Introduction

Ivacaftor (trade name: Kalydeco) is approved in Germany for the treatment of cystic fibrosis in babies aged 6 months and over, children, teenagers and adults.

Cystic fibrosis (sometimes shortened to CF) is an inherited metabolic disease. It is caused by changes (mutations) in both copies of the gene called the CFTR (cystic fibrosis transmembrane conductance regulator) gene. This gene is responsible for making a regulator that influences the balance of salt and water in the mucus-producing gland cells, for example in the pancreas, bronchi and small intestine. If mutations cause the regulator to become dysfunctional, the mucus is very thick and sticky. In the lungs, this thick mucus can’t be coughed up, which makes it hard to breathe and may cause a chronic cough. Bacteria can also collect in the mucus, repeatedly causing respiratory infections. Thick and sticky digestive juices damage the pancreas and reduce the body’s absorption of important nutrients in the bowel, increasing the risk of malnutrition and being underweight. The symptoms of cystic fibrosis already appear in children.

Over 2,000 different mutations of the CFTR gene have been identified. They are named according to the position in the gene where the mutation has occurred. The most common of these mutations is called F508del. Usually both copies of this gene have a defect ("homozygous"). All of these mutations prevent the CFTR from functioning properly.

Ivacaftor aims to improve the functioning of the CFTR, so that the mucus becomes less thick and sticky, and the symptoms improve.

Application

Ivacaftor is available in the form of tablets and granules:

  • 50 or 75 mg granules
  • 150 mg tablets

The recommended dose and form of application depends on the person's age, type of gene mutation, body weight and combined treatment. When used in tablet form, ivacaftor is taken with a meal containing fat. The granules are mixed with soft foods or liquids, and a meal or snack containing fat should also be eaten before or afterwards too.

Other treatments

Best supportive care (BSC) is an option for treating cystic fibrosis. BSC should be tailored to the patient’s individual needs. It aims to relieve symptoms such as breathing difficulties as well as improve quality of life. It may include inhalation therapy, antibiotics, mucus-thinning medications, physical therapy and changes to their diet.

The fixed-dose combination of lumacaftor / ivacaftor is an option for people aged 12 years and over who have a homozygous F508del mutation.

Assessment

In 2020, the Institute for Quality and Efficiency in Health Care (IQWiG, Germany) looked into whether ivacaftor has any advantages or disadvantages in the treatment of cystic fibrosis

  • compared to best supportive care in babies aged 6 months and over, children, teenagers and adults, and
  • compared to the combination of lumacaftor and ivacaftor in people aged 12 years and over who have a homozygous F508del mutation.

The manufacturer provided usable data for the following groups:

  • Children aged 6 years and over, teenagers aged 12 and over, and adults who have a G551D mutation
  • Adults who have an R117H mutation
  • Teenagers aged 12 and over and adults who have a homozygous F508del mutation (ivacaftor in combination with tezacaftor)

Learn more

More information

This information summarizes the main results of reviews produced by the IQWiG. The reviews were commissioned by the German Federal Joint Committee (G-BA) as part of the “early benefit assessment of medications.” On the basis of the reviews and the hearings received, the G-BA passed a resolution on the added benefit of ivacaftor (Kalydeco) for the treatment of cystic fibrosis.