First-Ever! FDA Approves New Drug Paltusotine for Rare Disease
This new non-peptide somatostatin receptor 2 (SST2) agonist has become the first FDA-approved once-daily oral treatment for acromegaly, providing patients with a convenient and effective new treatment option.
On September 25, 2025, Crinetics Pharmaceuticals announced that its drug, paltusotine, received U.S. FDA approval for the treatment of adult patients with acromegaly who are inadequately responsive to or ineligible for surgery. As the first FDA-approved, once-daily oral treatment for acromegaly, this non-peptide somatostatin receptor 2 (SST2) agonist modulates growth hormone (GH) secretion by activating the SST2 receptor, thereby controlling levels of the key biomarker insulin-like growth factor 1 (IGF-1), filling a gap in oral treatment options for this disease.
Two Phase 3 clinical trials have fully demonstrated the efficacy and safety of paltusotine. In the PATHFNDR-2 trial, 56% of treated patients achieved IGF-1 ≤1.0 times the upper limit of normal (ULN) compared to only 5% in the placebo group, and 67% achieved IGF-1 <1.3 times the upper limit of normal (ULN) compared to 14% in the placebo group. In the PATHFNDR-1 trial, 83% of patients previously achieving target levels with other medications maintained stable IGF-1 levels after switching to the drug (compared to only 4% in the placebo group). In terms of safety, the drug was generally well tolerated, with common adverse reactions including diarrhea and headache. Regular monitoring for risks such as cholelithiasis and dysglycemia is required.
This approval not only provides a convenient and effective new treatment option for patients with acromegaly, but also promotes the shift toward oral and convenient treatment for rare diseases. The high proportion of patients opting for extended treatment in the long-term trial demonstrates the value of oral dosage forms in improving patient compliance. It also provides a valuable reference for the industry's development of targeted drugs for endocrine diseases and highlights Crinetics' innovative strength in rare disease treatment.