Ivacaftor Accord: Uses, Dosage & Side Effects
A CFTR potentiator for the treatment of cystic fibrosis in patients with specific gating mutations in the CFTR gene
Ivacaftor Accord is a generic medicine containing ivacaftor, a CFTR (cystic fibrosis transmembrane conductance regulator) potentiator used to treat cystic fibrosis in patients aged 6 years and older who weigh at least 25 kg. It works by enhancing the function of the defective CFTR protein channel at the cell surface, improving chloride and fluid transport across epithelial membranes in the lungs, pancreas, and other organs. Ivacaftor Accord is bioequivalent to the reference product Kalydeco and is indicated for patients who carry specific gating mutations in the CFTR gene, including the G551D mutation. It is taken orally as a 150 mg tablet every 12 hours with fat-containing food and requires a prescription.
Quick Facts: Ivacaftor Accord
Key Takeaways
- Ivacaftor Accord is a generic version of ivacaftor (reference product: Kalydeco), a CFTR potentiator that increases chloride ion transport through defective CFTR protein channels in patients with cystic fibrosis carrying specific gating mutations.
- It must always be taken with fat-containing food (such as eggs, butter, cheese, or whole-milk products) to ensure adequate absorption; taking it without fat dramatically reduces blood levels and therapeutic effectiveness.
- Liver function tests (ALT and AST) are required before starting treatment, every 3 months during the first year, and at least annually thereafter, because hepatotoxicity including serious liver injury has been reported.
- Strong CYP3A inhibitors (such as ketoconazole, itraconazole, and clarithromycin) and CYP3A inducers (such as rifampicin and St. John’s Wort) significantly alter ivacaftor blood levels and require dose adjustments or avoidance.
- Cataracts have been reported in pediatric patients treated with ivacaftor; baseline and follow-up ophthalmological examinations are recommended for children and adolescents starting treatment.
What Is Ivacaftor Accord and What Is It Used For?
Ivacaftor Accord contains the active substance ivacaftor, which belongs to a class of medicines known as CFTR potentiators. The CFTR (cystic fibrosis transmembrane conductance regulator) protein is a chloride channel found on the surface of epithelial cells throughout the body, including the lungs, pancreas, liver, intestines, sweat glands, and reproductive tract. In cystic fibrosis, mutations in the CFTR gene lead to a defective or absent CFTR protein, resulting in impaired chloride and water transport across cell membranes. This creates the thick, sticky mucus that obstructs airways, promotes chronic infections, and damages multiple organ systems.
Ivacaftor works by a fundamentally different mechanism from traditional symptomatic CF treatments. Rather than treating the downstream consequences of CFTR dysfunction (such as airway infections or pancreatic insufficiency), ivacaftor directly targets the underlying molecular defect. Specifically, it increases the open probability (gating) of the CFTR channel at the cell surface, allowing more chloride ions to flow through the channel. This restores hydration of the airway surface liquid, improves mucociliary clearance, and helps normalize the function of affected organs. In clinical terms, this translates to improved lung function (measured by FEV1), reduced pulmonary exacerbations, improved nutritional status, and better quality of life.
Ivacaftor Accord is a generic medicine that has been approved by regulatory authorities after demonstrating bioequivalence to the reference product Kalydeco. Bioequivalence means that Ivacaftor Accord delivers the same amount of active substance (ivacaftor) to the body at the same rate as the reference product, ensuring equivalent therapeutic effect and safety profile. This allows patients to access the same clinically proven treatment, potentially at a lower cost.
Ivacaftor Accord is indicated for the treatment of cystic fibrosis in patients aged 6 years and older who weigh at least 25 kg and who have one of the following gating (Class III) mutations in the CFTR gene:
- G551D – the most common gating mutation, present in approximately 4–5% of all CF patients worldwide
- G1244E – a rare gating mutation
- G1349D – a rare gating mutation
- G178R – a rare gating mutation
- G551S – a rare gating mutation
- S1251N – a gating mutation with clinical evidence of ivacaftor benefit
- S1255P – a rare gating mutation
- S549N – a rare gating mutation
- S549R – a rare gating mutation
- R117H – a residual function mutation (Class IV) that also responds to ivacaftor potentiation
It is important to understand that ivacaftor is effective only in patients whose CFTR protein reaches the cell surface but does not open (gate) properly. It is not effective in patients who are homozygous for the F508del mutation (the most common CF mutation overall) when used alone, because in F508del-homozygous patients, the CFTR protein is misfolded and degraded before it reaches the cell surface. For F508del patients, combination therapies that include both a CFTR corrector (to help the protein reach the surface) and a potentiator are used instead, such as lumacaftor/ivacaftor (Orkambi), tezacaftor/ivacaftor (Symdeko/Symkevi), or elexacaftor/tezacaftor/ivacaftor (Trikafta/Kaftrio).
Cystic fibrosis is caused by mutations in the CFTR gene, of which more than 2,000 have been identified. These mutations are classified into six classes based on their effect on the CFTR protein. Gating mutations (Class III), such as G551D, produce a CFTR protein that reaches the cell surface but fails to open properly. Ivacaftor specifically targets these gating defects by increasing the channel’s open probability. Before starting treatment, genetic testing must confirm the presence of an eligible mutation.
What Should You Know Before Taking Ivacaftor Accord?
Contraindications
Ivacaftor Accord is contraindicated in patients with known hypersensitivity to ivacaftor or to any of the excipients contained in the film-coated tablet formulation. While absolute contraindications are relatively limited for this medication, there are several clinical scenarios where the benefit-risk assessment must be carefully considered before initiating therapy.
A confirmed diagnosis of cystic fibrosis with an eligible CFTR gating mutation must be established by an accredited laboratory before treatment can begin. Treatment should only be initiated by a physician experienced in the management of cystic fibrosis. Genetic testing using a validated mutation detection method is required to identify the specific CFTR mutation(s) present in each patient.
Warnings and Precautions
Elevated transaminases (ALT and AST) have been reported in patients treated with ivacaftor, including cases of serious hepatic injury. Liver function tests (LFTs) must be performed prior to initiating treatment, every 3 months during the first year of treatment, and annually thereafter. In patients with a history of transaminase elevations, more frequent monitoring should be considered. If ALT or AST exceeds 5 times the upper limit of normal (ULN), or ALT or AST exceeds 3 times ULN with bilirubin exceeding 2 times ULN, treatment should be interrupted and LFTs closely monitored until the abnormalities resolve.
Before and during treatment with Ivacaftor Accord, your doctor should be informed about the following:
- Liver disease: Patients with moderate hepatic impairment (Child-Pugh Class B) require a reduced dose of 150 mg once daily. No studies have been conducted in patients with severe hepatic impairment (Child-Pugh Class C), so ivacaftor should be used in these patients only if the benefits outweigh the risks, and with great caution at an initial dose of 150 mg every other day or less frequently.
- Kidney disease: No dose adjustment is necessary for patients with mild to moderate renal impairment. Caution is recommended in patients with severe renal impairment (creatinine clearance ≤30 mL/min) or end-stage renal disease, as ivacaftor has not been studied in these populations.
- Cataracts in children: Cases of non-congenital lens opacities (cataracts) without impact on vision have been reported in pediatric patients treated with ivacaftor. Although other risk factors were present in some of these cases (such as corticosteroid use and radiation exposure), a possible risk attributable to ivacaftor cannot be excluded. Baseline and follow-up ophthalmological examinations are recommended in pediatric patients starting ivacaftor treatment.
- Organ transplant recipients: Ivacaftor has not been studied in patients with cystic fibrosis who have received organ transplants. Use with caution due to potential drug interactions with immunosuppressive agents metabolized by CYP3A, such as ciclosporin and tacrolimus.
Pregnancy and Breastfeeding
There are limited data on the use of ivacaftor in pregnant women. Animal studies have not shown direct or indirect harmful effects with respect to reproductive toxicity at clinically relevant exposures. However, as a precautionary measure, it is preferable to avoid the use of Ivacaftor Accord during pregnancy unless the potential benefit to the mother justifies the potential risk to the fetus. The decision to continue or discontinue treatment during pregnancy should be made in close consultation with a CF specialist who can weigh the risks of untreated CF (including declining lung function) against the potential risks of the medication.
It is not known whether ivacaftor and/or its metabolites are excreted in human breast milk. Available pharmacokinetic data in animals have shown excretion of ivacaftor in the milk of lactating rats. A risk to breastfed newborns and infants cannot be excluded. A decision must be made whether to discontinue breastfeeding or to discontinue ivacaftor therapy, taking into account the benefit of breastfeeding for the child and the benefit of therapy for the mother.
Ivacaftor has no known effect on fertility. Animal studies have not demonstrated adverse effects on fertility at clinically relevant doses. However, the effects of ivacaftor on fertility in humans have not been specifically studied.
How Does Ivacaftor Accord Interact with Other Drugs?
Ivacaftor is extensively metabolized by the cytochrome P450 enzyme CYP3A4 and, to a lesser extent, CYP3A5. It is also a substrate of the P-glycoprotein (P-gp) transporter. Drug interactions involving CYP3A are clinically significant and can profoundly alter the exposure to ivacaftor, either increasing its concentration (and risk of adverse effects) or decreasing it (and reducing therapeutic efficacy).
Understanding these interactions is critically important because cystic fibrosis patients frequently take multiple medications simultaneously, including antibiotics, antifungals, pancreatic enzyme replacement therapy, mucolytics, and bronchodilators. The CF care team must carefully review all concomitant medications before starting ivacaftor therapy and at each clinical visit.
Major Interactions
| Interacting Drug | Type | Effect | Recommendation |
|---|---|---|---|
| Ketoconazole | Strong CYP3A inhibitor | Increases ivacaftor AUC by approximately 8.5-fold | Reduce dose to 150 mg twice per week |
| Itraconazole | Strong CYP3A inhibitor | Expected to significantly increase ivacaftor levels | Reduce dose to 150 mg twice per week |
| Posaconazole | Strong CYP3A inhibitor | Expected to significantly increase ivacaftor levels | Reduce dose to 150 mg twice per week |
| Voriconazole | Strong CYP3A inhibitor | Expected to significantly increase ivacaftor levels | Reduce dose to 150 mg twice per week |
| Clarithromycin | Strong CYP3A inhibitor | Expected to significantly increase ivacaftor levels | Reduce dose to 150 mg twice per week |
| Telithromycin | Strong CYP3A inhibitor | Expected to significantly increase ivacaftor levels | Reduce dose to 150 mg twice per week |
| Rifampicin | Strong CYP3A inducer | Decreases ivacaftor AUC by approximately 89% | Co-administration not recommended |
| Rifabutin | Strong CYP3A inducer | Expected to substantially decrease ivacaftor levels | Co-administration not recommended |
| Phenobarbital | Strong CYP3A inducer | Expected to substantially decrease ivacaftor levels | Co-administration not recommended |
| Carbamazepine | Strong CYP3A inducer | Expected to substantially decrease ivacaftor levels | Co-administration not recommended |
| Phenytoin | Strong CYP3A inducer | Expected to substantially decrease ivacaftor levels | Co-administration not recommended |
| St. John’s Wort | Strong CYP3A inducer (herbal) | Expected to substantially decrease ivacaftor levels | Co-administration not recommended |
Moderate Interactions
Moderate CYP3A inhibitors, such as fluconazole and erythromycin, are also expected to increase ivacaftor exposure, though to a lesser degree than strong inhibitors. When co-administered with moderate CYP3A inhibitors, the dose of Ivacaftor Accord should be reduced to 150 mg once daily. Fluconazole is commonly used in CF patients for the treatment of aspergillus infections, making this interaction particularly relevant in clinical practice.
Grapefruit juice and Seville oranges contain substances that inhibit CYP3A and may increase ivacaftor plasma concentrations. Patients should be advised to avoid food or drink containing grapefruit or Seville oranges during treatment with Ivacaftor Accord.
Minor Interactions
Ivacaftor itself is a weak inhibitor of CYP3A and CYP2C9. Co-administration of ivacaftor with CYP3A substrates with a narrow therapeutic index (such as ciclosporin, tacrolimus, sirolimus, and everolimus) may increase the systemic exposure of these substrates. Monitoring of appropriate drug levels is recommended when these medicines are used concomitantly with ivacaftor.
Ivacaftor may also inhibit P-glycoprotein (P-gp). Co-administration with digoxin, a sensitive P-gp substrate, increased digoxin AUC by 1.3-fold. Caution and appropriate monitoring are advised when using ivacaftor with P-gp substrates, particularly digoxin. Co-administration with the CYP2D6 substrate desipramine increased desipramine AUC by approximately 1.3-fold.
Many cystic fibrosis patients require antifungal treatment for aspergillus colonization or allergic bronchopulmonary aspergillosis (ABPA). Since azole antifungals (ketoconazole, itraconazole, voriconazole, posaconazole) are strong CYP3A inhibitors, dose adjustment of ivacaftor is essential. Fluconazole is a moderate CYP3A inhibitor that also requires dose reduction. Always inform your CF care team about any antifungal treatment you are receiving.
What Is the Correct Dosage of Ivacaftor Accord?
Ivacaftor Accord should only be prescribed by physicians who are experienced in the treatment of cystic fibrosis. An eligible CFTR mutation must be confirmed by genotyping before starting treatment. The dosage depends on the patient’s age, weight, concomitant medications, and hepatic function.
Adults
Standard Adult Dose
One 150 mg film-coated tablet taken orally every 12 hours (approximately 300 mg total daily dose) with fat-containing food. The interval between doses should be approximately 12 hours. Each dose must be taken with a meal or snack that contains fat, as this significantly improves absorption. Examples of fat-containing foods include eggs, avocado, nuts, butter, peanut butter, cheese pizza, and whole-milk dairy products.
Children
Children Aged 6 Years and Older (Weighing ≥25 kg)
The dose is the same as for adults: one 150 mg tablet every 12 hours with fat-containing food. The Ivacaftor Accord 150 mg film-coated tablet formulation is appropriate for children who weigh at least 25 kg and can swallow tablets.
For children aged 6 months to less than 6 years, or children who weigh less than 25 kg, other formulations (such as granules for oral administration) are available under different product names. The 150 mg tablet formulation should not be used in children who cannot swallow tablets whole.
Elderly
No dose adjustment is necessary based on age alone. Clinical experience with ivacaftor in patients aged 65 years and older is limited because cystic fibrosis primarily affects younger populations. However, as renal and hepatic function may decline with age, elderly patients should be monitored more closely.
Dose Adjustments
| Clinical Situation | Adjusted Dose | Rationale |
|---|---|---|
| With strong CYP3A inhibitors | 150 mg twice per week | ~8.5-fold increase in ivacaftor exposure |
| With moderate CYP3A inhibitors | 150 mg once daily | ~3-fold increase in ivacaftor exposure |
| Moderate hepatic impairment (Child-Pugh B) | 150 mg once daily | ~2-fold increase in ivacaftor exposure |
| Severe hepatic impairment (Child-Pugh C) | 150 mg once daily or less frequently | Not studied; use only if benefits outweigh risks |
| Mild hepatic impairment (Child-Pugh A) | No adjustment needed | No clinically significant change in exposure |
| Mild to moderate renal impairment | No adjustment needed | Minimal renal excretion of ivacaftor |
Missed Dose
If a dose of Ivacaftor Accord is missed within 6 hours of the time it is usually taken, the patient should take the missed dose with fat-containing food as soon as possible. If more than 6 hours have passed since the usual dosing time, the missed dose should be skipped, and the next dose should be taken at the regularly scheduled time. Patients should never take a double dose to make up for a missed one. Consistent adherence to the dosing schedule is important for maintaining stable blood levels of ivacaftor and achieving optimal therapeutic benefit.
Overdose
No specific antidote exists for ivacaftor overdose. There is limited experience with overdose of ivacaftor. In the event of overdose, treatment should consist of general supportive measures, including monitoring of vital signs and observation of the patient’s clinical status. The highest single dose used in healthy volunteers was 800 mg without dose-limiting toxicities. In clinical studies, the highest dose given for a sustained period was 450 mg twice daily for 28 days in healthy volunteers, with adverse events consistent with those observed at the recommended dose.
What Are the Side Effects of Ivacaftor Accord?
Like all medicines, Ivacaftor Accord can cause side effects, although not everybody gets them. The safety profile of ivacaftor has been well characterized through extensive clinical trials and post-marketing surveillance. Most side effects are mild to moderate in severity and tend to diminish with continued treatment. However, certain adverse reactions—particularly hepatotoxicity—require vigilant monitoring throughout the duration of therapy.
The frequency of side effects is classified using the following convention: very common (affects more than 1 in 10 people), common (affects 1 in 10 to 1 in 100 people), uncommon (affects 1 in 100 to 1 in 1,000 people), and rare (affects fewer than 1 in 1,000 people).
Very Common
Affects more than 1 in 10 people
- Headache
- Upper respiratory tract infections (common cold, nasal congestion, sore throat)
- Abdominal (stomach) pain
- Diarrhea
- Rash
- Oropharyngeal pain (sore throat, throat irritation)
- Nasal congestion (blocked or stuffy nose)
- Bacteria in sputum
Common
Affects 1 in 10 to 1 in 100 people
- Dizziness
- Nausea
- Elevated transaminases (ALT and/or AST increase)
- Ear pain
- Tinnitus (ringing in the ears)
- Ear congestion or discomfort
- Rhinitis (runny nose, sneezing)
- Breast mass or tenderness (including gynecomastia in males)
- Acne
Uncommon
Affects 1 in 100 to 1 in 1,000 people
- Breast inflammation (mastitis)
- Nipple disorder or pain
- Gynaecomastia (breast enlargement in males)
Rare / Post-Marketing Reports
Affects fewer than 1 in 1,000 people
- Severe hepatotoxicity (serious liver injury)
- Non-congenital lens opacities (cataracts) in pediatric patients
- Hypersensitivity reactions
The most clinically significant adverse reaction associated with ivacaftor therapy is hepatotoxicity. Elevations of transaminases (ALT and/or AST) are common, occurring in approximately 5–10% of patients in clinical trials. In most cases, these elevations are asymptomatic and resolve either spontaneously or after dose interruption. However, cases of serious liver injury with significantly elevated transaminases (greater than 8 times ULN) and elevated bilirubin have been reported. This is why regular liver function monitoring is mandatory throughout treatment.
In pediatric patients, non-congenital lens opacities (cataracts) have been reported. While a causal relationship has not been definitively established (as other risk factors such as corticosteroid use were often present), baseline and follow-up ophthalmological examinations are recommended for all pediatric patients initiating ivacaftor therapy.
Some patients have reported breast-related adverse events, including breast mass, gynecomastia (breast tissue enlargement in males), and breast tenderness. These events have been observed in both adult and adolescent patients and are generally reversible after discontinuation of treatment.
Contact your doctor immediately if you experience: yellowing of the skin or eyes (jaundice), dark urine, persistent nausea or vomiting, right-sided abdominal pain, unexplained fatigue, or loss of appetite. These may be signs of serious liver injury. Also report any sudden changes in vision, particularly in children and adolescents.
How Should Ivacaftor Accord Be Stored?
Proper storage of Ivacaftor Accord is essential to maintain the quality, safety, and efficacy of the medicine throughout its shelf life. Incorrect storage conditions can lead to degradation of the active substance, potentially reducing the therapeutic effect or producing harmful breakdown products.
- Temperature: Store below 30°C (86°F). Do not refrigerate or freeze. Ivacaftor Accord tablets should be kept at room temperature, away from excessive heat.
- Moisture protection: Store in the original packaging to protect from moisture. The blister packaging is designed to protect the tablets from humidity and should not be removed until immediately before administration.
- Light protection: While no special light protection requirements are specified, it is good practice to store the medicine in its original carton when not in use.
- Children: Keep this medicine out of the sight and reach of children. Consider using a locked medicine cabinet or high shelf.
- Expiry date: Do not use Ivacaftor Accord after the expiry date which is stated on the carton and blister after “EXP”. The expiry date refers to the last day of that month.
- Disposal: Do not throw away any medicines via wastewater or household waste. Ask your pharmacist how to throw away medicines you no longer use. These measures will help protect the environment.
Patients who travel with their medication should ensure that Ivacaftor Accord is kept within the recommended temperature range during transit. In hot climates, use insulated bags but avoid direct contact with ice or cold packs. If tablets have been exposed to temperatures significantly above 30°C for an extended period, consult your pharmacist about whether they are still suitable for use.
What Does Ivacaftor Accord Contain?
Understanding the full composition of Ivacaftor Accord is important for identifying potential allergens or excipients that may be relevant for patients with specific intolerances or allergies.
Active Substance
Each film-coated tablet contains 150 mg of ivacaftor. Ivacaftor is the international non-proprietary name (INN) for the active pharmaceutical ingredient. Its chemical name is N-(2,4-di-tert-butyl-5-hydroxyphenyl)-1,4-dihydro-4-oxoquinoline-3-carboxamide. Ivacaftor is a white to off-white crystalline powder that is practically insoluble in water.
Excipients
The excipients in the tablet core and film coating include:
- Tablet core: Cellulose microcrystalline, lactose monohydrate, hypromellose acetate succinate, croscarmellose sodium, sodium laurilsulfate, colloidal anhydrous silica, and magnesium stearate.
- Film coating: Polyvinyl alcohol, titanium dioxide (E171), macrogol (polyethylene glycol), talc, and indigo carmine aluminium lake (E132).
Ivacaftor Accord contains lactose monohydrate. Patients with rare hereditary problems of galactose intolerance, total lactase deficiency, or glucose-galactose malabsorption should not take this medicine. The amount of lactose per tablet is relatively small, but patients with severe lactose intolerance should discuss this with their doctor or pharmacist.
The 150 mg tablets are light blue, capsule-shaped, film-coated tablets with dimensions of approximately 16.5 mm × 8.4 mm. They are available in blister packs containing 56 film-coated tablets (sufficient for a 28-day supply at the standard dose of one tablet twice daily).
Frequently Asked Questions About Ivacaftor Accord
Ivacaftor Accord is a generic version of ivacaftor, while Kalydeco is the original (reference) product. Both contain the same active substance – ivacaftor – in the same dose (150 mg) and work in exactly the same way as CFTR potentiators. Ivacaftor Accord has been approved after demonstrating bioequivalence to Kalydeco, meaning it delivers the same amount of active substance to the body at the same rate. The excipients (inactive ingredients) may differ slightly between the two products, but the therapeutic effect and safety profile are equivalent.
Ivacaftor is a highly lipophilic (fat-soluble) compound that is poorly absorbed from the gastrointestinal tract without fat. Clinical pharmacology studies have shown that taking ivacaftor with fat-containing food increases its absorption (bioavailability) by approximately 2 to 4 times compared with fasting conditions. Without adequate fat in the meal, blood levels of ivacaftor may be too low to achieve the desired therapeutic effect on the CFTR channel. Good examples of fat-containing foods include eggs, butter, cheese, avocado, nuts, whole-milk yoghurt, and olive oil-based salad dressings.
No, Ivacaftor Accord does not cure cystic fibrosis. It is a disease-modifying treatment that addresses the underlying molecular defect by improving the function of the CFTR protein channel. While it can significantly improve lung function, reduce pulmonary exacerbations, and enhance quality of life, it does not correct the genetic mutation itself. Patients must continue taking ivacaftor indefinitely to maintain its therapeutic benefits, and they should continue their other CF treatments (such as airway clearance therapies, inhaled antibiotics, and pancreatic enzyme replacement) as directed by their CF care team.
Ivacaftor begins working rapidly. In clinical trials, improvements in sweat chloride (a biomarker of CFTR function) were observed within 2 weeks of starting treatment. Improvements in lung function (FEV1) were seen as early as 2 weeks and were sustained throughout treatment. Many patients also report improvements in respiratory symptoms, energy levels, and appetite within the first few weeks. However, the full therapeutic benefit may take longer to manifest, and the degree of improvement can vary between individuals depending on their specific mutation, disease severity, and other factors.
Rash is one of the more common side effects of ivacaftor and is usually mild and self-limiting. If you develop a mild rash, inform your doctor at your next scheduled visit. However, if the rash is severe, widespread, associated with itching, blistering, peeling of the skin, or accompanied by fever, swelling, or difficulty breathing, seek medical attention promptly as this may indicate a more serious allergic reaction. Do not stop taking your medication without consulting your doctor, as abrupt discontinuation can affect your CF management.
No, you should avoid grapefruit juice and grapefruit products while taking Ivacaftor Accord. Grapefruit contains compounds called furanocoumarins that inhibit the CYP3A enzyme responsible for metabolizing ivacaftor. Consuming grapefruit can increase ivacaftor blood levels, potentially leading to an increased risk of side effects. Seville oranges (often used in marmalade) also contain these inhibitory compounds and should similarly be avoided. Regular oranges, lemons, and limes are safe to consume.
References
- European Medicines Agency (EMA). Ivacaftor Accord – Summary of Product Characteristics. Available at: www.ema.europa.eu.
- Ramsey BW, Davies J, McElvaney NG, et al. A CFTR potentiator in patients with cystic fibrosis and the G551D mutation. N Engl J Med. 2011;365(18):1663-1672. doi:10.1056/NEJMoa1105185.
- Davies JC, Wainwright CE, Canny GJ, et al. Efficacy and safety of ivacaftor in patients aged 6 to 11 years with cystic fibrosis with a G551D mutation. Am J Respir Crit Care Med. 2013;187(11):1219-1225.
- De Boeck K, Munck A, Walker S, et al. Efficacy and safety of ivacaftor in patients with cystic fibrosis and a non-G551D gating mutation. J Cyst Fibros. 2014;13(6):674-680.
- U.S. Food and Drug Administration (FDA). Kalydeco (ivacaftor) Prescribing Information. Available at: www.fda.gov.
- Cystic Fibrosis Foundation. CFTR Modulator Therapies Fact Sheet. Available at: www.cff.org.
- European Cystic Fibrosis Society (ECFS). Standards of Care for Cystic Fibrosis. J Cyst Fibros. 2023.
- World Health Organization (WHO). WHO Model List of Essential Medicines, 23rd list, 2023. Geneva: WHO.
- Accurso FJ, Rowe SM, Clancy JP, et al. Effect of VX-770 in persons with cystic fibrosis and the G551D-CFTR mutation. N Engl J Med. 2010;363(21):1991-2003.
- McKone EF, Borowitz D, Drevinek P, et al. Long-term safety and efficacy of ivacaftor in patients with cystic fibrosis who have the Gly551Asp-CFTR mutation: a phase 3, open-label extension study (PERSIST). Lancet Respir Med. 2014;2(11):902-910.
Editorial Team
This article was written and reviewed by the iMedic Medical Editorial Team, a multidisciplinary group of licensed healthcare professionals with expertise in pulmonology, clinical pharmacology, and cystic fibrosis management.
Medical Review Process:
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