Why is this medication prescribed?

Orkambi is a medication prescribed for the treatment of cystic fibrosis (CF) in patients who have a specific genetic mutation known as the F508del mutation. CF is a genetic disorder that affects the production and function of mucus, digestive juices, and sweat. It primarily affects the lungs, digestive system, and other organs.

Orkambi is a combination drug that contains two active ingredients: lumacaftor and ivacaftor. Lumacaftor helps to improve the processing and trafficking of the defective protein that causes CF, while ivacaftor enhances the function of the defective protein once it reaches the cell surface. By addressing the underlying genetic defect, Orkambi aims to improve lung function and reduce symptoms associated with CF.

This medication is specifically indicated for individuals with CF aged 6 years and older who have two copies of the F508del mutation in their cystic fibrosis transmembrane conductance regulator (CFTR) gene. It is important to note that Orkambi is not effective for individuals with CF who have different genetic mutations.

How should this medicine be used?

Orkambi is typically available in the form of tablets, and the recommended dosage and administration instructions should be provided by a healthcare professional. However, I can provide you with general guidelines for the use of Orkambi. It’s important to note that these guidelines may not be applicable to every individual, so it’s crucial to follow the specific instructions given by your doctor.

Here are general guidelines for the use of Orkambi:

• Dosage: The typical dosage for Orkambi is one tablet taken orally every 12 hours (twice daily).
• Administration: Orkambi tablets should be taken with fat-containing food. This is important because it helps with the absorption of the medication. Examples of fat-containing foods include eggs, butter, cheese, avocados, and nuts. It is recommended to take Orkambi with a meal or snack that contains about 20 grams of fat.
• Swallowing: The tablets should be swallowed whole and not crushed, chewed, or dissolved. If you have difficulty swallowing tablets, talk to your doctor for alternative options.
• Adherence: It’s crucial to take Orkambi as prescribed by your doctor and at the specified times. Consistency in taking the medication is important to achieve the desired therapeutic effects.
• Other medications: If you are taking other medications, it’s important to inform your doctor. Some medications may interact with Orkambi and require adjustment of dosages or additional monitoring.
• Follow-up appointments: Regular follow-up appointments with your healthcare provider are important to monitor your response to Orkambi, assess its effectiveness, and manage any potential side effects.

Always consult your healthcare provider for personalized instructions on how to use Orkambi correctly. They will provide you with the most accurate and up-to-date information based on your specific medical condition and needs.

Other uses for this medicine

Orkambi is specifically approved for the treatment of cystic fibrosis (CF) in patients who have two copies of the F508del mutation in their cystic fibrosis transmembrane conductance regulator (CFTR) gene. It is designed to address the underlying genetic defect in CF and is not indicated for other conditions.

What special precautions should I follow?

Regarding special precautions, it’s important to note the following:

• Genetic testing: Orkambi is specifically targeted for individuals with CF who have the F508del mutation. Genetic testing is necessary to confirm the presence of this mutation before prescribing Orkambi.
• Age restrictions: Orkambi is approved for use in individuals aged 6 years and older. It should not be used in children younger than 6 years or in individuals without the specific genetic mutation mentioned above.
• Liver function: Orkambi may affect liver function, and therefore, liver function tests should be conducted prior to starting treatment and regularly monitored during therapy. If there are any abnormalities in liver function, the dose may need to be adjusted or treatment discontinued.
• Respiratory symptoms: Orkambi has been associated with an increased risk of respiratory symptoms such as shortness of breath and chest tightness. If these symptoms occur, it is important to contact a healthcare professional for further evaluation.
• Drug interactions: Orkambi may interact with other medications, potentially affecting their effectiveness or increasing the risk of side effects. It is important to inform your healthcare provider about all the medications, supplements, and herbal products you are taking before starting Orkambi.
• Pregnancy and breastfeeding: The safety and effectiveness of Orkambi have not been established in pregnant women or nursing mothers. If you are pregnant, planning to become pregnant, or breastfeeding, it is crucial to discuss the potential risks and benefits with your healthcare provider.

These precautions are not exhaustive, and it’s essential to consult with a healthcare professional for comprehensive information and guidance specific to your individual circumstances before starting Orkambi. They can provide personalized advice based on your medical history, current medications, and any other relevant factors.

What special dietary instructions should I follow?

Orkambi should be taken with fat-containing food. Consuming fat-containing foods around the time of taking Orkambi helps with the absorption of the medication. Examples of fat-containing foods include eggs, butter, cheese, avocados, and nuts. Aim to have a meal or snack that contains about 20 grams of fat when taking Orkambi.

What should I do if I forget a dose?

If you forget to take a dose of Orkambi, the general recommendation is to take it as soon as you remember, as long as it is within a few hours of the missed dose. However, if it is close to the time for your next scheduled dose, it is advised to skip the missed dose and continue with your regular dosing schedule. Taking a double dose to make up for a missed dose is not recommended.

What side effects can this medication cause?

Orkambi, like any medication, can cause side effects. Not everyone who takes Orkambi will experience these side effects, and their severity can vary from person to person. It’s important to discuss potential side effects with your healthcare provider before starting Orkambi. Here are some common side effects associated with Orkambi:

• Respiratory symptoms: Orkambi can cause an increase in respiratory symptoms such as shortness of breath, chest discomfort, or cough. If you experience any worsening of these symptoms, it’s important to contact your healthcare provider.
• Gastrointestinal issues: Common gastrointestinal side effects of Orkambi include nausea, diarrhea, stomach pain, and indigestion. These symptoms are usually mild to moderate but may require medical attention if severe or persistent.
• Elevated liver enzymes: Orkambi may lead to elevated liver enzymes, which can be detected through liver function tests. Regular monitoring of liver function is recommended during treatment with Orkambi.
• Increased risk of respiratory infections: Some individuals taking Orkambi may be more prone to respiratory infections such as common colds or sinusitis. If you experience any signs of infection, such as fever, cough, or worsening symptoms, it’s important to seek medical attention.
• Fatigue: Fatigue or tiredness can occur as a side effect of Orkambi. If it is persistent or affecting your daily activities, consult your healthcare provider.
• Abnormal liver function: In some cases, Orkambi may cause liver-related adverse events, including liver injury. Signs of liver problems can include yellowing of the skin or eyes (jaundice), dark urine, itching, or abdominal pain. Seek immediate medical attention if you experience these symptoms.

It’s important to note that these are not all the possible side effects of Orkambi. Other, less common side effects may occur. If you experience any unusual or severe side effects while taking Orkambi, it is important to report them to your healthcare provider. They can assess the situation, provide guidance, and determine the appropriate course of action.

Remember, the benefits of Orkambi in treating cystic fibrosis should be weighed against the potential risks and side effects. Your healthcare provider will help you assess the overall benefit-risk profile and monitor your response to the medication.

What should I know about storage and disposal of this medication?

• Storage: Orkambi should be stored at room temperature, typically between 68°F to 77°F (20°C to 25°C). Keep the medication in its original container, away from excessive heat, moisture, and direct light. Do not store Orkambi in the bathroom or near the kitchen sink.
• Childproofing: It is important to keep Orkambi out of the reach of children. Store it in a secure place where children cannot access it. Accidental ingestion of Orkambi by children can be harmful.
• Disposal: When disposing of Orkambi, it is recommended to follow local guidelines or regulations for medication disposal. Do not flush the medication down the toilet or pour it down the drain unless instructed to do so. Consult with your pharmacist or healthcare provider for specific instructions on how to safely dispose of Orkambi.

In case of emergency/overdose

In the case of an emergency or overdose, you should immediately contact your local emergency services or go to the nearest emergency room. It is important to bring the medication packaging or container with you to help healthcare professionals assess the situation accurately.

What other information should I know?

• Compliance: It is important to take Orkambi exactly as prescribed by your healthcare provider. Adherence to the prescribed dosing schedule is crucial for the medication to be effective.
• Follow-up appointments: Regular follow-up appointments with your healthcare provider are important to monitor your response to Orkambi and evaluate its effectiveness. During these appointments, your doctor may conduct tests, such as lung function tests or liver function tests, to assess your progress.
• Interactions with other medications: Inform your healthcare provider about all the medications, supplements, and herbal products you are taking, as Orkambi may interact with other drugs. This includes prescription medications, over-the-counter drugs, and any other substances you are using.
• Allergies: If you have known allergies or hypersensitivity to any of the components of Orkambi, inform your healthcare provider before starting the medication.
• Genetic counseling: If you have questions about genetic testing or the specific genetic mutation related to Orkambi treatment, you may consider speaking with a genetic counselor who can provide specialized guidance and support.

It’s important to consult with your healthcare provider or pharmacist for personalized instructions and any additional information specific to your individual situation. They can address any concerns, provide detailed guidance, and offer the most up-to-date information regarding Orkambi.