Trikafta

Trikafta is used to treat cystic fibrosis (CF) in adults and children 2 years and older who have at least one genetic change (variant) in the cystic fibrosis transmembrane conductance regulator (CFTR) gene that is either responsive to Trikafta or results in the production of protein.

• Talk to your healthcare provider to learn if you have an indicated CF gene variant.
• If a patient’s genotype is not known, an FDA-cleared genetic test should be used to check for at least one CFTR gene variant that may respond to Trikafta, or that allows the body to make CFTR protein.

Trikafta combines three medicines that target the defective CFTR protein in CF. Two correct its misfolding, one improves its function, reducing mucus buildup and improving symptoms. Trikafta consists of 2 different doses (a morning dose and an evening dose taken 12 hours apart). Each dose has different ingredients and must be taken with fat-containing foods.

Trikafta (elexacaftor/tezacaftor/ivacaftor, and ivacaftor) gained FDA approval on October 21, 2019, and since then, indications have been expanded to include more patients. There is no generic, although one is expected in 2026.

Take Trikafta exactly as your healthcare provider tells you to take it.

• Take Trikafta by mouth only.
• Trikafta consists of 2 different doses (a morning dose and an evening dose taken 12 hours apart). Each dose has different ingredients.
• Swallow the Trikafta tablets whole.
• Always take Trikafta oral granules with food that contains fat. Examples of fat-containingfoods include butter, oil, eggs, peanut butter, nuts, meat, and whole-milk dairy products such as whole milk, cheese, and yogurt.

Trikafta oral granules (child aged 2 to <6, <31 lb [14 kg])

Lower-strength: 80/40/60 mg combination (white/blue) + 59.5 mg ivacaftor (white/green)

• The white and blue packets each contain the medicines elexacaftor, tezacaftor, and ivacaftor. Take one morning dose packet in the morning.
• The white and green packets each contain the medicine ivacaftor. Take one evening dose packet in the evening.

Higher-strength: 100/50/75 mg combination (white/orange) + 75 mg ivacaftor (white/pink)

• The white and orange packets each contain the medicines elexacaftor, tezacaftor, and ivacaftor. Take one morning dose packet in the morning.
• The white and pink packets each contain the medicine ivacaftor. Take one evening dose packet in the evening.

To prepare Trikafta oral granules:

• Hold the packet with the cut line on top
• Shake the packet gently to settle the Trikafta oral granules
• Tear or cut the packet open along the cut line
• Carefully pour all the Trikafta oral granules in the packet into 1 teaspoon (5 mL) of soft food or liquid in a small container (like an empty bowl). The food or liquid should be at or below room temperature. Some examples of soft foods or liquids include pureed fruits or vegetables, yogurt, applesauce, water, milk, or juice
• Mix the Trikafta granules with food or liquid
• After mixing, give Trikafta within 1 hour with food that contains fat. Make sure all the medicine is taken.

Trikafta Tablets

Swallow Trikafta tablets whole.

Trikafta tablets (child aged 6 to <12 years, < 66 lb [30kg])

• The light orange tablet is marked with ‘T50’ and each tablet contains the medicines elexacaftor, tezacaftor, and ivacaftor. Take 2 light orange tablets in the morning.
• The light blue tablet is marked with ‘V 75’ and contains the medicine ivacaftor. Take 1 light blue tablet in the evening.

Trikafta tablets (child aged 6 to <12 years and ≥66 lb [30kg], or aged 12+)

• The orange tablet is marked with ‘T100’ and each tablet contains the medicines elexacaftor, tezacaftor, and ivacaftor. Take 2 orange tablets in the morning.
• The light blue tablet is marked with ‘V 150’ and contains the medicine ivacaftor. Take 1 light blue tablet in the evening.

Before taking Trikafta, tell your healthcare provider about all your medical conditions, including if you:

• Have or have had liver problems
• Are allergic to Trikafta, elexacaftor, ivacaftor, tezacaftor, or one of the inactive ingredients in the tablets or granules
• Have kidney problems
• Are pregnant or plan to become pregnant
• Are breastfeeding or plan to breastfeed.

Pregnancy

It is not known if Trikafta will harm your unborn baby. You and your doctor should decide if you should take Trikafta while you are pregnant.

Breastfeeding

It is not known if Trikafta passes into your breast milk. You and your doctor should decide if you should take it while you are breastfeeding.

Warnings and Important Information

Trikafta carries a Boxed Warning for drug-induced liver injury and liver failure.

Liver Injury and Liver Failure

Trikafta can cause serious liver damage and liver failure. Liver failure leading to transplantation and death has been seen in some people with or without a history of liver problems taking Trikafta.

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

• Before you start Trikafta
• Then every month during your first 6 months of taking it
• Then every 3 months during the next 12 months of taking it
• Then, at least every year while you are taking Trikafta.

Your healthcare provider may do blood tests to check the liver more often if you have had high liver enzymes in your blood in the past or are experiencing signs or symptoms of liver injury. Stop taking Trikafta and call your healthcare provider right away if you have any of the following symptoms of liver problems:

• Pain, swelling, or discomfort in the upper right stomach (abdominal) area
• Yellowing of your skin or the white part of your eyes
• Mental changes
• Nausea or vomiting
• Dark, amber-colored urine
• Loss of appetite
• Have fluid in your stomach area (ascites).

Common Side Effects

The most common Trikafta side effects are:

• Headache
• Upper respiratory tract infections (common cold), including a stuffy, runny nose
• Stomach (abdominal) pain
• Diarrhea
• Rash
• Increase in liver enzymes
• An increase in a blood enzyme called creatine phosphokinase
• Flu (influenza)
• Inflamed sinuses
• Increase in blood bilirubin
• Constipation.

Serious side effects and warnings

Trikafta may cause the following other serious side effects:

• Serious Allergic Reactions. Serious allergic reactions can happen to people who are treated with Trikafta. Call your healthcare provider or go to the emergency room right away if you have symptoms of an allergic reaction, which may include: Rash or hives Tightness of the chest or throat, or difficulty breathing Swelling of the face, lips, and/or tongue, difficulty swallowing Light-headedness or dizziness.
• Increased pressure around the brain (intracranial hypertension). Intracranial hypertension has happened in people treated with Trikafta. If you experience an unusual headache, blurred vision, double vision, or vision loss, call your healthcare provider right away.
• Serious mental health problems such as anxiety, depression, suicidal thoughts and behaviors, and trouble sleeping have happened in people treated with Trikafta or medicines containing the same or similar ingredients as Trikafta. If you experience new or worsening mental health problems, call your healthcare provider right away.
• Abnormality of the eye lens (cataract) has happened in some children and adolescents treated with Trikafta. If you are a child or adolescent, your healthcare provider should perform eye examinations before and during treatment to look for cataracts.

Notes:

It is not known if Trikafta is safe and effective in children under 2 years of age.

Tell your healthcare provider if you have any side effect that bothers you or that do not go away. 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 the FDA at 1-800-FDA-1088.

Trikafta combines three medicines (elexacaftor, tezacaftor, and ivacaftor) that target the underlying cause of CF, the defective CFTR protein.

• Two components (elexacaftor and tezacaftor) work as correctors, helping the misfolded protein reach the cell surface.
• The third (ivacaftor) functions as a potentiator, improving the protein's ability to transport chloride ions once positioned correctly.

This triple combination restores CFTR function, reducing mucus buildup in the lungs and improving symptoms for patients with specific CF mutations.

Trikafta belongs to the drug class called CFTR combination treatments.

Dose of Trikafta Granules for CF (2 to < 6 years and <31lb [14kg])

• Morning: One white and blue packet. This packet contains elexacaftor 80 mg/tezacaftor 40 mg/ivacaftor 60 mg oral granules.
• Evening: One white and green packet in the evening. This packet only contains ivacaftor 59.5 mg.

Dose of Trikafta Granules for CF (2 to < 6 years and ≥ 31lb [14kg])

• Morning: One white and orange packet. This packet contains elexacaftor 100 mg/tezacaftor 50 mg/ivacaftor 75 mg oral granules.
• Evening: One white and pink packet in the evening. This packet only contains ivacaftor 75 mg.

Dose of Trikafta Tablets for CF (6 to <12 years and < 66lb [30kg])

• Morning: Two tablets, each containing elexacaftor 50 mg/tezacaftor 25 mg/ivacaftor 37.5 mg
• Evening: One tablet of ivacaftor 75 mg.

Dose of Trikafta Tablets for CF (6 to <12 years and ≥ 66lb [30kg])

• Morning: Two tablets, each containing elexacaftor 100 mg/tezacaftor 50 mg/ivacaftor 75 mg
• Evening: One tablet of ivacaftor 150 mg.

Dose of Trikafta Tablets for CF (12 years+)

• Morning: Two tablets, each containing elexacaftor 100 mg/tezacaftor 50 mg/ivacaftor 75 mg
• Evening: One tablet of ivacaftor 150 mg.

If you miss a dose of Trikafta and it is:

• 6 hours or less from the time you usually take the morning dose or the evening dose, take the missed dose with food that contains fat as soon as you can. Then take your next dose at your usual time
• More than 6 hours from the time you usually take the morning dose, take the missed dose with food that contains fat as soon as you can. Do not take the evening dose
• More than 6 hours from the time you usually take the evening dose, do not take the missed dose. Take your next morning dose at the usual time with food that contains fat.

Do not take more than your usual dose of Trikafta to make up for a missed dose.

Do not take the morning and evening doses at the same time.

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

Avoid driving or hazardous activities until you know how Trikafta will affect you. Your reactions could be impaired.

Grapefruit may interact with Trikafta and lead to unwanted side effects. Avoid the use of grapefruit products.

Talk to your healthcare provider or pharmacist before taking any new medication or supplement.

Tell your healthcare provider about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements. Trikafta may affect the way other medicines work, and other medicines may affect how Trikafta works. The dose of Trikafta may need to be adjusted when taken with certain medicines. Ask your healthcare provider or pharmacist for a list of these medicines if you are not sure.

Especially tell your healthcare provider if you take:

• Antibiotics such as rifampin (Rifamate, Rifater) or rifabutin (Mycobutin)
• Seizure medicines such as phenobarbital, carbamazepine (Tegretol, Carbatrol, Equetro), or phenytoin (Dilantin, Phenytek)
• St. John’s wort
• Antifungal medicines, including ketoconazole, itraconazole (such as Sporonox), posaconazole (such as Noxafil), voriconazole (such as Vfend), or fluconazole (such as Diflucan)
• Antibiotics, including telithromycin, clarithromycin (such as BIAXIN), or erythromycin (such as ERYTAB).

Know the medicines you take. Keep a list of them to show your healthcare provider and pharmacist when you get a new medicine. This list is not complete. Not all possible drug interactions are listed here. See the Trikafta Prescribing Information for a complete list.

Store Trikafta tablets and granules at room temperature 68°F - 77°F (20°C - 25°C). Keep each tablet in its daily blister strip and the granules in their packet until you are ready to take your dose.

Keep out of reach of children.

Trikaftor Tablets

Active ingredients: elexacaftor, tezacaftor, and ivacaftor.

Inactive ingredients: croscarmellose sodium, hypromellose, hypromellose acetate succinate, magnesiumstearate, microcrystalline cellulose, sodium lauryl sulfate. The tablet film coat contains hydroxypropyl cellulose,hypromellose, iron oxide red, iron oxide yellow, talc, and titanium dioxide.

Ivacaftor Tablets

Active ingredients: ivacaftor.

Inactive ingredients: colloidal silicon dioxide, croscarmellose sodium, hypromellose acetate succinate, lactosemonohydrate, magnesium stearate, microcrystalline cellulose, and sodium lauryl sulfate.

The tablet film coat contains carnauba wax, FD&C Blue #2, PEG 3350, polyvinyl alcohol, talc, and titaniumoxide.

The printing ink contains ammonium hydroxide, iron oxide black, propylene glycol, and shellac.

Trikafta oral granules

Active ingredients: elexacaftor, tezacaftor, and ivacaftor.

Inactive ingredients: colloidal silicon dioxide, croscarmellose sodium, hypromellose, hypromellose acetatesuccinate, lactose monohydrate, magnesium stearate, mannitol, sodium lauryl sulfate, and sucralose.

Ivacaftor oral granules ingredients

Active ingredients: ivacaftor.

Inactive ingredients: colloidal silicon dioxide, croscarmellose sodium, hypromellose acetate succinate, lactosemonohydrate, magnesium stearate, mannitol, sodium lauryl sulfate, and sucralose.

Available formulations

Tablets:

• 50/25/37.5 mg (elexacaftor/tezacaftor/ivacaftor) + ivacaftor 75 mg
• 100/50/75 mg (elexacaftor/tezacaftor/ivacaftor) + ivacaftor 150 mg

Oral granules (sachets):

• Higher-strength: 100/50/75 mg combination (white/orange) + 75 mg ivacaftor (white/pink)
• Lower-strength: 80/40/60 mg combination (white/blue) + 59.5 mg ivacaftor (white/green).

Trikafta is manufactured for Vertex Pharmaceuticals Incorporated, 50 Northern Avenue, Boston, MA 02210.