMed, Protein Sci) - "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."

Journal • Preclinical • Melanoma • Oncology • Solid Tumor • IGF1

Age-related Immunosenescence In Bgh Mice: Insights From The Spleen And Bone Marrow (ENDO 2025) - "In conclusion, bGH mice showed shifts in the lymphocyte population that are characteristic of premature aging, suggesting that excess GH action accelerates immunosenescence. These findings suggest the potential therapeutic use of the drug that decreases GH action, such as the GH antagonist, Pegvisomant (FDA-approved drug to treat acromegaly), to mitigate immune aging and improve various aspects of healthspan."

Late-breaking abstract • Preclinical • Acromegaly • Endocrine Disorders • Immunology

Growth Hormone Receptor Antagonism Markedly Improves Chemotherapy Efficacy Against Human Lung Cancer (ENDO 2025) - "Additionally, by using a viability assay, we confirmed an increase in IC50 of doxorubicin and cisplatin in LC cells treated with GH and a decrease in IC50 treated with Pegvisomant. Presently, we are validating our observation in a mouse model of LC. In conclusion, our in vitro results confirm that GHR antagonism can drastically sensitize human and mouse LC cells by blocking mechanisms of drug resistance and EMT, thus providing a valuable window for improved efficacy of available anticancer therapies."

Clinical • Late-breaking abstract • Lung Cancer • Melanoma • Non Small Cell Lung Cancer • Oncology • Pancreatic Cancer • Solid Tumor

When Endocrine and Genetic Disorders Collide: Acromegaly with Recurrent Pituitary Macroadenoma and Familial Papillary Thyroid Carcinoma (ENDO 2025) - "Pegvisomant ultimately normalized IGF-1 levels (106 ng/mL), and MRIs showed stable residual tumor tissue...Germline mutations in the AIP gene have been implicated in familial and early-onset acromegaly, warranting genetic evaluation. This case highlights the need for a multidisciplinary approach to optimize management and investigate underlying genetic contributions to tumorigenesis in acromegaly."

Acromegaly • Basal Cell Carcinoma • Colonic Polyps • Colorectal Cancer • Endocrine Cancer • Endocrine Disorders • Erectile Dysfunction • Fatigue • Gastroenterology • Genetic Disorders • Melanoma • Metabolic Disorders • Musculoskeletal Diseases • Non-melanoma Skin Cancer • Oncology • Pain • Rheumatology • Solid Tumor • Squamous Cell Carcinoma • Thyroid Gland Carcinoma • Thyroid Gland Papillary Carcinoma • IGF1

Managing a Resistant GH-Secreting Adenoma: A Case Report Highlighting Barriers to Patient Care (ENDO 2025) - "Post-surgery, Octreotide and Pasireotide were ineffective, the latter discontinued due to hyperglycemia...In such cases, Pegvisomant, a GH antagonist, is preferred, though it has no impact on tumor size...Our patient highlights the impact of low health literacy and language barriers on treatment adherence and outcomes. Physicians must adopt a patient-centered approach that includes clear communication, culturally sensitive education, and the involvement of interpreters or community resources as needed."

Case report • Clinical • Acromegaly • Diabetes • Endocrine Disorders • Metabolic Disorders • Oncology • Pain • Pituitary Gland Carcinoma • IGF1 • SSTR

Development of a Novel Growth Hormone Receptor Antagonist Antibody for the Treatment of Acromegaly (ENDO 2025) - "Stronger inhibitory activity of MAR002 against GHR in vitro, combined with a superior PK/PD profile in cynomolgus monkeys, should provide a prolonged therapeutic effect in patients, requiring less-frequent dosing vs. pegvisomant (e.g. potentially biweekly, monthly, or less frequent). This work enables continued MAR002 development to assess its safety, efficacy, and potential to improve the care for patients with acromegaly."

Acromegaly • Endocrine Disorders • Pituitary Gland Carcinoma • Rare Diseases • IGF1

Disease-Specific Mortality in Patients With Acromegaly Treated With Pegvisomant - An ACROSTUDY Analysis (ENDO 2025) - "Younger attained age [0.93 (0.89,0.96), P2xULN vs. 50 yr, 4.50 (1.08,18.83), P=0.0394] and history of sleep apnea [4.98 (1.34,18.53), P=0.0168] were associated with higher SMR ratios for respiratory causes.In patients with acromegaly treated with pegvisomant, higher on-therapy serum IGF-I levels are associated with higher disease-specific mortality and higher SMR ratios for cardiovascular/cerebrovascular causes, highlighting the importance of achieving IGF-I normalization towards optimizing patient outcomes."

Clinical • Acromegaly • Cardiovascular • Endocrine Disorders • Obstructive Sleep Apnea • Oncology • Respiratory Diseases • Sleep Apnea • Sleep Disorder • IGF1

Cost-utility analysis of second line acromegaly treatment in France. (PubMed, Ann Endocrinol (Paris)) - "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."

HEOR • Journal • Acromegaly • Endocrine Disorders • Oncology • IGF1

Impact of pasireotide on lipid and glucose metabolism in patients with acromegaly: a systematic review and meta-analysis. (PubMed, J Endocrinol Invest) - "In this large meta-analysis, PasiLAR was associated with increased HDL-C, FPG, HbA1c, and frequency of DM in patients with acromegaly. There was no effect on triglycerides, total cholesterol, and LDL-C."

Journal • Retrospective data • Review • Acromegaly • Diabetes • Dyslipidemia • Endocrine Disorders • Metabolic Disorders