FDA Approves Trikafta®, Symdeko®, and Kalydeco® For Certain Rare Mutations

The U.S. Food and Drug Administration (FDA) has approved the expanded use of 3 cystic fibrosis (CF) drugs. These include:1

All 3 drugs can now be used for people with a wider range of CFTR (cystic fibrosis transmembrane conductance regulator) gene mutations. For Trikafta, the expansions will impact people 12 years and older. For Symdeko and Kalydeco, the expansions will affect even younger people, starting at 6 years old and 4 months old, respectively.1

People with CF can find out if their mutation is impacted by this new approval by using an online tool available through the makers of these 3 drugs, Vertex Pharmaceuticals. You can also get more details by talking with your doctor.1

Why is this approval important?

The new expansions will now make over 600 people with rare CFTR mutations eligible for treatment with these drugs. Trikafta will now be approved for 177 additional types of mutations. Previously, it only had 1 approved mutation. Symdeko and Kalydeco were originally approved for a larger number of different mutations than Trikafta. However, this approval will expand their use even further to include more than 120 additional mutations for Symdeko and nearly 60 more for Kalydeco.1

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This is big and exciting news in the field of CF, as more people than ever before will be able to use these new treatment options. Additionally, it allows for more flexibility and choice in treatment plans for those with CF. People who may have only been eligible for 1 (or no) CFTR-targeting drugs may now have several different options to choose from.

How do Trikafta, Symdeko, and Kalydeco work?

The active ingredients in all 3 drugs affect the CFTR, a protein made by the CFTR gene. The CFTR normally sits on the outside of cells and acts as a gateway. This plays a big role in salt and water balance in the body. In CF, the CFTR does not work properly and can lead to thick mucus buildup and symptoms. CF drugs like Trikafta, Symdeko, and Kalydeco that target CFTR help this channel work better and lead to thinning of mucus.1

Evidence for the new approval

The makers of these 3 drugs developed a way to test their effectiveness against different types of CFTR mutations in a laboratory setting. The results of these studies, called in vitro studies, showed that more mutations could benefit from their use than previously thought. This means no human participants were needed for the approval. The data and in vitro approach used was strong enough to lead to the expanded use. New drugs often have to go through rigorous safety and efficacy trials. However, drug expansions can sometimes take a different, faster route, like in this case.1

What are the possible side effects of Trikafta, Symdeko, and Kalydeco?

The most common side effects remain the same for each of the 3 drugs and are overall similar. These include:1

  • Headache
  • Common colds (sore throat, stuffy or runny nose)
  • Abdominal pain or diarrhea
  • Nausea
  • Dizziness
  • Rash

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

Things to know about Trikafta, Symdeko, and Kalydeco

Trikafta, Symdeko, and Kalydeco may impact the eye and increase a person’s risk of developing cataracts (clouding of the lens of the eye). Your doctor will monitor your eyes with regular eye exams while taking these drugs.1

Trikafta can cause changes in lab values, including liver function tests and bilirubin (a product in the blood involved in waste breakdown). Your doctor may need to monitor some of these lab tests closely while you are taking the drug.1

Trikafta, Symdeko, and Kalydeco can interact with other drugs. Before taking these medicines, tell your doctor about any other drugs, vitamins, or supplements you are taking. This includes over-the-counter drugs.

For more information, read the full prescribing information for each drug:

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