FDA Approves Crinetics' Palsonify, Ushering in New Era for Acromegaly Treatment

The U.S. Food and Drug Administration (FDA) has granted approval to Crinetics Pharmaceuticals' Palsonify (paltusotine), marking a significant milestone in the treatment of acromegaly. This oral, once-daily medication represents a paradigm shift in care for patients with the rare pituitary condition, offering a more convenient alternative to current injectable therapies.

A Breakthrough in Acromegaly Management

Palsonify, a selectively targeted somatostatin receptor type 2 (SST2) agonist, is the first FDA-approved oral treatment for acromegaly. The drug is indicated as a first-line therapy for adults with acromegaly for whom surgery was unsuccessful or is not an option.

Acromegaly, caused by benign tumors in the pituitary gland, leads to excessive production of growth hormone. This overproduction results in various symptoms, including enlarged hands, feet, and facial features, as well as potential cardiovascular complications.

Scott Struthers, Ph.D., co-founder and CEO of Crinetics, emphasized the transformative nature of Palsonify: "This is a whole new era of care for acromegaly—I think it will transform people's lives. Instead of monthly injections, Palsonify is a once-daily oral molecule that you take and move on with your day."

Compelling Clinical Data

The FDA's approval was based on data from two Phase III trials, PATHFNDR-1 and PATHFNDR-2. In PATHFNDR-1, 83% of patients taking Palsonify achieved normalized insulin-like growth factor 1 (IGF-1) levels, compared to only 4% in the placebo group. PATHFNDR-2 demonstrated similar efficacy, with 56% of Palsonify-treated patients reaching normal IGF-1 levels versus 5% for placebo.

Importantly, patients reported significant reductions in acromegaly symptoms, including headaches, joint pain, sweating, fatigue, and weakness, as measured by the FDA-aligned Acromegaly Symptom Diary (ASD).

Commercial Launch and Market Potential

Crinetics plans to make Palsonify available in the U.S. by early October 2025. The company has implemented a patient support program called CrinetiCARE and is working with payers, healthcare providers, and patient advocacy groups to ensure broad access to the medication.

The addressable market for Palsonify in the U.S. is estimated at 11,000 diagnosed acromegaly patients, with potentially 15,000 more yet to be diagnosed. Crinetics aims to bridge this diagnosis gap through educational initiatives and patient outreach programs.

Struthers highlighted the company's growth, stating, "We started out with four people and two dogs, and now we're at almost 600 people, and I don't know how many dogs. We're a dog-friendly workplace."

As Crinetics transitions from a clinical-stage to a commercial-stage company, industry analysts anticipate that Palsonify's launch will provide a strong foundation for the company's expansion into other endocrine indications. The approval marks a significant achievement for Crinetics, which has been developing endocrine therapies since its incorporation in 2008.