Palsonify is a medicine for certain people with acromegaly. Acromegaly is a rare disorder that happens when your body makes too much growth hormone (GH). This hormone is made and released in the pituitary gland in your brain. In children, GH stimulates growth and development. In adults, GH affects energy levels, muscle strength, bone health, and one’s sense of well-being.
When you have too much GH in your blood, your liver makes too much of another hormone called insulin-like growth factor (IGF-1). This hormone also makes your bones and other body parts grow more than they should. Too much IGF-1 can change the way your body handles blood sugar and fats, which causes other health conditions like diabetes, high blood pressure, and heart disease. Symptoms usually happen over a long period of time. You may have unusually large hands and feet, changes in ring size or your shoe width, or changes in your face. Other symptoms may include joint pain, stiff, rough body hair, a deep voice, sweating, headaches, low energy, weakness and being tired, depression, vision problems, less strength, loss of muscle mass, or an increase in body fat.
The most common cause of acromegaly is a benign (noncancerous) tumor in your pituitary gland called a pituitary adenoma. While very rare, tumors outside the pituitary (ectopic tumors) can cause acromegaly by producing growth hormone-releasing hormone (GHRH), which then stimulates the pituitary to make too much GH. The most common locations for these rare ectopic tumors are the lung, pancreas, and adrenal glands.
The main treatment for acromegaly that is caused by a pituitary tumor is surgery. About half of people who have surgery still have too much GH and IGF-1. Radiation therapy may be used if there are parts of a tumor left after surgery or to stop the tumor from growing. Medicines are used after surgery to help control the disease and bring hormone levels back to normal. The somatostatin analogs lanreotide, octreotide, and pasireotide are used to lower GH and IGF-1 levels. The medicine pegvisomant blocks the effects of growth hormone in your body.
Palsonify (paltusotine) is a new medicine taken by mouth to treat adults with acromegaly for whom surgery did not work or if surgery is not an option.
How Does Palsonify Work for Acromegaly?
The main ingredient in Palsonify is paltusotine, a medicine that acts like the natural hormone in your body called somatostatin. Somatostatin helps regulate growth hormone. Palsonify lowers the amount of GH and IGF-1 in your body, which controls the symptoms of acromegaly.
How Do I Take Palsonify?
Palsonify is a tablet that is taken by mouth once a day with a glass of water on an empty stomach. Take Palsonify at least six hours after eating, such as in the morning after an overnight fast. Do not eat food for at least one hour after taking Palsonify.
When you first start taking Palsonify, your health care provider may lower your dose temporarily if you have side effects and then increase your dose based on how you tolerate the medicine. After two to four weeks, based on your blood tests, your health care provider may adjust your dose. Take Palsonify exactly as your health care provider tells you to take it.
How Was Palsonify Studied for Acromegaly?
There were two clinical studies that looked at how well Palsonify worked for adults with acromegaly and how safe it was.
Study 1 compared Palsonify to a placebo (containing no medicine) in 111 people who had never taken medicine for acromegaly or who had been treated with a somatostatin analog. The people in the study did not know if they were getting Palsonify or a placebo. The average age of people in the study was 47 years old, and 53% were female. About half (52%) were White, 31% Asian, 3% Black or African American, 9% other, and 5% unknown race. The average time since diagnosis of acromegaly was about seven years (87 months). Most of the people in the study (95%) had pituitary gland surgery an average of 6.5 years (78 months) before the study started.
People in the study started Palsonify at a dose of 20 milligrams once per day, followed by 40 milligrams once per day after two weeks, or a matching placebo. The dose of Palsonify could be increased up to 60 milligrams during the first 12 weeks based on blood levels of IGF-1. After that, the Palsonify dose stayed the same for 12 more weeks. The study lasted 24 weeks. There were 54 people in the Palsonify group and 57 people in the placebo group.
The main goal of the study was to see what proportion of people who took Palsonify reached normal IGF-1 blood levels, compared to those who took a placebo.
Study 2 included 58 people with acromegaly who were previously treated and controlled on a somatostatin analog called lanreotide depot (long-acting) or octreotide injections. They had to have been getting a stable, effective dose of lanreotide or octreotide for at least 12 weeks. The average age of people in the study was 55 years old, and 55% were female. Most were White (72%), 3% Asian, 5% Black or African American, 12% other, and 7% unknown race. The average time since diagnosis of acromegaly was about 13 years (155 months). Most of the people in the study (86%) had pituitary gland surgery an average of 11.5 years (138 months) before the study started.
People in the study were switched from lanreotide or octreotide to Palsonify at a dose of 40 milligrams once per day or a placebo. The first dose of study medicine was taken when the next injection of lanreotide or octreotide was due. The dose of Palsonify could be increased to 60 milligrams during the first 24 weeks based on blood levels of IGF-1. After that, the Palsonify dose stayed the same for 12 more weeks. The study lasted 36 weeks. There were 30 people in the Palsonify group and 28 people in the placebo group.
The main goal of the study was to see what proportion of people who took Palsonify maintained normal IGF-1 blood levels, compared to those who took a placebo after switching from lanreotide depot or octreotide injections.
What Benefits of Palsonify Were Seen?
In Study 1, after 24 weeks, 56% of people who took Palsonify reached normal IGF-1 levels, compared to 5% of those who took a placebo. Most of the people who took Palsonify had their IGF-1 levels reach normal within the first two to four weeks of taking Palsonify, and the response lasted until the end of the study.
In Study 2, after 36 weeks, 83% of people who switched from lanreotide or octreotide injections to oral Palsonify maintained normal IGF-1 levels, compared to 4% of those who took a placebo.
In both studies, people who took Palsonify had an improvement in symptoms of acromegaly such as headaches, joint pain, sweating, fatigue, weakness, swelling, and/or numbness and tingling.
Your results may differ from what was seen in clinical studies.
What Are the Side Effects of Palsonify, and How Can I Prevent or Manage Them?
Common side effects include diarrhea, nausea, stomach pain, loss of appetite, or the stomach flu. Most digestive side effects happen in the first two months after starting Palsonify and usually last about one to two weeks. If you have diarrhea, your health care provider may recommend that you take antidiarrhea medicine and drink more fluids to avoid dehydration.
Palsonify can cause problems with how your body uses fat. Because of this, you might see too much fat in your poop. This can lead to signs like loose poop (diarrhea), feeling bloated (gassy and full), losing weight without trying, and poop that looks greasy, oily, pale, or smells very strong. Tell your health care provider if you have any of these problems, especially if your poop is loose, greasy, or you feel bloated.
Palsonify may cause gallbladder problems. Tell your health care provider if you have sudden pain in the upper right area of your stomach or in your back between your shoulder blades, yellowing of your skin or the whites of your eyes, or pale stools (poop).
Palsonify can change the hormones in your body. This might make your blood sugar go up or down. This is especially important if you have diabetes and take medicine for it, like insulin. Your health care provider will check your blood sugar when you first start Palsonify or when you get a new dose. If you check your own blood sugar, let your health care provider know right away if you see any unusual changes in your numbers.
Palsonify can make your heart beat too slowly. This problem is called bradycardia. Taking Palsonify with other medicines that also slow your heart down can make this problem worse. Always tell your health care provider about all the medicines you take. Also tell them if you feel like your heart is beating slower than usual, or if it doesn't feel like it's beating normally.
Palsonify can cause a thyroid problem called hypothyroidism. This happens when the drug stops your thyroid gland from releasing enough thyroid hormones. Watch out for signs of hypothyroidism, which can include feeling tired all the time, gaining weight, feeling cold, or having dry skin. Your health care provider will do tests to check how your thyroid is working while you take Palsonify.
What Type of Drug Interactions Can Happen?
Some medicines can lower the amount of Palsonify in your blood. If this happens, Palsonify might not work as well as it should. Your health care provider may need to adjust your dose of Palsonify if you are taking any of these medicines. Ask your health care provider if any of the medicines you are taking can reduce your Palsonify levels.
Palsonify can also increase the amount of certain other medicines in your blood, which might increase your risk of side effects from those medicines. Tell your health care provider about all the prescription and over-the-counter medicines and supplements you are taking.
Is There Any Cost Assistance Available?
There is a patient support program offered by the drugmaker called CrinetiCARE that may help you afford Palsonify. Your health care provider can help you get started. You can find out more at https://crineticare.com/ or by calling 844-276-4357.