FDA on Monday announced that it has approved Trikafta, the first-ever triple combination therapy to treat cystic fibrosis (CF) in patients ages 12 and older who have a genetic mutation that affects the vast majority of CF patients.
CF is a progressive, life-threatening disease that causes thick mucus to accumulate in parts of the body, including the lungs and digestive tract. The disease results in serious respiratory and digestive problems, along with other complications, according to FDA.
Vertex Pharmaceuticals' Trikafta is a combination of elexacaftor, ivacaftor, and tezacaftor. The drug is approved to treat patients 12 and older who have at least one F508del genetic mutation, which is estimated to affect about 90% of the CF population, or about 27,000 people in the United States, according to FDA.
Trikafta was shown to be effective in two clinical trials. The first was a 24-week, randomized, double-blind, placebo-controlled trial in 403 patients with an F508del mutation. The second was a four-week, randomized, double-blind, active-controlled trial in 107 patients with two identical F508del mutations.
In the trials, Trikafta was shown to improve lung function by about 14% compared to about a 3% improvement in patients treated with Orkambi, a double-combination CF treatment currently available.
Trikafta will cost $311,503 annually, or $23,896 per 28-day pack, according to the Securities and Exchange Commission. Analysts expect the drug to make $630 million in 2020, Reuters reports.
Acting FDA Commissioner Ned Sharpless in a release said, "In the past few years, we have seen remarkable breakthroughs in therapies to treat cystic fibrosis and improve patients' quality of life, yet many subgroups of cystic fibrosis patients did not have approved treatment options. That's why we used all available programs ... to help advance today's approval in the most efficient manner possible, while also adhering to our high standards."
Reshma Kewalramani, chief medical officer at Vertex, in a release said, "The incredible speed of this approval underscores our shared sense of urgency with the CF community for bringing this medicine to eligible people with CF, particularly those without a medicine targeting the underlying cause of their disease" (Fernandez, Axios, 10/21; Maddipatla/O'Donnell, Reuters, 10/21; Brooks, Medscape, 10/21; FDA release, 10/21).
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