FDA Approves Crinetics Acromegaly Drug

Rare Daily Staff

The U.S. Food and Drug Administration has approved Crinetics Pharmaceuticals’ Palsonify for the first-line treatment of adults with the rare endocrine disorder acromegaly who had an inadequate response to surgery or for whom surgery is not an option.

Palsonify is the first once-daily oral treatment approved for adults with acromegaly.

The approval is based on data from the PATHFNDR-1 and PATHFNDR-2 phase 3 pivotal trials, which evaluated Palsonify’s safety and efficacy in previously treated and medically untreated adults with acromegaly. Across both trials, Palsonify consistently demonstrated rapid onset, reliable biochemical control, and sustained efficacy.

Participants reported significant reductions in signs and symptoms associated with acromegaly, as measured by the Acromegaly Symptom Diary, an FDA-aligned patient-reported outcome tool developed to capture the symptoms that matter most to people living with acromegaly.

Acromegaly is typically caused by a pituitary adenoma, a benign tumor in the pituitary gland that secretes excessive growth hormone. This, in turn, triggers the overproduction of IGF-1 in the liver. Prolonged exposure to elevated IGF-1 and growth hormone levels leads to serious systemic complications, often resulting in bone, joint, cardiovascular, metabolic, cerebrovascular, or respiratory disease.

Symptoms of acromegaly include headaches, joint pain, fatigue, sleep apnea, excessive sweating, oily skin, bone and cartilage overgrowth, abnormal enlargement of the hands and feet, enlargement of the heart, liver, and other organs, and changes in facial features. Uncontrolled acromegaly results in increased mortality and has a debilitating impact on daily functioning and quality of life.

Palsonify (paltusotine) is the first oral, once-daily, selectively targeted somatostatin receptor type 2 agonist and is currently in Phase 3 investigational studies. Crinetics designed the therapy to provide a once-daily option for people living with acromegaly and neuroendocrine tumors. In Phase 2 studies and the recently completed PATHFNDR-1 Phase 3 trial, Palsonify maintained IGF-1 levels in people with acromegaly who switched from monthly injectable medications.

A Marketing Authorization Application for paltusotine in acromegaly is currently under review in the European Union. The Committee for Medicinal Products for Human Use is expected to issue an opinion in the first half of 2026. Crinetics is partnering with Sanwa Kagaku Kenkyuso to develop and commercialize paltusotine for acromegaly in Japan.

Paltusotine is also being evaluated as a treatment for carcinoid syndrome in a pivotal Phase 3 study.

“For people living with acromegaly, treatment once meant burdensome injections, breakthrough symptoms, and lifestyle sacrifices just to stay on track,” said Jill Sisco, president of Acromegaly Community. “What matters most to our community – maintaining consistent control so the disease doesn’t control us – led us to partner with the FDA on Externally Led Patient-Focused Drug Development meetings. This new treatment reflects that our voices have been heard in shaping the next generation of acromegaly care.”

Photo: Jill Sisco, president of Acromegaly Community