Osilodrostat

Osilodrostat is used in adults to treat Cushing's disease that cannot be treated with pituitary surgery.

Osilodrostat may also be used for reasons not mentioned in this medication guide.

Follow all directions on your prescription label and read all medication guides or instruction sheets. Your doctor may occasionally change your dose. Use the medicine exactly as directed.

You may take osilodrostat with or without food.

Your doctor will check your heart function using an electrocardiograph or ECG (sometimes called an EKG). Your next dose may be delayed based on the results of your ECG or other medical tests.

Do not change your dose or dosing schedule without your doctor's advice.

Store at room temperature away from moisture and heat.

Osilodrostat can cause serious side effects, such as heartbeat problems or decreased adrenal gland hormones. Call your doctor right away if you have irregular heartbeats, stomach pain, vomiting, loss of appetite, tiredness, or feel light-headed.

Tell your doctor if you have ever had:

• a heart rhythm disorder;
• long QT syndrome (in you or a family member);
• congestive heart failure;
• high blood pressure;
• an electrolyte imbalance (such as low levels of potassium or magnesium in your blood);
• fluid retention; or
• liver disease.

Tell your doctor if you are pregnant or plan to become pregnant. It is not known whether osilodrostat will harm an unborn baby. However, having Cushing's disease during pregnancy may cause complications such as diabetes or eclampsia (dangerously high blood pressure that can lead to medical problems in both mother and baby). The benefit of treating Cushing's disease may outweigh any risks to the baby.

Do not breastfeed while using osilodrostat, and for at least 1 week after your last dose.

Osilodrostat is not approved for use by anyone younger than 18 years old.

Get emergency medical help if you have signs of an allergic reaction: hives; difficult breathing; swelling of your face, lips, tongue, or throat.

Osilodrostat may cause serious side effects. Call your doctor right away if you have:

• fast or pounding heartbeats, fluttering in your chest, shortness of breath, and sudden dizziness (like you might pass out);
• swelling in your feet or ankles, feeling short of breath;
• decreased adrenal gland hormones--nausea, vomiting, stomach pain, loss of appetite, feeling tired or light-headed, muscle or joint pain, skin discoloration, craving salty foods;
• increased adrenal gland hormones--hunger, weight gain, swelling, skin discoloration, slow wound healing, sweating, acne, increased body hair, tiredness, mood changes, muscle weakness, missed menstrual periods, sexual changes; or
• low potassium level--leg cramps, constipation, irregular heartbeats, fluttering in your chest, increased thirst or urination, numbness or tingling, muscle weakness or limp feeling.

Common side effects of osilodrostat may include:

• nausea;
• feeling tired;
• headache; or
• swelling.

This is not a complete list of side effects and others may occur. Call your doctor for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088.

Usual Adult Dose for Cushing's Syndrome:

Initial dose: 2 mg orally twice a dayTitration: Initially by 1 to 2 mg twice a day, no more frequently than every 2 weeks-Further titration (after reaching 10 mg orally twice a day): By 5 mg twice a day every 2 weeksMaximum dose: 30 mg orally twice a dayComments:-The dose should be titrated based on rate of cortisol changes, individual tolerability, and individual improvement in signs/symptoms of Cushing's disease.-If a patient tolerates 10 mg twice a day and continues to have elevated 24-hour urine free cortisol (UFC) level above the upper limit of normal (ULN), the dosage can be titrated further by 5 mg twice a day every 2 weeks.-Cortisol levels should be monitored from at least two 24-hour UFC collections every 1 to 2 weeks until adequate clinical response is maintained.-The maintenance dosage is individualized and determined by titration based on cortisol levels and patient's signs/symptoms; the maintenance dosage varied between 2 and 7 mg twice a day in clinical trials.-Once maintenance dosage achieved, cortisol levels should be monitored at least every 1 to 2 months or as indicated.Use: For the treatment of patients with Cushing's disease for whom pituitary surgery is not an option or has not been curative

Skip the missed dose and take your next dose at the usual time. Do not take two doses at the same time.

Seek emergency medical attention or call the Poison Help line at 1-800-222-1222.

Overdose symptoms may include nausea, vomiting, stomach pain, extreme tiredness, feeling light-headed, or fainting.

Follow your doctor's instructions about any restrictions on food, beverages, or activity.

Osilodrostat can lead to a serious heart issue. Your risk might increase if you are also taking certain other medicines for infections, asthma, heart problems, high blood pressure, depression, mental illness, cancer, malaria, or HIV.

Other medications may impact osilodrostat, including both prescription and over-the-counter medicines, vitamins, and herbal products. Inform your doctor about all your current medications and any new ones you begin or stop using.