Drug discovery
21
drugs
With orphan designations
Overview
Acromegaly is a rare hormonal disorder caused by chronic hypersecretion of growth hormone (GH), typically from a pituitary adenoma, leading to progressive somatic enlargement and multisystem complications such as cardiovascular disease, diabetes mellitus, and osteoarthritis. Diagnosis relies on elevated IGF-1 levels and lack of GH suppression during oral glucose tolerance testing, complemented by pituitary MRI. First-line therapy involves transsphenoidal adenoma resection, with adjunctive medical treatments (somatostatin analogs, dopamine agonists, GH receptor antagonists) or radiotherapy for persistent disease [1][5][16][17].
Burden
Physical: 75–90% report arthralgia, cardiovascular/metabolic comorbidities (hypertension, diabetes), and sleep apnea [5][9][17]
Psychosocial: Impaired QoL due to fatigue (92%), depression/anxiety (75%), and social stigma from disfigurement [9][14]
Mortality: Untreated cases face 10-year reduced life expectancy; managed patients approach normal lifespan [11][17]
Therapies
First-line: Endoscopic transsphenoidal surgery (90% cured in microadenomas, <50% in macroaddenomas) [1][6][13]
Medical therapy: Somatostatin analogs (octreotide, lanreotide), dopamine agonists (cabergoline), and GH receptor antagonists (pegvisomant) [3][8][18]
Adjuvant: Radiation therapy for refractory cases; combination therapies improve biochemical control [3][13]
Categories: rare endocrine diseases, rare infertility disorders, rare neoplastic diseases
Drug Discovery Landscape
Drug | Therapy type | Regulator | Orphan designation | Approval | Sponsor |
|---|---|---|---|---|---|
Paltusotine [Palsonify] | small molecules | EMA | 2025-02-26 | 2026-04-27 | Crinetics Pharmaceuticals Europe GmbH |
paltusotine [Palsonify] | small molecules | FDA | 2020-07-07 | 2025-09-25 | Crinetics Pharmaceuticals, Inc. |
octreotide acetate (intranasal) | peptides | FDA | 2018-01-23 | — | Dauntless Pharmaceuticals, Inc. |
H-(4-amino-3-iodo)-D-Phe-c[Cys-(3-iodo)-Tyr-D-Trp-Lys-Val-Cys]-Thr-NH2 | peptides | FDA | 2016-11-03 | — | Amryt Research Ltd. |
Cimdelirsen | oligonucleotides | EMA | 2016-06-27 | — | IQVIA RDS Ireland Limited |
2'-O-(2-methoxyethyl)phosphorothioate antisense oligonucleotide | oligonucleotides | FDA | 2016-05-10 | — | Percheron Therapeutics Limited |
Octreotide acetate [Mycapssa] | peptides | EMA | 2013-08-06 | 2022-12-05 | Amryt Pharmaceuticals Designated Activity Company |
veldoreotide | peptides | FDA | 2013-06-24 | — | Xeris Pharmaceuticals, lnc. |
Cyclo(-gamma-aminobutyryl-L-phenylalanyl-L-tryptophanyl-D-tryptophanyl-L-lysyl-L-threonyl-L phenylalanyl-N-3-carboxypropyl)-glycine amide, acetate salt | peptides | EMA | 2012-12-06 | — | S-cubed Pharmaceutical Services ApS |
Modified growth hormone releasing-hormone fused to the light chain endopeptidase and the N-terminal domain of the heavy chain of botulinum toxin serotype D | proteins | EMA | 2012-01-11 | — | Ipsen Bioinnovation Limited |
octreotide (oral) [MYCAPSSA (formerly Octreolin)] | peptides | FDA | 2010-06-17 | 2020-06-26 | Chiesi USA, Inc. |
octreotide acetate subcutaneous implant | peptides | FDA | 2009-12-07 | — | Endo Pharmaceuticals Solutions, Inc. |
Pasireotide [Signifor] | peptides | EMA | 2009-10-08 | 2014-11-21 | Novartis Europharm Limited |
pasireotide | peptides | FDA | 2009-08-25 | 2014-12-15 | Novartis Pharmaceuticals Corporation |
Octreotide hydrochloride [Oczyesa] | small molecules | EMA | 2009-06-12 | — | Camurus AB |
vapreotide | peptides | FDA | 2003-11-04 | — | H3 Pharma, Inc. |
Pegvisomant [Somavert] | proteins | EMA | 2001-02-14 | — | Pfizer Limited |
Lanreotide [Somatuline Depot] | peptides | FDA | 2000-09-11 | 2007-08-30 | IPSEN, Inc. |
Octreotide [Sandostatin LAR] | peptides | FDA | 1998-08-24 | 1998-11-25 | Novartis Pharmaceuticals Corporation |
Pegvisomant [Somavert] | proteins | FDA | 1997-06-24 | 2003-03-25 | Sensus Corporation |
Butyrylcholinesterase | — | FDA | 1992-09-30 | — | Shire Laboratories Inc. |