PALSONIFY is a non-peptide, selectively targeted somatostatin receptor type 2 (SST2) agonist known as the first oral drug approved by the U.S Food and Drug Administration (FDA) for the treatment of acromegaly. The approval was based on information from two pivotal Phase 3 studies that showed PALSONIFY was well-tolerated, effectively provided reliable biochemical control, and significantly decreased symptom burden in patients.
Crinetics, the biopharmaceutical company, has achieved a major milestone with the launch of its primary product. According to the CEO of Crinetics, this approval marks the beginning of a new era not only for acromegaly patients but also for the company itself. This approval reflects the contributions of dedicated researchers, support staff, helpers, and medical professionals who made this breakthrough possible.
Based on information from the PATHFNDR-1 and PATHFNDR-2 Phase 3 clinical studies, which assessed safety and efficacy in people with acromegaly who had received prior medical treatment and those who did not take any treatment. Both trials incorporated the Acromegaly Symptom Diary (ASD) to identify the symptoms in patients. It suggests that participants also reported substantial reductions in acromegaly-related signs and symptoms.
In the randomized controlled phases, PALSONIFY was generally well tolerated with no reports of severe adverse events. In the recent annual conference of the Endocrine Society, the long-term findings from the open-label extension (OLE) phases of both clinical studies were presented. These results highlighted PALSONIFY’s capacity to provide long-lasting improvements in patient outcomes, a consistent safety profile, and durable IGF-1 control. At the same time, the OLE enrollment rate was observed in 91% of patients from PATHFNDR-1 and 97% of participants from PATHFNDR-2, who chose to continue therapy.
PALSONIFY approval is considered a significant step forward for patients with acromegaly by October 2025. PALSONIFY is expected to be available in the United States. To help individuals, Crinetics has collaborated closely with payers, healthcare professionals, and advocacy groups to ensure broad access. A comprehensive support program called CrinetiCARE® was introduced by Crinetics to help individuals during their therapy.
The financial resources, illness management, and product knowledge access can help with treatment onboarding and continued adherence, all of which are provided by the CrinetiCARE program. The European Union (EU) is reviewing a Marketing Authorization Application (MAA) for patients with acromegaly treated with paltusotine. The first half of 2026 is the current deadline of the Committee for Medicinal Products and Human Use (CHMP). Paltusotine for acromegaly is developed and marketed in Japan by Crinetics in collaboration with Sanwa Kagaku Kenkyuso (SKK).
The key Phase 3 CAREFNDR study was evaluated for the treatment of carcinoid syndrome. The frequency of bowel movements and flushing episodes was considered the most prevalent symptoms of carcinoid syndrome and decreased in the results of Phase 2 research. Paltusotine is also developed clinically for neuroendocrine tumor-related carcinoid disease. With PALSONIFY, Crinetics has not only delivered the first oral acromegaly therapy but also reinforced its commitment to advancing innovative medicines that target small molecules and G-protein-coupled receptors (GPCRs).
Reference: Crinetics Pharmaceuticals, Inc. Crinetics Announces FDA Approval of PALSONIFY (paltusotine) for the Treatment of Acromegaly in Adults. Crinetics. Published on September 25, 2025. Accessed on September 30, 2025. Crinetics | Crinetics Announces FDA Approval of PALSONIFY™ (paltusotine) for the Treatment of Acromegaly in Adults
