FDA Approves Crinetics Pharma's First Daily Pill For Rare Growth Disorder

On Thursday, the U.S. Food and Drug Administration (FDA) approved Crinetics Pharmaceuticals, Inc.’s (NASDAQ: CRNX) Palsonify (paltusotine) for the first-line treatment of adults with acromegaly who had an inadequate response to surgery and/or for whom surgery is not an option.

Acromegaly is a rare disorder characterized by the excessive production of growth hormone (GH), leading to abnormal growth of hands, feet, and facial features in adults.

Palsonify, a selectively-targeted somatostatin receptor type 2 nonpeptide (SST2) agonist, is now the first once-daily, oral treatment approved for adults with acromegaly.

Also Read: Crinetics Reports Durable Symptom, Hormone Control With Oral Drug In 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 also reported significant reductions in signs and symptoms associated with acromegaly as measured by the Acromegaly Symptom Diary (ASD), an FDA-aligned patient-reported outcome tool developed to capture the symptoms, including headaches, joint pain, sweating, fatigue, weakness, swelling, and/or numbness/tingling.

Palsonify was generally well-tolerated, with no serious adverse events reported in the randomized controlled portion of the trials. 

Palsonify is expected to be available in the U.S. in early October.

A Marketing Authorization Application (MAA) for paltusotine in acromegaly is currently under review for use in the European Union, and the current timeline for the Committee for Medicinal Products and Human Use (CHMP) opinion is the first half of 2026.

Crinetics is in partnership with Sanwa Kagaku Kenkyuso (SKK) to develop and commercialize paltusotine for acromegaly in Japan.

Paltusotine is also being evaluated for the treatment of carcinoid syndrome in the pivotal Phase 3 CAREFNDR trial.

Carcinoid syndrome is a collection of symptoms, including flushing, diarrhea, and breathing problems, caused by hormones and other chemicals released from a carcinoid tumor.

Price Action: CRNX stock was trading higher by 27.35% to $45.71 at last check Friday.

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