Product image of Kalydeco (Ivacaftor) supplied by GNH India

Kalydeco

Active Ingredient:
Ivacaftor
Origin:
EU

Kalydeco (Ivacaftor)

Ivacaftor, the active ingredient in Kalydeco, is a CFTR potentiator presented as a 150 mg tablet for oral use. It is indicated for the treatment of cystic fibrosis in patients who carry at least one CFTR gating mutation, such as G551D, and is approved for individuals aged six years and older.

GNH India supplies Kalydeco as a licensed, GDP‑compliant international pharmaceutical supplier serving hospitals, pharmacies and clinics worldwide. The company adheres to stringent quality standards and provides reliable logistics for global distribution.

Manufacturer / TM Owner

Vertex Pharmaceuticals (Ireland) Limited

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Indications & Clinical Uses

  • Kalydeco (ivacaftor) is used to treat cystic fibrosis by targeting the CFTR protein.
  • Cystic fibrosis with G551D mutation: Improves lung function and reduces pulmonary exacerbations.
  • Cystic fibrosis with other CFTR gating mutations: Enhances chloride transport and respiratory outcomes.
  • Cystic fibrosis in patients ≥6 years with eligible mutations: Improves nutritional status and overall disease progression.

How It Works

  • Ivacaftor binds to the nucleotide‑binding domain of the CFTR protein and increases the probability that the channel remains open. By potentiating the gating activity of mutant CFTR proteins, it enhances chloride ion transport across epithelial cell membranes, thereby improving hydration of airway surface liquid and reducing mucus viscosity in cystic fibrosis patients. This action restores more normal ion balance in the lungs and other affected organs.

Side Effects

Ivacaftor may be associated with several adverse reactions, ranging from mild to serious.

Common Side Effects

  • Headache: mild to moderate intensity.
  • Diarrhea: watery stools, may be transient.
  • Nausea: occasional feeling of sickness.
  • Upper respiratory tract infection: nasopharyngitis.

Serious or Rare Side Effects

  • Liver enzyme elevation: increased ALT/AST requiring monitoring.
  • Cataracts: lens opacity observed in some patients.
  • Rash or severe skin reactions: including Stevens‑Johnson syndrome.

Precautions & Warnings

  • When prescribing ivacaftor, several safety considerations should be observed.
  • Liver function monitoring: baseline and periodic ALT/AST tests are recommended.
  • Drug interactions: review concomitant medications, especially CYP3A inducers or inhibitors.
  • Pregnancy and lactation: safety not established; use only if benefits outweigh risks.
  • Respiratory status: monitor for worsening symptoms, especially in severe disease.
  • Cardiac considerations: assess QT interval if patient has risk factors.
  • Store at ambient temperature: keep below 25 °C, retain in original packaging, protect from moisture.

Avoid Interactions

Ivacaftor can interact with other medicines; some combinations should be avoided while others require careful monitoring.

Major Interactions (Avoid)

  • Strong CYP3A4 inducers (e.g., rifampin, carbamazepine): may markedly reduce ivacaftor exposure.
  • Strong CYP3A4 inhibitors (e.g., ketoconazole, clarithromycin): can increase ivacaftor levels and risk of toxicity.

Moderate Interactions (Monitor Closely)

  • Azole antifungals (e.g., fluconazole): may increase ivacaftor concentrations modestly.
  • Macrolide antibiotics (e.g., erythromycin): may elevate ivacaftor exposure.
  • Oral contraceptives: ivacaftor may reduce efficacy, consider alternative contraception.

Frequently Asked Questions

Kalydeco is prescribed for the treatment of cystic fibrosis in patients who have at least one CFTR gating mutation, such as G551D, and who are six years of age or older. It works by improving the function of the defective CFTR protein, which helps to enhance lung function and reduce disease complications.

Ivacaftor binds to the CFTR protein and increases the likelihood that the chloride channel stays open. This potentiation restores chloride transport across epithelial cells, leading to better hydration of airway surfaces, thinner mucus, and improved respiratory function in individuals with specific CFTR mutations.

The dosage of Kalydeco is individualized by the prescribing clinician based on the patient’s age, weight, and specific CFTR mutation profile. The prescriber determines the appropriate tablet strength and dosing schedule, and patients should follow the exact regimen provided in their prescription.

Safety data for ivacaftor during pregnancy and lactation are limited, and the product is classified as having an unknown pregnancy category. It should only be used when the potential benefits to the mother outweigh any possible risks to the fetus or infant, and the decision should be made in consultation with a healthcare professional.

To request Kalydeco, submit an enquiry on the product page at gnhindia.com specifying the required quantity. GNH India supplies hospitals, pharmacies and procurement teams across international markets, verifies trade or institutional credentials, and then provides pricing, availability, shipping details (including any cold‑chain requirements) and the necessary import documentation.

Kalydeco (ivacaftor) is a CFTR potentiator used for gating mutations, whereas combination products such as lumacaftor/ivacaftor (Orkambi) or tezacaftor/ivacaftor (Symdeko) combine a corrector with a potentiator to treat the F508del mutation. Kalydeco is typically chosen when a patient has a gating mutation that responds directly to potentiation, offering a more targeted approach.

Kalydeco tablets should be stored at ambient temperature, preferably below 25 °C. Keep the tablets in the original blister pack to protect them from moisture and light. Do not store in a refrigerator or freezer, and keep out of reach of children.

Kalydeco is taken orally as a tablet, usually with a glass of water. The tablets can be swallowed whole or, if necessary, crushed and mixed with soft food, provided the patient consumes the entire mixture promptly. Administration should follow the schedule prescribed by the treating physician.

Serious risks include elevations in liver enzymes (ALT/AST), which require regular monitoring, the possible development of cataracts, and severe skin reactions such as Stevens‑Johnson syndrome or toxic epidermal necrolysis. Patients should report any signs of liver dysfunction, visual changes, or rash to their healthcare provider immediately.

Kalydeco should not be used by individuals with a known hypersensitivity to ivacaftor or any of the tablet excipients. It is also contraindicated in patients with severe liver impairment or those taking strong CYP3A4 inducers that would markedly reduce drug exposure. Clinical judgment is required for patients with other significant comorbidities.

Published by

GNH India Pharmaceuticals Limited

Licensed pharmaceutical supplier

Last updated:

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