The U.S. Food and Drug Administration (FDA) has approved Palsonify (paltusotine) for the first-line treatment of adults with acromegaly with inadequate response to surgery and/or for whom surgery is not an option.
Acromegaly is a hormonal disorder that results from the pituitary gland producing too much growth hormone (GH). It is most often diagnosed in middle-aged adults, although symptoms can appear at any age. Signs and symptoms include abnormal growth and swelling of the hands and feet, bone changes that alter various facial features, arthritis, carpal tunnel syndrome, enlargement of body organs, and various other symptoms. The condition is usually caused by benign adenomas on the pituitary. Ocassionally, it is caused by tumors of the pancreas, lungs, and other parts of the brain stimulating the pituitary gland to produce GH.
Paltusotine is a selectively-targeted somatostatin receptor type 2 nonpeptide agonist, the first once-daily, oral treatment approved for adults with acromegaly.
The approval follows positive data from the PATHFNDR-1 (NCT04837040) and PATHFNDR-2 (NCT05192382) clinical trials. Both phase 3 studies evaluated the safety and efficacy of paltusotine in previously treated and medically untreated adults with acromegaly. Paltusotine consistently demonstrated rapid onset, reliable biochemical control, and sustained efficacy.
Significant reductions in signs and symptoms measured by the Acromegaly Symptom Diary patient-reported outcome tool were also observed. These symptoms included headaches, joint pain, sweating, fatigue, weakness, swelling, and numbness/tingling. Additionally, paltusotine was well-tolerated with no serious adverse events reported in the randomized controlled trial portion.
Long-term results from the open-label extension phase were recently presented at ENDO 2025. This included data on paltusotine’s ability to deliver durable IGF-1 control, sustained improvement in patient symptom burden, and a consistent safety profile.
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To learn more about acromegaly and other rare endocrine disorders, visit https://checkrare.com/diseases/endocrine-disorders/