DRUG APPROVED TO TREAT CYSTIC FIBROSIS’ ROOT CAUSE
The first drug that treats the root cause of cystic fibrosis won approval yesterday, offering a life-changing treatment to a small percentage of patients who suffer from a rare form of the deadly illness, and broader hope to thousands more.
About 30,000 Americans live with cystic fibrosis, a disease that causes sticky mucus buildup in the lungs and other organs, leading to infections, digestive problems, and death in young adulthood. The typical life expectancy is about 37 years, according to the Cystic Fibrosis Foundation.
The Food and Drug Administration approved Vertex Pharmicuticals Inc’s Kalydeco for patients with a rare form of the disease that affects just 1,200 people in the US, about 4 percent of the affected population nationwide. These patients have a protein defect that prevents their cells from properly absorbing and excreting salt and water. Studies of the drug showed it significantly improved lung function and reduced other symptoms of cystic fibrosis.
“Even though this drug isn’t for the majority of people, it proves that you can look at the mistake in the gene and design a drug in a rational way that will fix the problem,” said Dr. Drucy Borowitz of the State University of New York at Buffalo, where she directs the cystic fibrosis program.
The twice-a-day pill is among the first drugs designed to correct a specific genetic defect. Its development characterizes both the promise and challenges of that approach. Scientists first identified the gene that causes cystic fibrosis in 1989, but it took more than two decades and more than $75 million in outside funding to develop a drug to treat it.
Borowitz enrolled several of her patients in the key study for Kalydeco, which showed that patients taking the drug increased their lung strength more than 10 percent when compared to patients taking a placebo. Patients also had fewer infections and gained nearly seven pounds on average, significant for patients who typically have trouble retaining weight. All patients in the study continued taking older medications to loosen mucus.
“Two weeks after using the drug my lung tests were above average for a healthy 15 year old who didn’t have cystic fibrosis,” said Nick Mangano, 17, a Borowitz patient who has been taking the drug for two years. Before starting on Kalydeco, Mangano said he was hospitalized for lung infections five times in four years. Now he says he usually recovers from a cold within a week or two.
“I don’t need medicine for it anymore, it’s totally different,” says Mangano, who is considering leaving Buffalo for college next year--a step he hadn’t considered before because of his dependence on family and physicians.
Only a few decades ago, children with cystic fibrosis seldom survived elementary school. Today, thanks to earlier diagnosis and new focus on diet and physical therapy, 47 percent live to be 18 or older.
The FDA approved the drug for patients six years old and up, though Vertex plans to study the drug in patients as young as two. Using the drug earlier might prevent permanent lung damage, which is the primary cause of death for cystic fibrosis patients.
Vertex executives said Kalydeco would cost $294,000 for a year’s supply.
Vertex Executive vice president Nancy Wysenski said Vertex would provide the medicine free to people with no insurance and household income of $150,000 or less. The company will also cover 30 percent of copay costs for select insured patients.