Kalydeco (ivacaftor)

Kalydeco (ivacaftor) is a prescription medicine that treats the underlying cause of cystic fibrosis (CF) in patients who have certain mutations in the CFTR gene. CF is a rare, inherited disease that affects the lungs, digestive system and other organs by producing thick, sticky mucus that clogs the airways and prevents normal function. Kalydeco works by increasing the activity of the CFTR protein, which helps to balance the flow of salt and water across the cell membrane. This helps to thin the mucus and improve lung function, digestion and nutrition. Kalydeco is not a cure for CF, but it can significantly improve the quality of life and reduce the risk of complications for eligible patients.

Who can take Kalydeco?

Kalydeco is approved for the treatment of CF in patients aged 4 months and older who have at least one of the following mutations in the CFTR gene:

• R117H
• G551D
• G1244E
• G1349D
• G178R
• G551S
• S1251N
• S1255P
• S549N
• S549R

Kalydeco can also be used in combination with another medicine containing tezacaftor and ivacaftor to treat patients aged 6 years and older who have inherited two copies of the F508del mutation in the CFTR gene or one copy of the F508del mutation plus one of the following mutations:

• P67L
• R117C
• L206W
• R352Q
• A455E
• D579G
• 711+3A→G
• S945L
• S977F
• R1070W
• D1152H
• 2789+5G→A
• 3272 26A→G
• 3849+10kbC→T

Kalydeco can also be used in combination with another medicine containing ivacaftor, tezacaftor and elexacaftor to treat patients aged 6 years and older who have at least one copy of the F508del mutation in the CFTR gene. To find out if you or your loved one is eligible for Kalydeco, you need to know your CF gene mutations. You can ask your doctor to perform a genetic test or use an online tool to check your mutations.

How to take Kalydeco?

Kalydeco is available as tablets or granules in a sachet. The dose and frequency depend on whether Kalydeco is used alone or together with other medicines. The dose may also need to be adjusted if you have liver or kidney problems or if you are taking certain other medicines that may interact with Kalydeco. You should always follow your doctor’s instructions on how to take Kalydeco. Kalydeco tablets should be swallowed whole with fat-containing food, such as eggs, cheese, butter, nuts or yogurt. Kalydeco granules should be mixed with soft food or liquid and taken by mouth within one hour of preparation. Do not crush or chew the granules.

What are the benefits of Kalydeco?

Kalydeco has been shown to improve lung function, weight gain, sweat chloride levels and quality of life in patients with CF who have eligible mutations. Kalydeco can also reduce the frequency of pulmonary exacerbations (worsening of lung symptoms) and hospitalizations due to CF. The benefits of Kalydeco may vary depending on your age, mutation type, disease severity and other factors. Your doctor will monitor your response to Kalydeco and adjust your treatment plan accordingly.

What are the side effects of Kalydeco?

Kalydeco is generally well tolerated, but like all medicines, it can cause side effects. The most common side effects of Kalydeco are:

• Headache
• Upper respiratory tract infection (common cold)
• Abdominal pain
• Diarrhea
• Rash
• Nausea
• Dizziness

Some side effects may be serious and require medical attention. These include:

• Allergic reactions, such as swelling of the face, lips, tongue or throat, difficulty breathing, hives or itching
• Liver problems, such as increased liver enzymes, yellowing of the skin or eyes, dark urine or abdominal pain
• Cataracts (clouding of the lens in the eye)
• Low blood sugar (hypoglycemia)
• High blood pressure (hypertension)

If you experience any of these side effects, or any other unusual or bothersome symptoms, you should contact your doctor immediately.

How much does Kalydeco cost?

Kalydeco is a very expensive medicine, costing around $300,000 per year in the US. The price may vary depending on your insurance coverage, co-payments, discounts and other factors. You may be eligible for financial assistance programs to help you afford Kalydeco. You can contact your doctor, pharmacist or the manufacturer of Kalydeco to find out more about these programs.

Kalydeco price comparison table

Country	Price per tablet (USD)	Reference
USA	$834.72	GoodRx
Canada	$556.67	PharmacyChecker
Australia	$37.80	PBS
UK	$22.86	BNF
India	$18.33	Medindia

Top 5 global brands of Kalydeco (ivacaftor)

• Kalydeco – manufactured by Vertex Pharmaceuticals, approved in the US, Canada, Australia, New Zealand, EU and other countries.
• Lumacaftor – manufactured by Vertex Pharmaceuticals, approved in the US, Canada, Australia, New Zealand, EU and other countries. It contains ivacaftor and lumacaftor, another CFTR modulator.
• Symkevi – manufactured by Vertex Pharmaceuticals, approved in the EU and other countries. It contains ivacaftor and tezacaftor, another CFTR modulator.
• Trikafta – manufactured by Vertex Pharmaceuticals, approved in the US and Canada. It contains ivacaftor, tezacaftor and elexacaftor, another CFTR modulator.
• Kaftrio – manufactured by Vertex Pharmaceuticals, approved in the EU and other countries. It contains ivacaftor, tezacaftor and elexacaftor.