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Acromegaly is an acquired disorder characterized by excessive growth hormone (GH) production, resulting in progressive somatic disfigurement affecting primarily the face and extremities alongside systemic manifestations. The condition is registered in the GARD (GARD:5725) and Orphanet (Orphanet:963) rare disease databases, with an estimated prevalence of 1–9 per 100,000 individuals. Acromegaly Community, Inc. provides patient advocacy resources for the acromegaly community. A patient assistance program, the Acromegaly Fund through HealthWell Foundation, is documented in the packet with closed enrollment status.
Clinical feature data, including specific symptom frequencies and phenotypic distributions, are not certified in the current packet. The packet-certified definition describes progressive somatic disfigurement of the face and extremities alongside systemic manifestations associated with GH excess.
No inherited causative genetic variants or germline inheritance patterns are documented for acromegaly in the current packet. The disease is classified as an acquired disorder linked to excessive GH production. No OMIM identifier is on record in this packet.
Diagnostic criteria and methods for acromegaly are not certified in the current packet. The disease definition characterizes the condition as an acquired disorder related to excessive GH production with somatic and systemic manifestations.
Multiple FDA-approved pharmacologic agents are documented for acromegaly with active market status. Somatostatin analogs include octreotide acetate in formulations approved across multiple decades: Sandostatin (approved 1988), Sandostatin LAR Depot (approved 1998), and Bynfezia Pen (approved 2024). Oral octreotide (Mycapssa) was approved in 2020. Pasireotide, a multireceptor somatostatin analog, is approved as Signifor (2012) and Signifor LAR (2014). Pegvisomant (Somavert), a GH receptor antagonist, has been FDA-approved since 2003. Paltusotine (Palsonify), an oral nonpeptide somatostatin receptor agonist, received FDA approval in September 2025, representing the most recently approved agent in the packet. Multiple additional agents carry orphan drug designation status for acromegaly.
26 trials found
Prognosis data and natural history metrics for acromegaly are not certified in the current packet beyond the characterization of progressive somatic disfigurement in the disease definition.
Acromegaly has 265 classified publications documented, with review and meta-analysis articles as the dominant research type (96 of 265), alongside biomarker and recent trial publications. Active acromegaly-specific clinical trials include a Phase 3 study of Debio 4126 in participants previously treated with somatostatin analogs (NCT06930625, Debiopharm, recruiting), a Phase 2 study of ALXN2420 combined with somatostatin analogs (NCT07037420, Alexion, recruiting), a Phase 2 study of MAR002 (NCT07641179), and a Phase 3 octreotide long-acting injection study (NCT07623824).
Data assembled from 5 of 12 sources · Last updated Oct 3, 2026, 3:54 AM UTC
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Patient Advocacy Groups (PAGs) provide support, resources, and community for patients and caregivers.
European rare disease database
Genetic and Rare Diseases Info Center
AI-curated news mentioning acromegaly
Updated Aug 7, 2026
Researchers have developed a long-acting allosteric antagonist targeting the growth hormone receptor for the treatment of acromegaly. This advancement could enhance treatment options for patients suffering from this rare endocrine disorder.
A cross-sectional study evaluates nurses' knowledge and awareness of acromegaly in a high-volume tertiary center. The findings may inform training programs to enhance care for patients with this rare disease.
A recent study demonstrates that calcaneal quantitative ultrasound parameters have a strong correlation with DXA measurements in patients with acromegaly. This finding could enhance diagnostic accuracy and monitoring for this rare disease.
AstraZeneca has discontinued development on four pipeline assets, including one for acromegaly, while advancing a bispecific antibody targeting the EGFR protein. This strategic shift reflects a focus on enhancing their cancer portfolio.