FDA Approves Expansion of Modulators for People With Certain Rare Mutations

The U.S. Food and Drug Administration (FDA) today expanded its approval of three CFTR modulators to include additional people with CF who have certain rare mutations. The approval enables more than 600 individuals with CF who were not previously eligible for modulators to access drugs that treat the underlying cause of their disease for the first time.

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The U.S. Food and Drug Administration today expanded its approval of three CFTR modulators to include additional people with CF who have certain rare mutations. The approval enables more than 600 individuals with CF who were not previously eligible for modulators to access drugs that treat the underlying cause of their disease for the first time.

The FDA decision expands the labels for Trikafta® (elexacaftor/tezacaftor/ivacaftor), Symdeko® (tezacaftor/ivacaftor), and Kalydeco® (ivacaftor) to include additional rare mutations that were previously not approved for CFTR modulators. Trikafta is now approved for individuals who are 12 years and older with at least one of 177 newly-approved mutations; Symdeko is now approved for individuals who are 6 years and older with one of 127 additional mutations; and Kalydeco is now approved for individuals who are 4 months and older with one of 59 additional mutations.

In addition, the approval enables certain people with CF who are currently eligible for Kalydeco to also gain access to Symdeko and Trikafta. For certain people who are currently eligible for Symdeko, today's decision approves them for Trikafta as well.

The FDA based its decision on laboratory evidence that demonstrates the effectiveness of these modulators for specific mutations, as well as established safety and efficacy data for the drugs. To see a list of all of the mutations currently approved by the FDA for these CFTR modulators, please visit the following web pages:

  • Trikafta (see page 8 for a list of approved mutations)
  • Symdeko (see page 8 for a list of approved mutations)
  • Kalydeco (see page 7 for a list of approved mutations)

This decision shows the progress of “theratyping” in which rare mutations are tested in the laboratory to predict whether people with CF will respond to approved CFTR modulators. The FDA previously relied on a combination of lab results, clinical data, and the drug's established safety record to expand the use of ivacaftor to people with 23 additional mutations in 2017. 

The CF Foundation is committed to ensuring that every person with CF has a treatment for the underlying cause of CF. With more than 1,700 disease-causing mutations of the CFTR gene, CF researchers will continue to use theratyping to generate data that may support the expansion of the number of CFTR mutations treated by CFTR modulators. While only Vertex can submit a request to the FDA to expand the label of its drugs for approval, the Foundation is actively funding and conducting research in order to provide Vertex with relevant information to help facilitate these expansions.

For additional information, please see the Vertex press release.

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Topics
Research | CFTR Modulators
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