KALYDECO is the first and only
FDA-approved CFTR potentiator

For patients with cystic fibrosis age 6 years and older who have a G551D mutation

What is KALYDECO?

KALYDECO is a prescription medicine used for the treatment of cystic
fibrosis (CF) in patients age 6 years and older who have a certain mutation in their
CF gene called the G551D mutation.

KALYDECO is not for use in people with CF due to other mutations in the CF gene. It is not
effective in patients with CF with 2 copies of the F508del mutation (F508del/F508del) in the
CF gene.

It is not known if KALYDECO is safe and effective in children under 6 years of age.

Important Safety Information

Who should not take KALYDECO?

KALYDECO should not be used with certain medicines or herbal supplements. Talk to your doctor before taking KALYDECO if you are taking the antibiotics rifampin (Rifamate^®, Rifater^®) or rifabutin (Mycobutin^®); seizure medications such as phenobarbital, carbamazepine (Tegretol^®, Carbatrol^®, Equetro^®) or phenytoin (Dilantin^®, Phenytek^®); or St. John’s Wort.

What should I tell my doctor before taking KALYDECO?

Before you take KALYDECO, tell your doctor if you:

• have liver or kidney problems
• drink grapefruit juice, or eat grapefruit or Seville oranges
• are pregnant or plan to become pregnant. It is not known if KALYDECO will harm your unborn
  baby. You and your doctor should decide if you will take KALYDECO while you are pregnant
• are breastfeeding or planning to breastfeed. It is not known if KALYDECO passes into your breast
  milk. You and your doctor should decide if you will take KALYDECO while you are breastfeeding

KALYDECO may affect the way other medicines work, and other medicines may affect how KALYDECO works.

Tell your doctor about all the medicines you take, including prescription and non-prescription medicines, vitamins, and herbal supplements as the dose of KALYDECO may need to be adjusted when taken with certain medications.

Especially tell your doctor if you take antifungal medications such as ketoconazole (e.g., Nizoral^®), itraconazole (e.g., Sporanox^®), posaconazole (e.g., Noxafil^®), voriconazole (e.g., Vfend^®), or fluconazole (e.g., Diflucan^®); or antibiotics such as telithromycin (e.g., Ketek^®), clarithromycin (e.g., Biaxin^®), or erythromycin (e.g., Ery-Tab^®).

What should I avoid while taking KALYDECO?

KALYDECO can cause dizziness in some people who take it. Do not drive a car, use machinery, or do anything that needs you to be alert until you know how KALYDECO affects you.
You should avoid food containing grapefruit or Seville oranges while you are taking KALYDECO.

What are the possible side effects of KALYDECO?

• KALYDECO can cause serious side effects
• High liver enzymes in the blood have been reported in patients receiving KALYDECO. Your
  doctor will do blood tests to check your liver before you start KALYDECO, every 3 months during your first year of therapy, and every year while you are taking KALYDECO
  Call your doctor right away if you have any of the following symptoms of liver problems: pain or discomfort in the upper right stomach (abdominal) area; yellowing of your skin or the white part of your eyes; loss of appetite; nausea or vomiting; or dark, amber-colored urine
• The most common side effects include headache; upper respiratory tract infection (common cold), which includes sore throat, nasal or sinus congestion, and runny nose; stomach (abdominal) pain; diarrhea; rash; nausea; and dizziness

Tell your doctor if you have any side effect that bothers you or that does not go away. These are not all the possible side effects of KALYDECO. For more information, ask your doctor or pharmacist.

You are encouraged to report negative side effects of prescription drugs to the FDA at www.fda.gov/medwatch or call 1-800-FDA-1088.

KALYDECO is pronounced kuh-LYE-deh-koh.

Please see the Full U.S. Prescribing Information and Patient Information on this website.

Reference

KALYDECO [package insert]. Cambridge, MA: Vertex Pharmaceuticals Incorporated; 2012.