Orkambi

Orkambi is used to treat certain types of cystic fibrosis (an inborn disease that causes problems with breathing, digestion, and reproduction) in adults and children 1 year of age and older.

Orkambi tablets can be used in children aged 6 years and older.

Orkambi granules can be used in children at least 1 year old.

Your doctor will make sure you have the specific gene mutation related to cystic fibrosis to be treated with Orkambi.

Lumacaftor belongs to a class of medications called cystic fibrosis transmembrane conductance regulator (CFTR) correctors and ivacaftor belongs to a class of medications called cystic fibrosis transmembrane conductance regulator (CFTR) potentiators.

Orkambi works by improving the function of a protein in the body to decrease the build-up of thick mucus in the lungs and improving other symptoms of cystic fibrosis.

Take Orkambi exactly as prescribed by your doctor. Follow all directions on your prescription label and read all medication guides or instruction sheets. Your doctor may occasionally change your dose.

Take this medicine with food that contains fat, such as eggs, milk products such as whole milk, cheese, yogurt, breast milk or infant formula. Follow your doctor's instructions carefully.

Swallow the tablet whole and do not crush, chew, or break it.

Mix the Orkambi granules with 1 teaspoon of soft food or liquid such as pureed fruits or vegetables, yogurt, applesauce, water, breast milk, infant formula, milk, or juice. The food or liquid should be at or below room temperature. Mix only 1 dose at a time and use within 1 hour after mixing. Feed the child a high-fat food after or before giving the granule mixture.

Take Orkambi at evenly spaced intervals every 12 hours.

Doses are based on weight in children. Your child's dose may change if the child gains or loses weight.

You will need frequent blood tests to check your liver function, and your blood pressure may need to be checked often. You may also need other medical tests.

Your child may need frequent vision exams.

Store Orkambi at room temperature away from moisture and heat. Keep the medicine in the original packaging.

Orkambi can cause serious side effects. High liver enzymes in the blood, which can be a sign of liver injury, have been reported in patients receiving this medicine. Your doctor will do blood tests to check your liver.

Serious drug interactions can occur when certain medicines are used together with Orkambi. Tell each of your healthcare providers about all medicines you use now, and any medicine you start or stop using.

You should not be treated with Orkambi if you are allergic to ivacaftor or lumacaftor.

To make sure Orkambi is safe for you, tell your doctor if you have ever had:

• high blood pressure;
• lung disease;
• an organ transplant; or
• liver or kidney disease.
• mental health problems.

It is not known if ivacaftor and lumacaftor will harm an unborn baby. Tell your doctor if you are pregnant or plan to become pregnant.

Ask a doctor if it is safe to breastfeed while using this medicine.

Orkambi can make hormonal birth control less effective, including birth control pills, injections, implants, skin patches, and vaginal rings. Use barrier birth control to prevent pregnancy: condom, diaphragm, cervical cap, or contraceptive sponge.

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

Orkambi may cause serious side effects. Call your doctor at once if you have:

• severe headache, blurred vision, pounding in your neck or ears;
• eye pain or redness, seeing halos around lights;
• chest tightness, trouble breathing; or
• liver problems - loss of appetite, nausea, vomiting, confusion, stomach pain (upper right side), tiredness, itching, dark urine, clay-colored stools, jaundice (yellowing of the skin or eyes).
• mental health problems - anxiety, depression, suicidal thoughts or behaviors, or trouble sleeping.

Common Orkambi side effects may include:

• breathing problems;
• feeling tired;
• nausea, gas, diarrhea;
• rash;
• changes in your menstrual periods, heavy menstrual bleeding;
• flu or cold symptoms such as stuffy or runny nose, sneezing, sore throat; or
• abnormal blood tests.

Some side effects are more common in children taking Orkambi such as increase in mucus, cough with mucus, headache, or stomach pain.

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 Cystic Fibrosis:

Lumacaftor 400 mg/Ivacaftor 250 mg orally every 12 hours with fat-containing food Comments: -A fat containing meal or snack should be consumed just before or just after dosing. Examples include eggs, avocados, nuts, butter, peanut butter, cheese pizza, or whole-milk dairy products. -If the patient's genotype is unknown, an FDA-cleared CF mutation test should be used to detect the presence of the F508del mutation on both alleles of the CFTR gene; safety and efficacy in patients with cystic fibrosis other than homozygous F508del mutation has not been established. Use: For the treatment of cystic fibrosis (CF) in patients who are homozygous for the F508del mutation in the CFTR gene.

Usual Pediatric Dose for Cystic Fibrosis:

Age: 1 through 2 years; weight 7 kg to less than 9 kg: Lumacaftor 75 mg/Ivacaftor 94 mg orally every 12 hours with fat-containing food Age: 1 through 2 years; weight 9 kg to less than 14 kg: Lumacaftor 100 mg/Ivacaftor 125 mg orally every 12 hours with fat-containing food Age: 1 through 2 years; weight 14 kg or greater: Lumacaftor 150 mg/Ivacaftor 188 mg orally every 12 hours with fat-containing food Age: 2 through 5 years; weight less than 14 kg: Lumacaftor 100 mg/Ivacaftor 125 mg orally every 12 hours with fat-containing food Age: 2 through 5 years: weight 14 kg or greater: Lumacaftor 150 mg/Ivacaftor 188 mg orally every 12 hours with fat-containing food Age: 6 through 11 years: Lumacaftor 200 mg/Ivacaftor 250 mg orally every 12 hours with fat-containing food Age: 12 years or older: Lumacaftor 400 mg/Ivacaftor 250 mg orally every 12 hours with fat-containing food Comments: -Pediatric patients 1 through 5 years of age should receive oral granules; pediatric patients 6 years of age and older should be dosed with tablets. -A fat containing meal or snack should be consumed just before or just after dosing. Examples include eggs, avocados, nuts, butter, peanut butter, cheese pizza, or whole-milk dairy products. -If the patient's genotype is unknown, an FDA-cleared CF mutation test should be used to detect the presence of the F508del mutation on both alleles of the CFTR gene; safety and efficacy in patients with cystic fibrosis other than homozygous F508del mutation has not been established. Use: For the treatment of cystic fibrosis (CF) in patients 1 year or older who are homozygous for the F508del mutation in the CFTR gene.

Take the medicine as soon as you can, with a fatty meal, but skip the missed dose if you are more than 6 hours late for the dose. Do not take two doses at one time.

Call your doctor for instructions if you stop taking Orkambi for more than 1 week.

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

Grapefruit may interact with ivacaftor and lumacaftor and cause side effects. Avoid consuming grapefruit products during the first week of treatment.

Sometimes it is not safe to use certain medicines at the same time. Some drugs can affect your blood levels of other drugs you use, which may increase side effects or make the medicines less effective.

Tell your doctor about all your current medicines. Many drugs can affect ivacaftor and lumacaftor, especially:

• St. John's wort;
• seizure medicine such as carbamazepine, phenobarbital, phenytoin;
• medicine to prevent organ transplant rejection - cyclosporine, everolimus (Zortress), sirolimus, tacrolimus;
• a sedative - triazolam or midazolam;
• an antibiotic> - clarithromycin, erythromycin, telithromycin, rifabutin, rifampin; or
• antifungal medicine - itraconazole, ketoconazole, posaconazole, voriconazole.

This list is not complete. Other drugs may interact with ivacaftor and lumacaftor, including prescription and over-the-counter medicines, vitamins, and herbal products. Not all possible drug interactions are listed here.

Active ingredients: lumacaftor and ivacaftor Orkambi tablets: Inactive ingredients: cellulose, microcrystalline; croscarmellose sodium; hypromellose acetate succinate; magnesium stearate; povidone; and sodium lauryl sulfate. The tablet film coat contains: carmine, FD&C Blue #1, FD&C Blue #2, polyethylene glycol, polyvinyl alcohol, talc, and titanium dioxide. The printing ink contains: ammonium hydroxide, iron oxide black, propylene glycol, and shellac. Orkambi oral granules Inactive ingredients: cellulose, microcrystalline, croscarmellose sodium, hypromellose acetate succinate, povidone, and sodium lauryl sulfate.

Manufactured for: Vertex Pharmaceuticals Incorporated, 50 Northern Avenue, Boston, MA 02210, USA.

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