The Food and Drug Administration has approved a drug for cystic fibrosis patients who have the most common mutations associated with the disease.
The agency said Monday that it had approved Boston-based Vertex Pharmaceuticals’ Trifakta (elexacaftor/ivacaftor/tezacaftor and ivacaftor) as the first triple-combination therapy for patients aged 12 and older who have at least one F508del mutation in the CF transmembrane conductance regulator, or CFTR gene, estimated to represent about 90 percent of patients overall. The approval came five months ahead of the March 19, 2020 target date that the agency had set when it accepted the application for the drug in August.
Shares of Vertex were up around 9 percent Monday afternoon and into Tuesday morning following the news.
The company said that the drug’s approval means that about 6,000 patients with one minimal function mutation and one F508del mutation have a medicine to treat the underlying cause of their disease, while 12,000 people with two F508del mutations currently eligible for its three other FDA-approved CF drugs will be eligible for Trifakta. Vertex also markets Kalydeco (ivacaftor), Orkambi (lumacaftor/ivacaftor) and Symdeko (tezacaftor/ivacaftor and ivacaftor). Vertex has also submitted a regulatory approval application to the European Medicines Agency and is evaluating the drug in younger patients, those aged 6-11.
“Today’s approval is a historic moment in cystic fibrosis care, with the potential for more people to benefit from CFTR modulator therapy to treat the basic defect of their disease,” said Dr. Steven Rowe, director of the University of Alabama’s CF research center, in a statement for the company.
In a note to investors, Cowen analyst Phil Nadeau wrote that initial uptake of the drug would likely come from those with one minimal function mutation and one F508del mutation – referred to as “Het Min” patients – who aren’t currently taking a Vertex drug, while those with two F508del mutations who are taking Orkambi or Symdeco and one F508del mutation and a gating mutations who are taking Kalydeco would switch more gradually. Although patients with gating mutations are treated well with Kalydeco, the drug’s broad label would allow even them to switch, Nadeau wrote.
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