Statement from Emily’s Entourage regarding FDA approval of breakthrough therapy for Cystic Fibrosis
Today is a monumental day in the history of Cystic Fibrosis (CF) treatments and the history of medicine. The FDA announced its approval of the highly-anticipated triple combination therapy, Trikafta, developed by Vertex Pharmaceuticals, for 90% of the CF population ages 12 and older who have at least one copy of the most common CF mutation, F508del. Trifakta does not benefit those with two nonsense mutations or other hard to treat mutations.
Trikafta is a landmark advance for two reasons:
- Its efficacy. The phase 3 trial of Trikafta increased mean lung function by 13.8%, exceeding all expectations and restoring lung function to a level many in the CF community had not experienced in decades.
- Its scope. Trikafta benefits 90% of the CF community with the most common CF mutation (F508del). To date, the most effective therapies have benefited fewer than half the CF population and none have shown increases in lung function at this rate.
To call Trikafta a medical breakthrough would be a vast understatement. Eight years ago, there were no mutation-targeted treatments to treat the underlying cause of CF. As of today, there are now four approved mutation-targeted treatments that benefit 90% of the CF community. This is an extraordinary development, but it also serves as a stark reminder that an additional 10% of the CF community—of which those with nonsense mutations make up the largest group—remain without any kind of treatment.
We formed Emily’s Entourage in 2011 because we saw developments like Trikafta on the horizon and realized that those with nonsense mutations of CF like Emily, would not benefit from any of these advances. People with nonsense mutations of CF were being left behind as their diseases raged on. We refused to accept that fate.
Today, we celebrate a massive victory for the CF community. We are elated for the hope Trikafta provides for 90% of the CF population and the path this blazes for so many other rare diseases. We also believe more strongly than ever that 90% is not enough. We remain steadfast in our commitment and laser focus to extend the progress to 100% of the CF community, including those with nonsense mutations, and to innovate on the drug development process, enabling us to do it faster than has ever been done before.
And while Trikafta is revolutionary, it is not a cure. We must remain vigilant to counter any perception that CF has been eradicated or “cured” for the 10% who still do not have any mutation-targeted treatments, and also for the 90% that benefit from Trikafta, for whom this new therapy serves as a “control” but not a cure for CF.
The work of Emily’s Entourage continues with all due speed until there are breakthroughs and a cure for everyone with CF.
About Emily’s Entourage
Emily’s Entourage (EE) is an innovative 501(c)3 foundation that has raised millions of dollars since 2011 to speed groundbreaking treatments and a cure for rare nonsense mutations of Cystic Fibrosis, a fatal genetic lung disease. The organization was founded by Emily Kramer-Golinkoff, a young woman with advanced stage Cystic Fibrosis, and her devoted friends and family.
The urgency of Emily’s condition is spurring revolutionary research and drug development that could help not only those with Cystic Fibrosis (CF), but people who suffer from a wide variety of genetic diseases caused by nonsense mutations, including forms of muscular dystrophy and cancer. Nonsense mutations, while rare among those with CF, are the cause of approximately 12% of all genetic diseases and affect 30 million people around the world.
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