What Is Palsonify, and Why Does It Matter?
Palsonify (paltusotine) is a new once-daily oral medicine approved to treat adults with acromegaly who either didn’t get much help from surgery or cannot have surgery for certain reasons. Acromegaly is a rare condition caused by too much growth hormone (GH), usually due to a tumor in the pituitary gland. This increases levels of another substance called insulin-like growth factor 1 (IGF-1) that can damage bones, joints, the heart, and other vital organs over time. Symptoms of acromegaly include joint pain, tiredness, swelling, headaches, bigger hands and feet, and changes in the way you look. If not treated, acromegaly can also affect your heart and other organs, shorten your life span, and affect your everyday activities.
Palsonify works by mimicking a natural hormone called somatostatin and targets a specific protein called somatostatin receptor type 2, helping lower GH and IGF-1 levels. This eases the symptoms and possibly slows the worsening of the disease.
Unlike current injection-based treatments, Palsonify is a once-daily oral pill that offers a simpler and more convenient way to treat acromegaly, manage your treatment plan, and improve your daily life.
Why Was It Approved?
Palsonify was approved because studies showed it was safe, effective, and well-tolerated and provided quick, reliable, and longer-lasting hormone control in adults with acromegaly.
In one study, people with acromegaly who had not undergone any prior treatment were given Palsonify or a placebo once every day. After 24 weeks, significantly more people taking Palsonify vs. a placebo (56% vs 5%) had IGF-1 hormone levels in the normal range. Palsonify improved symptomatic score by 2.67 points, while placebo worsened it by 2.75 points. Also, 57% of people taking Palsonify vs. 18% of those taking a placebo achieved low GH levels.
In another study, people with acromegaly who had already been using injectable treatments switched to Palsonify or placebo. After 36 weeks, IGF-1 levels were maintained under control in people who took Palsonify vs. 4% who took a placebo. Symptoms stayed stable or improved in the Palsonify group but worsened in the placebo group; 87% of those in the Palsonify group vs. 28% in the placebo group were able to maintain low GH levels.
People taking this medicine also said they felt better overall, with fewer symptoms like joint pain, tiredness, and headaches. The most common side effects included diarrhea, stomach pain, nausea, loss of appetite, slow heart rate, high blood sugar, heart palpitations, and stomach infections. Serious side effects were rare and included gall bladder issues, fluctuations in blood sugar levels, slow heart rate, heart-related problems, poor absorption of fats or vitamin B12, and impaired thyroid function. But no new safety concerns were found during the studies.
What Do I Need to Know?
You take Palsonify once a day by mouth with water. It should be taken on an empty stomach at least six hours after your last meal and one hour before your next one. The usual starting dose is 40 milligrams, but your health care provider may adjust it based on your IGF-1 hormone levels.
Some medicines, like those for acid reflux or immune suppression, can affect Palsonify’s action or vice versa. Be sure to tell your health care provider about your medical history and all the medicines you take, including over-the-counter products, herbal remedies, and vitamin supplements. Your provider may adjust your dose or monitor you closely. Also, let them know if you are pregnant, planning to get pregnant, or breastfeeding as it is not known if this medicine harms a fetus or passes into breast milk.
This medicine can cause side effects like gallstones, blood sugar changes, slow heart rate, thyroid issues, and trouble absorbing fats or vitamin B12, so your health care provider will monitor you for these during treatment. Let your provider know right away if you feel very tired or dizzy or faint; notice an irregular heartbeat, yellowing of the skin or eyes (jaundice), or dark urine; or have pain in your abdomen; digestive issues like bloating or fatty, pale-colored, and loose stools; or tingling or numbness in your hands and feet.
This medicine offers a new option for treating acromegaly that doesn’t require monthly injections. But it’s important to follow your provider’s instructions and go for regular checkups to make sure the medicine is working safely and effectively.
It is expected to be available in the U.S. in early October.