Vertex Asks FDA to Expand CFTR Modulators to Additional Mutations

Vertex Pharmaceuticals Inc. has submitted three supplemental New Drug Applications to the U.S. Food and Drug Administration (FDA) to expand eligibility for Trikafta® (elexacaftor/tezacaftor/ivacaftor), Symdeko® (tezacaftor/ivacaftor and ivacaftor) and Kalydeco® (ivacaftor) to additional rare CFTR mutations.

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Today, Vertex announced that it has submitted three supplemental New Drug Applications to the FDA to expand the eligibility for Trikafta® (elexacaftor/tezacaftor/ivacaftor), Symdeko® (tezacaftor/ivacaftor and ivacaftor) and Kalydeco® (ivacaftor) to additional rare CFTR mutations.

If the applications are approved, Vertex estimates that more than 600 individuals who were not previously eligible for CFTR modulators could gain access. In addition, the applications included requests to allow certain people with CF who are currently eligible for Kalydeco to be approved for Symdeko or Trikafta, and certain people with CF who are eligible for Symdeko to gain access to Trikafta. These expansions could provide new therapeutic options for an estimated 1,100 people with CF. Vertex did not disclose in their announcement which rare mutations were included in its applications to the FDA.

The FDA submissions are based on laboratory evidence that suggest these medicines may be effective for additional rare CFTR mutations. This most recent submission to the FDA shows the progress and promise of “theratyping” in which rare mutations are tested in the lab (using an in vitro cell assay) to determine whether they respond to 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. A decision on the applications is anticipated by December 30, 2020.

For additional information, please see the Vertex press release.

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