A New Era for Acromegaly Treatment
After almost 20 years in the endocrine space, Crinetics is entering the commercial arena with its first approved product for the uncommon growth disease acromegaly.
On Thursday, the FDA cleared paltusotine – now branded as Palsonify – as a frontline treatment for adults whose acromegaly persists after surgery or who aren’t eligible for surgical intervention.
The Shift from Injections to Oral Medication
Palsonify targets somatostatin receptor type 2. Unlike most current acromegaly therapies, which are peptide-based and delivered through frequent, often painful injections, this drug is a small-molecule oral option, offering patients a more convenient alternative.
Scott Struthers, Crinetics’ co-founder and CEO of FDA, said in an interview that this therapy represents a major advancement for people with acromegaly. He pointed to the drug’s ability to manage both hormone levels and symptoms in a convenient once-daily dose as the primary reason for that claim.
Understanding Acromegaly
Acromegaly is a rare disorder caused by a noncancerous tumor in the pituitary gland. The tumor leads the brain to release excessive amounts of growth hormone. That hormone then circulates through the bloodstream and signals the liver to produce high levels of insulin-like growth factor-1 (IGF-1), ultimately resulting in abnormal tissue enlargement throughout the body. While acromegaly is not dissimilar to gigantism in children, the effects in adults play out differently because their bones have already stopped growing. Instead of height increases, adults typically see enlargement in areas like the hands, feet and facial features.
Struthers noted that the disorder can also trigger joint complications and may cause the heart to enlarge, which in turn raises the risk of cardiovascular problems and other serious health concerns. He said he has spoken with many people living with acromegaly over the years, and the ongoing issue is that current treatments do not adequately manage their symptoms.
Clinical Trial and Symptom Improvement
The typical approach to treating acromegaly – after potential pituitary surgery – relies on somatostatin therapies, which use a natural peptide to suppress growth hormone production. However, these medications must be administered as monthly injections, which is a process that Struthers said can be painful for patients and challenging for providers who give the shots.
Crinetics earned FDA approval for Palsonify based on results from the phase 3 PATHFNDR-1 and PATHFNDR-2 trials. These studies evaluated the drug in patients who had previously received medical treatment as well as those who had not. According to the company’s September 25 announcement, the therapy provided rapid and consistent biochemical control and maintained its effectiveness over time in both trials.
People treated with Palsonify also experienced major improvements in common acromegaly symptoms – including headaches, joint discomfort, excessive sweating, fatigue, muscle weakness, swelling and sensations like tingling – based on assessments from the FDA-endorsed Acromegaly Symptom Diary.
Market Availability and Future Outlook
In a note issued ahead of approval, analysts at Leerink Partners described the findings as conclusive and predicted that Palsonify will give patients a notable convenience advantage while still maintaining strong effectiveness.
The FDA label instructs an initial dose of 40 mg daily on an empty stomach, with possible uptitration to 60 mg based on IGF-1 response.
Crinetics plans to have the therapy on the U.S. market by early October and says it’s collaborating with relevant groups to help patients access Palsonify. The pricing details were not included in Crinetics’ approval announcement.
Strategic & Market Implications (New Content)
A key test will be real-world durability of effect, long-term safety, and adherence. Endocrinologists will watch for sustained IGF-1 control beyond two or three years, and whether oral therapy reduces dose-reverting cycles or therapy switching.
Crinetics also faces market access and pricing pressures. Insurance payers will scrutinize cost versus benefit, especially when competing against older, cheaper injectables. Patient assistance and reimbursement strategy will play a decisive role in adoption.
