Breakthrough Cystic Fibrosis Treatment on the Horizon
June 7, 2019
Elated. Nervous. Hopeful. That’s how thousands of cystic fibrosis patients felt upon hearing last week’s news: the Food and Drug Administration will soon review a new triple combination therapy that could drastically improve their quality of life.
Many cystic fibrosis patients spend hours each day inhaling breathing treatments and using a specialized vest that breaks up secretions in their lungs. Breakthrough treatments can reduce the need for these measures and free patients to engage more fully with family and friends, be more active in their communities or more present at school or work.
The new drug is a first-of-its-kind treatment that includes a CFTR modulator called elexacaftor along with drugs tezacaftor/ivacaftor, which the FDA approved in 2017. As a CFTR modulator, the new medication treats the underlying cause of cystic fibrosis, not just its symptoms.
Late-stage clinical trials show the triple combination therapy increased lung function by more than 14 percent for a subset of cystic fibrosis patients whose genetic mutations isn’t treatable with current medications.
Because elexacaftor will be the first drug to treat this cystic fibrosis mutation, it’s a prime candidate for the FDA’s Fast Track process. Being on the fast track reduces how long it takes to review a new drug application without compromising safety. In short, it makes the treatment available to patients sooner.
Yet amid excitement about the new drug, access challenges are already on advocates’ minds. Cystic fibrosis patients often endure utilization management barriers like prior authorization and step therapy, which can delay patients’ ability to get medications. Having to work through these barriers would devastate patients who have waited their whole lives for a drug designed specifically for them.
The percentage of cystic fibrosis patients who will have a drug that treats their mutation type will expand to 90 percent when the new triple combination therapy hits the market. In addition to bringing the novel treatment to patients in the United States, the drug’s manufacturer also signaled it will submit an application to the European Medicines Agency before the end of 2019.
Read the Cystic Fibrosis Engagement Network’s “Making Treatment Accessible for Cystic Fibrosis Patients” for more information about treatment options and the access barriers patients face.
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