FDA Has Fast-Tracked a Long-Awaited Cystic Fibrosis Drug That May Help 90% of Patients

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A new cystic fibrosis therapy dramatically improved patients’ lung function and showed clear signs of targeting the genetic root of the disease, instead of just alleviating symptoms — a breakthrough so long-sought that many doctors and patients are moved to tears when talking about it.
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The data, being unveiled Thursday at a national conference in Tennessee and simultaneously published in two leading medical journals, was so persuasive that the Food and Drug Administration last week approved the three-drug combination, called Trikafta — five months ahead of the agency’s deadline.

The drug could benefit 90 percent of patients with the disease, a major advance over previous drugs that worked in a tiny fraction of the people with the disease or had more modest effects.

“I’m overjoyed,” said Francis Collins, the director of the National Institutes of Health, who was part of one of the teams that in 1989 discovered the gene defect that causes cystic fibrosis.

“Thirty years along, with many bumps along the road and so many people waiting and hoping that something like this would happen — and here we are.”

The drug is the product of decades of steady, incremental scientific work that began with research in academic laboratories and was pushed forward and funded by patient advocates through an unusual “venture philanthropy” model now being emulated by other patient groups.

The leap forward was preceded by many steps — Trikafta is the fourth therapy developed by Vertex Pharmaceuticals, a Boston-based company that has built a lucrative franchise around the disease.

Cystic fibrosis affects an estimated 30,000 people in the United States. Thick mucus builds up in the body’s organs, damaging people’s lungs and digestive systems.

Patients wear vibrating vests to break up the mucus and spend hours each day coughing to keep their lungs clear. They assiduously protect themselves from respiratory illnesses that can send them to the hospital.

They often take antibiotics, enzymes and vitamins to stay healthy. The life expectancy of patients has been increasing, and patients born today live on average 44 years.

Doctors who began their careers at a time when there were few adults with cystic fibrosis because patients died in their teens are now cautiously anticipating that the disease will be transformed into a chronic condition, akin to diabetes, that can be managed with a drug regimen — particularly if Trikafta is eventually approved for use in younger children and babies, before any lung damage has occurred. (It is initially approved for patients 12 and older.)

Patients who were unsure about whether they should bother attending college because they had always known they would die young are now being told they should think about planning for retirement.

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