FDA Approves New Treatment for Acromegaly: Daily Dose

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On September 26, 2025, we reported on the FDA approval of paltusotine (Palsonify™, Crinetics Pharmaceuticals), the first once-daily oral treatment for acromegaly in adults.

The approval

Paltusotine is a selectively targeted somatostatin receptor type 2 (SST2) agonist developed as a convenient oral alternative to injectable therapies traditionally used in acromegaly management. The approval was supported by results from the phase 3 PATHFNDR-1 and PATHFNDR-2 trials, which demonstrated consistent biochemical control, symptom reduction, and a favorable safety profile. No serious adverse events were reported in the randomized controlled portions of either trial.

The PATHFNDR program included 2 double-blind, placebo-controlled studies. PATHFNDR-1 enrolled 58 adults with acromegaly who maintained insulin-like growth factor 1 (IGF-1) levels ≤1.0 times the upper limit of normal (ULN) on stable injectable therapy with octreotide or lanreotide. Participants were randomized to receive oral paltusotine or placebo for 36 weeks. PATHFNDR-2 enrolled 112 adults with acromegaly who were either treatment-naïve or not receiving pharmacotherapy and had elevated IGF-1 at baseline. The primary endpoint in both studies was the proportion of participants achieving IGF-1 ≤1.0 x ULN compared with placebo. PATHFNDR-1 met its primary endpoint with statistical significance (P < .0001). Eighty-three percent of participants treated with paltusotine maintained IGF-1 ≤1.0 x ULN compared with 4% of placebo-treated participants. All secondary endpoints, including measures of symptom improvement, also met statistical significance.

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