Product image of Ivacaftor Elpen (Ivacaftor) supplied by GNH India

Ivacaftor Elpen

Active Ingredient:
Ivacaftor
Origin:
EU

Ivacaftor Elpen (Ivacaftor)

Ivacaftor, the active ingredient in Ivacaftor Elpen, is a CFTR potentiator presented as an oral tablet for oral use. It treats cystic fibrosis in patients who carry specific gating mutations of the CFTR gene, improving lung function and reducing disease severity.

GNH India supplies Ivacaftor Elpen as a licensed, GDP‑compliant international pharmaceutical supplier serving hospitals worldwide.

Manufacturer / TM Owner

Elpen Pharmaceutical Co. Inc.

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Indications & Clinical Uses

Ivacaftor is a CFTR potentiator used to manage cystic fibrosis caused by specific gating mutations. It works by enhancing chloride transport in affected epithelial cells.

  • Cystic fibrosis with G551D mutation: improves pulmonary function and lowers sweat‑chloride levels.
  • Cystic fibrosis with R117H mutation: enhances airway clearance and reduces exacerbations.
  • Cystic fibrosis with other approved gating mutations: increases overall respiratory health and quality of life.

How It Works

  • Ivacaftor binds to the CFTR protein at the cell surface and stabilises its open conformation, thereby potentiating the gating function of the channel. This increases the flow of chloride ions across epithelial membranes, correcting the defective ion transport that underlies cystic fibrosis and improving hydration of airway secretions.

Side Effects

Ivacaftor is generally well tolerated, but several adverse reactions have been reported.

Common Side Effects

  • Headache: mild to moderate intensity.
  • Diarrhoea: occasional, usually self‑limiting.
  • Nausea: transient, may improve with food.
  • Upper respiratory tract infection: mild symptoms.
  • Abdominal pain: intermittent discomfort.
  • Rash: mild skin irritation.

Serious or Rare Side Effects

  • Elevated liver enzymes: requires monitoring of hepatic function.
  • Cataracts: especially in pediatric patients, regular eye exams advised.
  • Severe rash or Stevens‑Johnson syndrome: discontinue if observed.
  • Arrhythmia: monitor cardiac status in susceptible individuals.
  • Pulmonary haemorrhage: rare but potentially life‑threatening.

Precautions & Warnings

Before initiating therapy, clinicians should consider several safety measures.

  • Monitor liver function: obtain baseline tests and repeat periodically.
  • Assess for drug interactions: especially strong CYP3A4 modulators.
  • Evaluate for cataract development: schedule regular ophthalmologic exams in children.
  • Avoid use in patients with known hypersensitivity to ivacaftor or its excipients.
  • Store at ambient temperature below 25°C: keep in the original pack, protect from moisture.

Avoid Interactions

Ivacaftor is metabolised primarily by CYP3A4, making it susceptible to pharmacokinetic interactions.

Major Interactions (Avoid)

  • Strong CYP3A4 inhibitors (e.g., ketoconazole, itraconazole): may increase ivacaftor exposure.
  • Strong CYP3A4 inducers (e.g., rifampin, carbamazepine): may reduce efficacy.

Moderate Interactions (Monitor Closely)

  • Hormonal contraceptives: reduced effectiveness, consider alternative methods.
  • Warfarin: potential alteration of anticoagulant effect, monitor INR.
  • Digoxin: possible increase in serum levels, observe for toxicity.

Frequently Asked Questions

Ivacaftor Elpen is used to treat cystic fibrosis in patients who have specific gating mutations of the CFTR gene. By improving the function of the defective protein, it helps enhance lung function, reduce respiratory symptoms, and improve overall quality of life for eligible patients.

Ivacaftor binds to the CFTR protein located on the surface of epithelial cells and stabilises its open channel configuration. This potentiation increases chloride ion transport across cell membranes, correcting the ion imbalance that characterises cystic fibrosis and leading to better hydration of airway secretions.

The dosage of ivacaftor is determined individually by a qualified prescriber based on the patient’s age, weight, mutation type, and clinical response. Healthcare professionals set the appropriate regimen and adjust it as needed during treatment.

The safety of ivacaftor in pregnancy and lactation has not been fully established, and the product label lists the pregnancy category as unknown. Clinicians should weigh potential benefits against possible risks and discuss options with the patient before prescribing.

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

Ivacaftor is a CFTR potentiator that directly enhances the gating function of the protein, whereas other classes such as correctors (e.g., lumacaftor) improve protein folding and trafficking. For patients with gating mutations, ivacaftor alone often provides greater clinical benefit than combination regimens that target different mechanisms.

Ivacaftor tablets should be stored at ambient temperature below 25 °C, in the original container, protected from moisture and direct sunlight. Keep the product out of reach of children and avoid storing in humid environments such as bathrooms.

Ivacaftor is taken orally, usually with a meal to enhance absorption. The tablets are swallowed whole with water and should be taken exactly as prescribed by the treating physician.

Serious risks include significant elevations in liver enzymes, which require regular hepatic monitoring, and the development of cataracts, especially in younger patients. Rare but severe reactions such as Stevens‑Johnson syndrome, arrhythmias, or pulmonary haemorrhage also warrant immediate medical attention.

Ivacaftor is contraindicated in individuals with known hypersensitivity to ivacaftor or any of its excipients. Patients without a confirmed gating mutation of the CFTR gene are also not eligible for this therapy, as the drug’s mechanism targets that specific defect.

Published by

GNH India Pharmaceuticals Limited

Licensed pharmaceutical supplier

Last updated:

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