FDA Approves Crinetics' Palsonify, Marking New Era in Acromegaly Treatment
The U.S. Food and Drug Administration (FDA) has granted approval to Crinetics Pharmaceuticals' Palsonify (paltusotine), ushering in a potentially transformative treatment option for patients with acromegaly. This milestone marks Crinetics' entry into the commercial pharmaceutical market and represents a significant advancement in the management of this rare endocrine disorder.
A Paradigm Shift in Acromegaly Care
Palsonify, a once-daily oral medication, has been approved as a first-line treatment for adults with acromegaly for whom surgery was either unsuccessful or not an option. The drug's novel mechanism of action as a selective somatostatin receptor type 2 (SST2) agonist offers patients an alternative to the current standard of care, which typically involves monthly injections.
Scott Struthers, Ph.D., co-founder and CEO of Crinetics, emphasized the drug's potential impact: "This is a whole new era of care for acromegaly—I think it will transform people's lives." The oral formulation of Palsonify is expected to significantly improve patient convenience and compliance compared to existing injectable therapies.
Compelling Clinical Data Supports Approval
The FDA's decision was based on positive results 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 treated patients reaching normal IGF-1 levels versus 5% for placebo.
Beyond biochemical control, patients reported significant reductions in acromegaly symptoms, including headaches, joint pain, sweating, and fatigue, as measured by the FDA-aligned Acromegaly Symptom Diary (ASD).
Crinetics' Commercial Debut and Market Outlook
This approval propels Crinetics into the commercial pharmaceutical arena after nearly two decades of research and development. The company plans to make Palsonify available in the U.S. by early October, supported by a patient assistance program called CrinetiCARE.
Crinetics estimates an initial addressable market of 11,000 diagnosed acromegaly patients in the U.S., with potential for expansion as awareness increases. The company has built a sales force of approximately 30 representatives and has been actively engaging with the endocrinology community to prepare for the launch.
As Crinetics transitions from a clinical-stage to a commercial-stage company, CEO Struthers reflected on the journey: "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." This approval is expected to provide a foundation for Crinetics' future growth and pipeline development in the endocrinology space.
References
- FDA Approves Crinetics’ Once-Daily Acromegaly Treatment
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.”
- Crinetics breaks the commercial barrier with FDA nod for oral acromegaly med Palsonify
The FDA on Thursday signed off on Crinetics’ paltusotine—now christened Palsonify—as a first-line treatment for certain adults with acromegaly. Unlike other somatostatin drugs that make up the bulk of current acromegaly care options, Palsonify is a small molecule, non-peptide therapy that can be taken orally, potentially freeing up patients from frequent and often painful injections.
Explore Further
What distinguishes Palsonify's mechanism of action as a selective SST2 agonist from current injectable treatments for acromegaly?
What were the safety outcomes reported in the Phase III trials PATHFNDR-1 and PATHFNDR-2 for Palsonify?
How does Crinetics plan to overcome potential competition from injectable therapies currently available in the acromegaly market?
What are the key components and objectives of the CrinetiCARE patient assistance program mentioned in the article?
What is Crinetics' strategy for expanding beyond the initial market of 11,000 diagnosed acromegaly patients in the U.S.?