The regulatory greenlight was backed by two Phase III trials that showed normalized growth hormone levels in patients with the rare pituitary condition. It’s the first approval for Crinetics Pharmaceuticals and something CEO Scott Struthers predicted “will transform people’s lives.”
The FDA has approved Crinetics’ once-daily, SST2 agonist oral therapy paltusotine for the first-line treatment of the rare pituitary condition acromegaly, the company announced Thursday. The drug will be marketed as Palsonify.
Crinetics’ treatment steps into an indication with difficult treatment regimens for patients. Acromegaly is caused by tumors growing in the pituitary gland, which in turn causes the liver to secrete excess growth hormone. In children, this results in gigantism; in adults, it results in acromegaly, where soft tissues in the face and heart can result in disruptive growth and cardiomyopathy, as well as enlargement of the liver and growth in the skin and cartilage.
Common standard treatments include surgery on the pituitary gland, which often fails to clear all tumor material. “It’s very difficult to get all of the tumor out unless you’re lucky,” CEO Scott Struthers told BioSpace in an interview ahead of the approval. Patients often then take monthly depot injections of Ipsen’s Somatuline, which reduces growth hormones.
These monthly injections are “big and ugly” and “difficult to do correctly,” Struthers said. Palsonify’s mechanism of action works by increasing the expression of somatostatin, which counteracts the over-growth symptoms of the condition.
“This is a whole new era of care for acromegaly—I think it will transform people’s lives,” Struthers said. Instead of monthly injections, Palsonify is a once-daily oral molecule “that you take and move on with your day,” as Struthers put it.
Crinetics filed the new drug application back in December 2024 backed by data from two Phase III trials—PATHFNDR-1 and PATHFNDR-2. In the first trial, data presented in 2023 showed 83% of patients taking the medication achieved an insulin-like growth factor 1 (IGF-1) at the upper limit of normal, versus 4% of patients taking placebo.
PATHFNDR-2, reading out in 2024, showed that 56% of patients taking Palsonify reached normal IGF-1 levels versus 5% in the placebo group.
For Crinetics, the approval marks the beginning of its commercial stage.
“I started this [company] in 2008 and we bootstrapped until 2015,” Struthers said. “We had no venture financing until 2015. Now we’re a 600 person SMID cap company on the NASDAQ starting to sell its first drug.”
“We’re entering rarefied air,” Struthers continued. “There aren’t that many companies that conceive, design, and make their own drugs in their own labs. Our goal is to be a fully integrated, broad endocrinology company, and there’s just not many profiles like that.”
“And,” Struthers added, gesturing around the room, “we’re dog friendly.”
