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 tablet for oral use. It is indicated for cystic fibrosis in patients who have specific CFTR gating mutations, helping to improve chloride transport in epithelial cells.

GNH India supplies Kalydeco as a licensed, GDP‑compliant international pharmaceutical supplier serving hospitals, pharmacies and clinics worldwide.

Manufacturer / TM Owner

Vertex Pharmaceuticals (Ireland) Limited

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Indications & Clinical Uses

  • Kalydeco is a CFTR potentiator used in the management of cystic fibrosis caused by specific gating mutations.
  • Cystic fibrosis (gating mutations): improves lung function and reduces the frequency of pulmonary exacerbations.
  • Cystic fibrosis (G551D mutation): enhances chloride transport, leading to better respiratory outcomes.
  • Cystic fibrosis (other responsive mutations): supports nutritional status by improving pancreatic function.

How It Works

  • Ivacaftor binds to the CFTR protein at the cell surface and increases the probability that the chloride channel remains open. By stabilizing the open conformation, it enhances chloride ion transport across epithelial membranes, which improves hydration of airway surface liquid and reduces mucus viscosity in patients with responsive CFTR mutations.

Side Effects

Adverse reactions to ivacaftor may vary in frequency and severity.

Common Side Effects

  • Headache
  • Diarrhea
  • Upper respiratory tract infection
  • Nausea
  • Abdominal pain

Serious or Rare Side Effects

  • Elevated liver enzymes (ALT, AST)
  • Pulmonary hemorrhage
  • Severe skin reactions such as rash or Stevens‑Johnson syndrome

Precautions & Warnings

  • When prescribing ivacaftor, clinicians should consider several safety measures.
  • Liver function monitoring: assess transaminases before initiating therapy and periodically thereafter.
  • Drug interaction review: evaluate concomitant use of strong CYP3A4 inhibitors.
  • Pregnancy risk: safety data are limited; discuss potential risks with the patient.
  • Breastfeeding considerations: unknown safety; weigh benefits against possible infant exposure.
  • Adherence advice: take with a fat‑containing meal to maximize absorption.
  • Store at ambient temperature: keep below 25°C, retain in the original packaging, and protect from moisture.

Avoid Interactions

Ivacaftor may interact with other medicines, requiring careful management.

Major Interactions (Avoid)

  • Ketoconazole: potent CYP3A4 inhibitor that can increase ivacaftor exposure.
  • Itraconazole: strong CYP3A4 inhibitor, may raise plasma levels of ivacaftor.
  • Clarithromycin: CYP3A4 inhibitor that can lead to toxicity.
  • HIV protease inhibitors (e.g., ritonavir): significantly elevate ivacaftor concentrations.

Moderate Interactions (Monitor Closely)

  • Rifampin: CYP3A4 inducer that may reduce ivacaftor effectiveness.
  • Oral contraceptives: ivacaftor can decrease hormonal efficacy, consider alternative contraception.

Frequently Asked Questions

Kalydeco (ivacaftor) is prescribed for the treatment of cystic fibrosis in patients who carry specific CFTR gating mutations. By improving chloride channel function, it helps enhance lung function, reduce pulmonary exacerbations, and support overall respiratory health.

Ivacaftor binds to the CFTR protein on the cell surface and increases the likelihood that the chloride channel stays open. This action enhances chloride ion transport across epithelial cells, leading to better hydration of airway surfaces and thinner mucus in patients with responsive mutations.

The dosage of Kalydeco is individualized and determined by the prescribing clinician based on the patient’s age, weight, and specific CFTR mutation. Patients should follow the prescriber’s instructions and not adjust the dose without medical guidance.

Safety data for ivacaftor in pregnancy and lactation are limited. The potential risks and benefits should be discussed with a healthcare professional. Women who are pregnant, planning to become pregnant, or breastfeeding should consult their physician before using Kalydeco.

To place an order, submit an enquiry on the product page at gnhindia.com, specifying the required quantity. GNH India supplies hospitals, pharmacies, and procurement teams worldwide, verifies trade or institutional credentials, and then provides pricing, availability, shipping details, and any necessary import documentation.

Kalydeco is a CFTR potentiator that directly enhances the function of the defective chloride channel, whereas other therapies such as lumacaftor/ivacaftor combine a corrector and potentiator to improve protein processing and function. The choice depends on the patient’s specific CFTR mutation profile and clinical response.

Kalydeco tablets should be stored at ambient temperature, preferably below 25 °C. Keep the medication in its original packaging, protect it from moisture, and store it out of direct sunlight to maintain stability.

Kalydeco is taken orally as a tablet. For optimal absorption, it should be swallowed with a fat‑containing meal. Patients must follow the dosing schedule prescribed by their healthcare provider.

Serious risks include liver enzyme elevations, which require regular monitoring, and rare but severe skin reactions such as Stevens‑Johnson syndrome. Pulmonary hemorrhage has also been reported. Patients should report any unusual symptoms promptly to their clinician.

Kalydeco is not indicated for patients without the specific CFTR gating mutations it targets. Individuals with severe liver disease, known hypersensitivity to ivacaftor, or those taking contraindicated strong CYP3A4 inhibitors should avoid using this medication.

Published by

GNH India Pharmaceuticals Limited

Licensed pharmaceutical supplier

Last updated:

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