Paltusotine
 Molecular structure of paltusotine | |
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| Trade names | Palsonify |
| AHFS/Drugs.com | Multum Consumer Information |
| MedlinePlus | a625106 |
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| Routes of administration | By mouth |
| Drug class | Somatostatin receptor agonist |
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| Formula | C27H22F2N4O |
| Molar mass | 456.497 g·mol−1 |
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Paltusotine, sold under the brand name Palsonify, is a medication used for the treatment of acromegaly.[1] It is a somatostatin receptor 2 agonist.[1] It is taken by mouth.[1] It was developed by Crinetics Pharmaceuticals.
The most common side effects include diarrhea, abdominal pain, nausea, decreased appetite, bradycardia, hyperglycemia, and gastroenteritis (stomach inflammation).[4]
Paltusotine was approved for medical use in the United States in September 2025.[4]
Medical uses
[edit]Paltusotine is indicated for the treatment of adults with acromegaly who had an inadequate response to surgery and/or for whom surgery is not an option.[1][4]
Acromegaly is a rare endocrine disorder that causes some bones, organs, and other tissue to grow bigger.[4] The pituitary gland in the brain causes these changes by making too much growth hormone due to the presence of a non-cancerous tumor.[4]
Adverse effects
[edit]Paltusotine increases the risk of cholelithiasis (gallstones); hyperglycemia (high blood sugar); hypoglycemia (low blood sugar); bradycardia (low heart rate); thyroid function abnormalities; steatorrhea (excessive fat in the stool) and malabsorption of dietary fats; and changes in vitamin B12 levels.[4]
The most common side effects are diarrhea, abdominal pain, nausea, decreased appetite, bradycardia, hyperglycemia, and gastroenteritis (stomach inflammation).[4]
History
[edit]The safety and efficacy of paltusotine were evaluated in two randomized, double-blind, placebo-controlled, phase III studies.[4]
In study 1, 111 adults with acromegaly received paltusotine or placebo.[4] The primary endpoint was the proportion of participants achieving biochemical control (defined as insulin-like growth factor [IGF-1] and GH levels within the normal range).[4] At 24 weeks, 56% of participants who received paltusotine had achieved biochemical control compared to 5% of participants who had received placebo.[4]
In study 2, 58 adults with acromegaly who were previously treated with and responded to other medical therapy received paltusotine or placebo.[4] At 36 weeks, 83% of participants switching to paltusotine in study 2 maintained biochemical control compared to 4% of participants receiving placebo.[4]
Society and culture
[edit]Legal status
[edit]Paltusotine was approved for medical use in the United States in September 2025.[5]
Names
[edit]Paltusotine is the international nonproprietary name.[6]
Paltusotine is sold under the brand name Palsonify.[1]
References
[edit]- 1 2 3 4 5 6 "Palsonify- paltusotine tablet, film coated". DailyMed. 16 September 2025. Retrieved 12 July 2026.
- ↑ "Palsonify EPAR". European Medicines Agency (EMA). 27 February 2026. Retrieved 7 June 2026.
- ↑ "Palsonify PI". Union Register of medicinal products. 27 April 2026. Retrieved 7 June 2026.
- 1 2 3 4 5 6 7 8 9 10 11 12 13 "FDA approves new treatment for acromegaly, a rare endocrine disorder". U.S. Food and Drug Administration (FDA). 26 September 2025. Retrieved 27 September 2025.
This article incorporates text from this source, which is in the public domain. - ↑ "Crinetics Announces FDA Approval of Palsonify (paltusotine) for the Treatment of Adults with Acromegaly" (Press release). Crinetics Pharmaceuticals. 25 September 2025. Retrieved 27 September 2025 – via GlobeNewswire.
- ↑ "International nonproprietary names for pharmaceutical substances (INN): recommended INN: list 83". WHO Drug Information. 34 (1). 2020. hdl:10665/339768.
Further reading
[edit]- Gadelha, Monica R; Gordon, Murray B; Doknic, Mirjana; Mezősi, Emese; Tóth, Miklós; Randeva, Harpal; et al. (April 2023). "ACROBAT Edge: Safety and Efficacy of Switching Injected SRLs to Oral Paltusotine in Patients With Acromegaly". The Journal of Clinical Endocrinology & Metabolism. 108 (5): e148–e159. doi:10.1210/clinem/dgac643. PMC 10099171. PMID 36353760. S2CID 253445337.
- Madan, Ajay; Markison, Stacy; Betz, Stephen F.; Krasner, Alan; Luo, Rosa; Jochelson, Theresa; et al. (April 2022). "Paltusotine, a novel oral once-daily nonpeptide SST2 receptor agonist, suppresses GH and IGF-1 in healthy volunteers". Pituitary. 25 (2): 328–339. doi:10.1007/s11102-021-01201-z. PMC 8894159. PMID 35000098.
- Zhao, Jian; Wang, Shimiao; Markison, Stacy; Kim, Sun Hee; Han, Sangdon; Chen, Mi; et al. (January 2023). "Discovery of Paltusotine (CRN00808), a Potent, Selective, and Orally Bioavailable Non-peptide SST2 Agonist". ACS Medicinal Chemistry Letters. 14 (1): 66–74. doi:10.1021/acsmedchemlett.2c00431. PMC 9841592. PMID 36655128.
- Zhao, Jie; Fu, Hong; Yu, Jingjing; Hong, Weiqi; Tian, Xiaowen; Qi, Jieyu; et al. (February 2023). "Prospect of acromegaly therapy: molecular mechanism of clinical drugs octreotide and paltusotine". Nature Communications. 14 (1): 962. Bibcode:2023NatCo..14..962Z. doi:10.1038/s41467-023-36673-z. ISSN 2041-1723. PMC 9944328. PMID 36810324.
External links
[edit]- Clinical trial number NCT05192382 for "A Study to Evaluate the Safety and Efficacy of Paltusotine for the Treatment of Acromegaly (PATHFNDR-2) (PATHFNDR-2)" at ClinicalTrials.gov
- Clinical trial number NCT04837040 for "A Study to Evaluate the Safety and Efficacy of Paltusotine for the Treatment of Acromegaly (PATHFNDR-1)" at ClinicalTrials.gov
