Vertex Seeks Approval of Trikafta, Symdeko, and Kalydeco for Rare Mutations

The U.S. Food and Drug Administration (FDA) has accepted 3 supplemental new drug applications (sNDAs) from Vertex Pharmaceuticals that seek approval for cystic fibrosis (CF) drugs for additional rare CFTR mutations. The drugs include:1

Trikafta® (elexcaftor/tezacaftor/ivacaftor and ivacaftor)
Symdeko® (tezacaftor/ivacaftor and ivacaftor)
Kalydeco® (ivacaftor)

If these drugs are approved for this use, Trikafta, Symdeko, and Kalydeco will be able to help about 600 more people living with rare CF mutations in the United States.1

In late 2019, Trikafta was approved for people 12 years and older with the CF F508del mutation. That approval was significant for the community, since the mutation is found in about 90 percent of people who have CF. If Trikafta, Symdeko, and Kalydeco are approved for rare mutations, this would signal groundbreaking progress for treatment options in the CF community.2

How is this application different?

If these sNDAs are approved, people living with these rare CF mutations would have access to these different therapies. The sNDAs are also requesting approval for certain people receiving one of the therapies to then be eligible for the other approved therapy:1,3

  • People who are eligible for Symdeko may be eligible for Trikafta
  • People who are taking Kalydeco should also be eligible for Trikafta or Symdeko

Right now, each of these therapies is approved by the FDA to treat those with specific CF-causing mutations in the CFTR gene. Researchers believed that each CFTR modulator drug would only be effective for people with the specific mutation. However, new research has shown that many rare CFTR mutations respond to one or more of these drugs – not just the mutation that specific drug is for. Data from a phase 3 clinical trial has also led to the inclusion of about 30 additional ultra-rare and rare mutations in the United States for Kalydeco and Symdeko.3

In the past, similar research data was used to support an application that expanded the number of mutations that Kalydeco is approved for. That application was approved.3

How can this help people with rare mutations?

The FDA is reviewing the applications and is expected to make a decision on the applications by the end of the year. If approved, this means that approximately 600 more people living in the United States with rare CF mutations can be treated with these drugs. Also, more than 1,100 people with CF in the United States who are currently eligible for Symdeko or Kalydeco may have the option of another CFTR modulator. More treatment options and accessibility to treatment is essential in medical conditions, especially ones like CF.3

Things to think about

Each person with CF is different. If you are currently taking one of these drugs, talk with your doctor about what this might mean for your treatment options. Depending on the genetic mutations you have with your CF, these drugs may or may not be right for your treatment. Your doctor can give you more information about these drugs and what to expect if these sNDAs are approved.

Do you have a rare CF mutation? Participate in our forum specifically for those who are part of this 10%.

Editor's Note: Since the publication of this article, Vertex has approved Trikafta®, Symdeko®, and Kalydeco® for certain rare mutations.

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