September 26, 2025
2 min read
Key takeaways:
- The FDA approved paltusotine for first-line treatment of patients with acromegaly who did not respond to or could not have surgery.
- The drug is the first once-daily oral therapy approved for this population.
The FDA has approved paltusotine, a selectively targeted somatostatin receptor type 2 nonpeptide agonist, for first-line treatment of certain patients with acromegaly, Crinetics Pharmaceuticals announced.
With the decision, paltusotine (Palsonify) became the first once-daily oral therapy approved to treat acromegaly, according to a press release issued by Crinetics.
The drug is indicated for first-line treatment of patients with acromegaly who had an inadequate response to surgery or who are not eligible for surgery, according to the release.
The approval was based on the results of the PATHFNDR-1 and PATHFNDR-2 trials, the company stated in the release.
As Healio previously reported, in PATHFNDR-1, a trial of 58 adults with acromegaly biochemically controlled with octreotide (Mycapssa, Chiasma) or lanreotide (Somatuline Depot, Ipsen) monotherapy, at 36 weeks, 83% of patients receiving paltusotine maintained an insulin-like growth factor I level at or below the upper limit of normal compared with 4% of the placebo group (P < .0001), and the paltusotine group had less change in IGF-I levels than the placebo group (P < .0001).
In PATHFNDR-2, a trial of 111 adults with acromegaly who did not previously receive pharmacologic treatment, as Healio previously reported, at 24 weeks, 56% of adults receiving paltusotine had an IGF-I level at or below the upper limit of normal compared with 5% of the placebo group (P < .0001).
In both trials, patients in the paltusotine group reported reductions in signs and symptoms as assessed by the Acromegaly Symptom Diary, according to the release.
Shlomo Melmed
“The PATHFNDR clinical development program set a new standard for acromegaly treatment by demonstrating the ability of Palsonify to drive both biochemical and symptom control, regardless of the degree of underlying disease severity,” Shlomo Melmed, MBCh, executive vice president of medicine and health sciences and dean of the medical faculty at Cedars-Sinai, said in the release. “The approval of Palsonify is a significant advancement for our patients, as there is an unmet need for an easy-to-administer and safe therapeutic option with a rapid action and durable response that can consistently manage acromegaly.”
“For people living with acromegaly, treatment once meant burdensome injections, breakthrough symptoms, and lifestyle sacrifices just to stay on track,” Jill Sisco, president of Acromegaly Community, said in the release. “What matters most to our community — maintaining consistent control so the disease doesn’t control us — led us to partner with the FDA on externally led patient-focused drug development meetings. This new treatment reflects that our voices have been heard in shaping the next generation of acromegaly care.”
According to the release, the drug will be commercially available in the U.S. in early October, and the company has launched CrinetiCARE, a program to provide disease and product education, benefit verification, financial assistance resources and access to nurse educators to help with treatment onboarding and adherence.
Sources/Disclosures
Source:
Disclosures:
Melmed reports receiving institutional research grants from Recordati and consulting for Crinetics and Novo Nordisk. Sisco reports no relevant financial disclosures.
