What Are the Possible Side Effects of ORKAMBI®?

Watch a video to learn about the possible side effects of ORKAMBI. You can also read about the possible side effects of ORKAMBI below.

How the safety of ORKAMBI was studied in clinical trials

Two 24-week trials were done to evaluate the safety and efficacy of ORKAMBI in people age 12 years and older with 2 copies of the F508del mutation.

Across both trials, a total of 369 people took ORKAMBI every 12 hours with fat-containing food, and 370 received placebo. Both groups continued to take all of their other prescribed cystic fibrosis (CF) therapies.

The safety data from the 2 clinical trials were combined and reviewed together, or “pooled,” to arrive at the results.

Remember, this website is for informational purposes only and is not intended to be a substitute for professional medical advice. You should talk to your healthcare provider about the clinical trial results and the possible side effects of ORKAMBI.

ORKAMBI can cause serious side effects

High liver enzymes in the blood, which can be a sign of liver injury, have been reported in people receiving ORKAMBI. In two 24-week clinical trials, less than 1% of people taking ORKAMBI experienced serious side effects related to high liver enzymes.

Your healthcare provider will do blood tests to check your liver:

  • Before you start ORKAMBI
  • At 3, 6, 9, and 12 months after starting treatment
  • Every year thereafter while you are taking ORKAMBI

You may be able to get these tests done during your regular CF Center visits. Talk to your healthcare provider about how best to schedule these tests.

Call your healthcare provider 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
  • Dark, amber-colored urine
  • Confusion

Respiratory events such as shortness of breath or chest tightness were observed in patients when starting ORKAMBI. The majority of respiratory events began during the first week of treatment and were more common in people who had poor lung function. If you have poor lung function, your healthcare provider may monitor you more closely when you start ORKAMBI.


In two 24-week clinical trials, 9% of people experienced chest tightness compared with 6% taking placebo. 13% of people experienced shortness of breath compared with 8% taking placebo.

For people who had respiratory events within 1 to 2 days after starting ORKAMBI and who continued treatment, the majority of these events resolved within the first 2 to 3 weeks of taking ORKAMBI.

If you experience any of these side effects, talk to your healthcare provider right away.


An increase in blood pressure has been seen in some patients treated with ORKAMBI. Your doctor should monitor your blood pressure during treatment with ORKAMBI.

Abnormality of the eye lens (cataract) has been noted in some children and adolescents receiving ORKAMBI and ivacaftor, a component of ORKAMBI.

If you are a child or adolescent, your healthcare provider should perform eye examinations prior to and during treatment with ORKAMBI to look for cataracts.

What are the most common side effects of ORKAMBI?

Below are the most common side effects of ORKAMBI and percentage of people who experienced them during two 24-week clinical trials.*

Select a side effect from the table below to learn more.

SIDE EFFECT PEOPLE TAKING ORKAMBI PEOPLE TAKING PLACEBO
Respiratory events
Respiratory Icon
Shortness of breath 13% 8%
Chest tightness 9% 6%
Respiratory tract infections
Tissue Icon
Common cold 13% 11%
Upper respiratory tract infection 10% 5%
Runny nose 6% 4%
Gastrointestinal events
Gastro Icon
Nausea 13% 8%
Diarrhea 12% 8%
Gas 7% 3%
Other events
Woman Icon
Irregular, missed, or abnormal periods and increase in the amount of menstrual bleeding
10% 2%

Some women taking ORKAMBI had irregular, missed, or abnormal periods or an increase in the amount of menstrual bleeding. These events occurred more frequently in women taking ORKAMBI who were using hormonal contraceptives (27%) than those not using hormonal contraceptives (3%).

Hormonal contraceptives, including oral, injectable, transdermal, or implantable forms should not be used as a method of birth control when taking ORKAMBI. Talk to your doctor about the best birth-control method you should use while taking ORKAMBI.

Fatigue IconFatigue Icon
Fatigue
9% 8%

Fatigue is the feeling of exhaustion that can last a long time and doesn’t feel relieved by rest or sleep.

Vial Icon
Elevated muscle enzymes
7% 5%

High blood levels of the enzymes usually found in your muscles can be a sign of muscle stress or injury.

Rash Icon
Rash 7% 2%
Flu Icon
Flu or flu-like symptoms 5% 2%

*Both groups continued to take all of their other prescribed CF therapies.
These events occurred more frequently in women taking ORKAMBI who were using hormonal contraceptives (27%) than those not using hormonal contraceptives (3%).

Tell your doctor if you have any side effect that bothers you or does not go away.

These are not all the possible side effects of ORKAMBI. For more information, ask your doctor or pharmacist. Call your doctor for medical advice about side effects. You are encouraged to report side effects to the FDA at 1-800-FDA-1088.


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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 ORKAMBI.

Your child's doctor will do blood tests to check your child's liver:

  • Before starting ORKAMBI
  • Every 3 months during the first year of taking ORKAMBI
  • Every year while taking ORKAMBI

Call your child's doctor right away if he or she has any of the following symptoms of liver problems:

  • Pain or discomfort in the upper right stomach (abdominal) area
  • Yellowing of the skin or the white part of the eyes
  • Loss of appetite
  • Nausea or vomiting
  • Dark, amber-colored urine
  • Confusion

Respiratory events such as shortness of breath or chest tightness were observed in patients when starting ORKAMBI. If your child has poor lung function, your child's doctor may monitor him or her more closely when starting ORKAMBI.


An increase in blood pressure has been seen in some patients treated with ORKAMBI. Your child's doctor should monitor your child's blood pressure during treatment with ORKAMBI.


Abnormality of the eye lens (cataract) has been noted in some children and adolescents receiving ORKAMBI and ivacaftor, a component of ORKAMBI. Your child’s doctor should perform eye examinations prior to and during treatment with ORKAMBI to look for cataracts.



The most common side effects of ORKAMBI include: shortness of breath and/or chest tightness; upper respiratory tract infection (common cold), including sore throat, stuffy or runny nose; gastrointestinal symptoms, including nausea, diarrhea, or gas; rash; fatigue; flu or flu-like symptoms; increase in muscle enzyme levels; and irregular, missed, or abnormal periods (menses) and increase in the amount of menstrual bleeding.


Tell your child’s doctor if he or she has any side effect that is bothersome or does not go away.

These are not all the possible side effects of ORKAMBI. For more information, ask your child’s doctor or pharmacist. Call your child’s doctor for medical advice about side effects. You are encouraged to report side effects to FDA at 1-800-FDA-1088.