1 What is Orkambi?

Brand: Orkambi

Generic: Lumacaftor and Ivacaftor

Orkambi is used in the treatment of cystic fibrosis in patient 12 years of age and older.

Your doctor may need a medical test to make sure that you have cystic fibrosis transmembrane conductance regulator (CFTR) gene mutation before starting the treatment.

It is available in the form of the tablet only with your doctor’s prescription.

2 What to Know Before Using

Your doctor will inform you of what you need to know before using Orkambi.

Before starting the treatment your doctor should be informed about condition such as:

Allergy: Inform your doctor if you are allergic to any food, preservatives, dyes, or had an allergic reaction when you used this drug before.

Children: There are no adequate studies done which prove Lumacaftor and Ivacaftor have harmful effects in children. However, safety and efficacy have not been established in children younger than 12 years of age.

Elderly: This medicine does not cause any different side effects or problem than it does in younger adults.

Pregnancy: It is a category B drug. It is safe to use in pregnancy.

Breast Feeding: There are no adequate studies which prove that Lumacaftor and Ivacaftor pass into breast milk. If you are using this drug while nursing, discuss it with your doctor for possible risk factors.

Drug Interactions: Lumacaftor and Ivacaftor may interact with other drugs. Your doctor should be informed of any other medicines which you are taking. He may want to change the dosage or drug.

Some of them are listed below:

  • Rifabutin or rifampin
  • St. John's wort
  • Medicine to prevent organ transplant rejection: Everolimus (zortress), sirolimus, tacrolimus.
  • A sedative: Triazolam or oral midazolam.
  • Seizure medicine: Carbamazepine, phenobarbital, phenytoin.
  • Colchicine

Several other conditions may reduce its effectiveness.

Make sure you inform your doctor of conditions such as:

3 Proper Usage

Proper usage of Orkambi requires strict adherence to your doctor’s orders. Read the medication guide carefully. Ask your pharmacist if you have any queries.

Take the medicine every 12 hours with a fat-containing food such as butter, cheese, eggs, whole milk, avocado, pizza. Your doctor may need to change your dosage if you stop taking it for 1 week.

Dosage: It is different for different patients. Dosage mentioned below is an average dose.it is best to take each dose at equally spaced times a day and night. You must follow your doctor’s command.

Adults and children 12 years of age and older: 2 tablets every 12 hours apart. Each tablet contains Lumacaftor 200 milligram (mg) and 125 mg of Ivacaftor.

Missed dose: If you miss a dose, take it as soon as you remember it. If it is almost a time for your next dose, skip the missed dose and continue your daily schedule. Do not double the dose.

Storage: Store it at room temperature. Keep it away from children

4 Precautions to Take

Before using DRUG, there are some precautions you must take. Your doctor should check your daily progress at regular visits. Your doctor may need your Blood test to check for any adverse effects.

Use a birth control method other than hormone based such as condoms, diaphragm, contraceptive foams, and jellies. The hormone-based contraceptive is not effective enough to prevent pregnancy while using this drug.

5 Potential Side Effects

As with many medications, there are several potential side effects associated with Orkambi. Some people may experience none or minor side effects.

Consult with your doctor if they become problematic or frequent:

More common are:

  • Breathing problems
  • Feeling tired
  • Nausea, gas
  • Diarrhea
  • Rash
  • Flu symptoms
  • Changes in your menstrual periods.
  • Cold symptoms such as the runny or stuffy nose, sinus pain, sore throat.

Some severe side effects are:

  • Shortness of breath,
  • Tightness in your chest
  • Nausea
  • Upper stomach pain
  • Itching
  • Loss of appetite
  • Dark urine
  • Clay-colored stools
  • Jaundice (yellowing of the skin or eyes).

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.

6 Related Clinical Trials