ENCOURAGEMENT AND SUPPORT FROM PEOPLE WHO KNOW

ISTURISA^® (osilodrostat) is a prescription medicine used to treat elevated levels of cortisol in the blood (endogenous hypercortisolemia) in adults with Cushing’s syndrome (CS) who cannot have surgery, or who have had surgery which did not cure their CS. It is not known if ISTURISA is safe and effective in children.

A benign (noncancerous) tumor in your pituitary gland (Cushing’s disease); or a benign or malignant (cancerous) tumor in your adrenal gland(s); or a tumor outside of your pituitary or adrenal gland(s) that causes your body produce too much of the hormone, cortisol.

The primary treatment goal for patients with Cushing’s syndrome is to bring the body’s cortisol levels to a normal range.

ISTURISA is the first FDA-approved medicine for Cushing's syndrome that works by blocking an enzyme that helps make cortisol. In turn, the adrenal glands make less cortisol, and less cortisol is released into your bloodstream. This may help you reach and maintain normal cortisol levels.^a ISTURISA may cause side effects. You can find more information about side effects here.

ISTURISA is a tablet that's taken twice a day. ISTURISA is for adult patients with Cushing's syndrome who can't have surgery, or who have had surgery but were not cured of their Cushing's syndrome.

The safety and effectiveness of ISTURISA were studied in a 48-week clinical trial with 137 patients with Cushing's disease that involved a dose titration period, a maintenance dose period, a randomized withdrawal period, and an open-label period during which all patients received ISTURISA. Period 3 (weeks 26 to 34) was the 8-week, randomized, double-blind withdrawal period where patients were randomly assigned to receive ISTURISA or placebo. At the end of Period 3, 31 out of 36 patients who received ISTURISA maintained normal cortisol levels vs 10 out of 34 patients who received placebo. Individual results may vary.

While you are taking ISTURISA, your healthcare provider will do regular testing to ensure that you are reaching and maintaining normal cortisol levels. Monitoring your cortisol levels will help your healthcare provider determine if any changes in your ISTURISA dosage are required.

You should also tell your healthcare provider how you're feeling during your treatment with ISTURISA. This information will also be used to help optimize your treatment plan.

ISTURISA can cause serious side effects, including:

• Low cortisol levels in your blood (hypocortisolism)

  Tell your healthcare provider right away if you experience any of the following symptoms, as these may be symptoms of very low cortisol level, known as adrenal insufficiency:

• nausea
• vomiting
• tiredness (fatigue)
• low blood pressure
• problems with body salt (electrolyte) levels in your blood
• stomach (abdominal) pain
• loss of appetite
• dizziness
• low blood sugar

If you get symptoms of hypocortisolism while taking ISTURISA, your healthcare provider may change your dose or ask you to stop taking it.

• Heart problem or a heart rhythm problem, such as an irregular heartbeat which could be a sign of a heart problem called QT prolongation. Call your healthcare provider right away if you have irregular heartbeats.

• Increase in other adrenal hormone levels. Your other adrenal hormones may increase when you take ISTURISA. Your healthcare provider may monitor you for the symptoms associated with these hormonal changes while you are taking ISTURISA:
  • Low potassium (hypokalemia)
  • High blood pressure (hypertension)
  • Swelling (edema) in the legs, ankles or other signs of fluid retention
  • Excessive facial or body hair growth (hirsutism)
  • Acne (in women)

Call your healthcare provider if you have any of these side effects.

The most common side effects of ISTURISA include:

• very low cortisol levels (adrenal insufficiency)
• tiredness (fatigue)
• nausea
• headache
• swelling of the legs, ankles or other signs of fluid retention (edema) decreased appetite
• pain in your joints (arthralgia)
• muscle pain or soreness (myalgia)
• weakness (asthenia)
• diarrhea

These are not all of the possible side effects of ISTURISA.

Call your healthcare provider for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088 or visit www.fda.gov/medwatch.

The R.A.R.E.^® Patient Support Program is designed to make it easier for you to start and stay on your treatment. With R.A.R.E., insurance specialists will support you by completing a benefits investigation to help you understand your insurance coverage.

R.A.R.E. can help commercially insured patients get their medication for no more than $20 per month.^b

For patients who are not eligible for co-pay support or who need additional financial assistance, R.A.R.E. can help connect you with alternative forms of medication coverage, or provide referrals to other possible sources of funding.^b

Patient Assistance Program—Patients who lack insurance and meet certain financial requirements may be eligible for additional financial support from a Patient Assistance Program.^b

Restrictions, limitations, and/or eligibility requirements apply.

Shortly after beginning treatment, a specially trained R.A.R.E. Nurse Educator will call you to ensure your treatment is off to a good start, answer general questions about Cushing’s syndrome or ISTURISA, and discuss your treatment goals. You can also look forward to regular phone calls from your R.A.R.E. Nurse Educator. Information provided by the R.A.R.E. Nurse Educator is for educational purposes only and is not intended to replace the advice of your healthcare provider.

To help you stay on track with your treatment, the specialty pharmacy will remain in contact with you regarding medication refills and deliveries. You can also speak to a clinical pharmacist over the phone 24 hours a day, 7 days a week by calling 1-888-855-RARE (7273).