The cystic fibrosis community received news worth celebrating this week.
The Food and Drug Administration approved a new drug that helps address the underlying cause of cystic fibrosis, not simply its symptoms. The drug, a combination of tezacaftor/ivacaftor, is for patients aged 12 and older with certain cystic fibrosis mutations.
In clinical trials, the drug combination was responsible for improved lung function and reduced exacerbation. Patients likewise showed improvement in quality-of-life indicators. The new drug may also help patients who were unable to tolerate an alternative treatment.
Roughly 46 percent of cystic fibrosis patients who have two copies of the most common mutation stand to benefit from the new drug. Beyond that, patients who have one of 26 specified mutations can also use the newly approved drug.
This fast-tracked approval is exciting, because few treatments for cystic fibrosis exist. And more good news could be on the horizon. Researchers plan to begin late-stage clinical testing of the combination drug jointly with a third therapy in mid-2018. The triple combination could potentially benefit up to 90 percent of cystic fibrosis patients.
Nevertheless, ongoing research and development are of paramount importance. For a small segment of the cystic fibrosis community, effective treatment remains elusive.
Meanwhile, even patients who can benefit from the approved combination drug may struggle to access them. Medicaid barriers can complicate access, and patients are frequently subjected to prior authorization before they can receive the necessary cystic fibrosis medication.
For more on cystic fibrosis therapies and access barriers, read the Cystic Fibrosis Engagement Network’s “Making Treatment Accessible for Cystic Fibrosis Patients.”