Vertex Pharmaceuticals won approval Monday for a combination drug that is expected to benefit 90 percent of patients with cystic fibrosis, the rare and deadly genetic disease that has long been the focus of the Boston-based biotech company.
The approval by the Food and Drug Administration came five months sooner than the agency had said it would issue its decision on the drug, called Trikafta. The swiftness appeared to surprise executives at Vertex, whose lobby in the Seaport District is adorned with the pledge “All in for CF.”
“The incredible speed of this approval underscores our shared sense of urgency with the FDA and the CF community for bringing this medicine to eligible people with CF, particularly those without a medicine targeting the underlying cause of their disease,” said Dr. Reshma Kewalramani, chief medical officer for Vertex, who will become CEO next year.
The FDA’s acting commissioner, Ned Sharpless, said the agency has been striving to speed the development of therapies for complex diseases.
“Today’s landmark approval is a testament to these efforts, making a novel treatment available to most cystic fibrosis patients, including adolescents who previously had no options, and giving others in the cystic fibrosis community access to an additional effective therapy,” he said.
Vertex has three other cystic fibrosis drugs on the market: Kalydeco, Orkambi, and Symdeko.
The new drug combines the two medicines in Symdeko with a third compound Vertex developed. Trikafta was approved for patients 12 years and older with a specific genetic mutation. It is expected to help 90 percent of the 27,000 cystic fibrosis patients in the country, according to the FDA.
Like Vertex’s three other cystic fibrosis drugs, Trikafta won’t come cheap. Its list price is $311,000 a year, the same as Kalydeco.
Cystic fibrosis is caused by a defective gene that results in a thick, sticky buildup of mucus in the lungs, pancreas, and other organs. People with the disease have inherited two copies of the defective gene, one from each parent.
They have to take a slew of drugs to improve their breathing and ability to process foods. The average life expectancy is about 37, according to the National Institutes of Health.
Among the families celebrating the FDA’s action was Robert K. Coughlin, chief executive of the Massachusetts Biotechnology Council trade group. Coughlin’s son, Bobby, a 17-year-old senior at Dedham High School, has cystic fibrosis. The elder Coughlin called Trikafta’s approval “the best news my family has gotten.”
Bobby takes 50 to 100 pills daily to aid digestion, ward off infections, and treat his organs, he and his family told the Globe last year. He spends at least 45 minutes a day inhaling medicated mists through a nebulizer. For about 20 minutes a day he also uses a contraption that resembles a defibrillator and emits sound waves to loosen mucus in his lungs.
His father said that clinical trial results indicate the combination therapy will be effective to treat Bobby’s mutation. The family is optimistic that “not only will it improve the quality of his life, it will keep him alive,” Coughlin said in a brief phone interview Monday evening.
“This is the best day I have had in 17 years!!!” he added in a text message.
Dr. Steven Rowe, director of the Gregory Fleming James Cystic Fibrosis Research Center at the University of Alabama, said that in clinical trials, Trikafta helped relieve respiratory symptoms and reduce episodes of acute worsening of symptoms known as pulmonary exacerbations.
While the Vertex drugs have already transformed some patients’ lives, their price tags have drawn fire.
Paul M. Quinton, a professor of pediatrics at the University of California San Diego School of Medicine, has cystic fibrosis himself, has spent his career researching the disease, and takes Symdeko.
Quinton told the Globe last December, when he was 74 — and a self-described “outlier” for having survived so long — that the medicine has dramatically improved his health, but the $292,000 cost was “horrible.”
Although Medicare and his wife’s health insurer pick up most of the expense, he said, that’s passed on to taxpayers and insurance customers.
The FDA approved the new drug as Vertex’s CEO, Dr. Jeffrey Leiden, prepares for a major career transition. In July, Leiden announced he will step down as chief executive in April, after seven years in those roles, and become executive chairman.
His handpicked successor is Kewalramani, the chief medical officer, who will become the first female CEO at a top-tier US biotech company.
Given Vertex’s success in treating cystic fibrosis, the company has begun turning to other rare disorders that need effective medicines, from Duchenne muscular dystrophy to sickle cell diseases.
In an interview in August, Leiden said that Vertex was also making progress in developing a new class of drugs for an all-too-common malady: pain.
On Monday, Leiden called Trikafta’s approval “a milestone for CF patients, their families and Vertex.”