Why Is Theratyping Important?
For this reason, researchers, supported by the Foundation, are testing CFTR modulators on
In December 2020, the FDA used theratyping data from Vertex Pharmaceuticals to expand approval of three CFTR modulators to include additional people with CF who have certain rare mutations. The approval enabled 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.
In addition to this testing, the Foundation also is funding theratyping testing at Cincinnati Children’s Hospital Medical Center on nasal cells from people with CF who don't currently qualify for a modulator. The results are shared with treating physicians and can be used for discussions with public and private insurers about prescribing modulators off-label.