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The U.S. Food and Drug Administration (FDA) approved Palsonify (paltusotine) tablets to treat adults with acromegaly who have had an inadequate response to surgery and/or for whom surgery is not an option.

Disease or Condition

Acromegaly is a rare endocrine disorder that causes some bones, organs, and other tissue to grow bigger. The pituitary gland in the brain causes these changes by making too much growth hormone (GH) due to the presence of a non-cancerous tumor.

Effectiveness

Palsonify’s safety and efficacy was evaluated in two randomized, double-blind, placebo-controlled, phase 3 studies.

In Study 1, 111 adults with acromegaly received Palsonify or placebo. The primary endpoint was the proportion of participants achieving biochemical control (defined as insulin-like growth factor [IGF-1] and GH levels within the normal range). At 24 weeks, 56% of participants who received Palsonify had achieved biochemical control compared to 5% of participants who had received placebo.

In Study 2, 58 adults with acromegaly who were previously treated with and responded to other medical therapy received Palsonify or placebo. At 36 weeks, 83% of participants switching to Palsonify in Study 2 maintained biochemical control compared to 4% of participants receiving placebo.

Safety Information

Palsonify increases the risk of cholelithiasis (gallstones); hyperglycemia (high blood sugar); hypoglycemia (low blood sugar); bradycardia (low heart rate); thyroid function abnormalities; steatorrhea (excessive fat in the stool) and malabsorption of dietary fats; and changes in vitamin B12 levels. The most common side effects are diarrhea, abdominal pain, nausea, decreased appetite, bradycardia, hyperglycemia, and gastroenteritis (stomach inflammation).

Dosing and Administration

The recommended initial dosage is 40 mg taken orally once daily with water on an empty stomach, at least 6 hours after a meal (i.e., after overnight fasting) and at least 1 hour before the next meal. During the initiation period, Palsonify may be temporarily reduced to 20 mg once daily if needed, based on tolerability. Once adverse reactions have resolved, patients should resume Palsonify 40 mg once daily.

After 2 to 4 weeks on Palsonify 40 mg once daily and based on their IGF-1 levels, patients may be advised to increase the dosage to 60 mg once daily.