FDA Approves Palsonify™: A New Era in Acromegaly Treatment for Adults
FDA Approves Palsonify™: A New Hope for Acromegaly Patients
In a groundbreaking development for those living with acromegaly, the U.S. Food and Drug Administration (FDA) has approved Palsonify™ (paltusotine), marking a significant advancement in treatment options for adults who have not responded adequately to surgery or are not candidates for surgical intervention. This approval introduces the first once-daily, oral medication specifically designed for acromegaly, a rare hormonal disorder characterized by excessive growth hormone production.
Scott Struthers, Ph.D., Founder and CEO of Crinetics, expressed his enthusiasm for the approval, stating, “Today marks a new era for those living with acromegaly and also for Crinetics as a company. We are fulfilling our commitment to transforming patient lives.” He emphasized the collaborative effort behind Palsonify’s development, thanking patients, caregivers, and healthcare professionals for their invaluable contributions.
The FDA’s decision is backed by robust data from the PATHFNDR-1 and PATHFNDR-2 Phase 3 pivotal trials, which assessed Palsonify’s safety and efficacy in both previously treated and untreated adults. Results indicated that Palsonify consistently achieved rapid biochemical control and sustained efficacy, with participants reporting significant reductions in acromegaly-related symptoms such as headaches, joint pain, and fatigue. Notably, the treatment was well-tolerated, with no serious adverse events recorded during the trials.
Dr. Shlomo Melmed, Executive Vice President of Medicine and Health Sciences at Cedars-Sinai, highlighted the importance of this approval, stating, “The PATHFNDR clinical development program set a new standard for acromegaly treatment.” He noted that Palsonify addresses an unmet need for a safe, easy-to-administer therapy that provides rapid action and durable responses.
Jill Sisco, President of the Acromegaly Community, echoed these sentiments, emphasizing the shift from burdensome injections to a more manageable oral treatment. “What matters most to our community is maintaining consistent control so the disease doesn’t control us,” she said, reflecting on the collaborative efforts with the FDA to ensure patient voices were heard in the development of new therapies.
Palsonify is expected to be available in the U.S. by early October, with Crinetics committed to ensuring broad access through partnerships with payers, healthcare providers, and advocacy organizations. To support patients throughout their treatment journey, Crinetics has launched CrinetiCARE®, a comprehensive program offering education, financial assistance, and access to dedicated nurse educators.
Looking ahead, a Marketing Authorization Application for paltusotine is currently under review in the European Union, with a decision expected in the first half of 2026. Additionally, Crinetics is collaborating with Sanwa Kagaku Kenkyuso to develop and commercialize paltusotine in Japan, while also evaluating its potential for treating carcinoid syndrome in ongoing clinical trials.
As the medical community celebrates this milestone, Palsonify stands as a beacon of hope for those affected by acromegaly, promising a new chapter in their treatment journey.
