FDA Approves Kalydeco (Ivacaftor), The First CFTR Modulator For Eligible Infants With Cystic Fibrosis

The U.S. Food and Drug Administration (FDA) has expanded the use of Kalydeco (ivacaftor) for some infants with cystic fibrosis.1

Previously, Kalydeco was for people with CF ages 6 months and older with specific mutations in their CFTR genes. Now, Kalydeco can be used for infants between the ages of 4 months and 6 months with qualifying mutations. This will make it the first treatment option of its kind (CFTR modulator) approved for infants this young.1

How does Kalydeco work?

In CF, an important gene called the cystic fibrosis transmembrane conductance regulator (CFTR) gene is abnormal. This abnormality, called a mutation, is what leads to the development of CF and its symptoms. The CFTR gene makes a protein called the CFTR protein. This protein is involved in the balance of chloride (part of salt) in the body and its fluids. CFTR acts as a gateway to let chloride in and out of cells.1,2

When chloride balance is off due to a malfunctioning CFTR gene and CFTR protein, bodily secretions like mucus can become thick and sticky. This is what leads to the symptoms of CF in the lungs and other areas of the body.1,2

The active ingredient in Kalydeco is ivacaftor. Ivacaftor belongs to a class of drugs called CFTR modulators. More specifically, ivacaftor is called a potentiator. Potentiators help keep a faulty CFTR protein open to allow more chloride to pass through. This helps improve overall chloride balance and can help thin mucus, thus reducing CF symptoms.1,2

Kalydeco for infants in clinical trials

The FDA granted the expanded approval of Kalydeco based on data from a small group of infants between the ages of 4 months to less than 6 months with CF. The group included 6 infants who were studied for 24 weeks to assess the safety of Kalydeco in this age range. Overall, the researchers found that the safety of Kalydeco in infants between 4 months old and less than 6 months old was similar to that of older individuals.1,2

Those involved in the study had one of 10 mutations in their CFTR gene. For this reason, these 10 mutations are the only ones Kalydeco is approved for in the 4- to 6-month age group at this time. These mutations include:1,2

  • G551D
  • G178R
  • S549N
  • S549R
  • G551S
  • G1244E
  • S1251N
  • S1255P
  • G1349D
  • R117H1

If a person’s CF mutation is not known, an FDA-approved test can be used to determine the mutation and find out which treatment options are appropriate.

What are the potential side effects?

The most common side effects of Kalydeco include:1,2

These are not all the possible side effects of Kalydeco. Talk to your doctor about what to expect or if you experience any changes that worry you.

Things to know about Kalydeco

As with any drug, there are several rare but serious side effects to consider. Before starting Kalydeco, tell your doctor if you have a history of liver issues. Kalydeco may affect liver function tests and may require regular monitoring.2

There is not enough information about Kalydeco and pregnancy to know if this drug is safe to use when pregnant. The risk of birth defects and miscarriage is not known. Before using this drug, tell your doctor if you are pregnant, plan to become pregnant, or are breastfeeding.2

Kalydeco may impact a person’s eyesight or increase their risk of cataracts. Consult your doctor if you notice vision changes, and see your doctor for regular eye exams while taking the drug.2

Kalydeco can have major interactions with other drugs. Before starting Kalydeco, tell your doctor about any other medicines you take, including over-the-counter vitamins and supplements.2

For more information, read the full prescribing information of Kalydeco.

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