‘It’s a game changer,’ new drug to treat cystic fibrosis holds promise

Posted at 12:10 AM, Jul 07, 2015
and last updated 2015-07-07 06:00:06-04

RICHMOND, Va. -- A new drug just approved by the Food and Drug Administration is showing promise in treating the underlying cause of cystic fibrosis, a debilitating disease that attacks the lungs, digestive system, and other parts of the body.

Doctors believe the drug, known as Orkambi, might eventually help treat more than half of patients suffering from the fatal disease and could help lead to a cure.

Six-year-old Philip Hopkins of Amelia County is just one young patient who doctors believe will eventually be helped by Orkambi. The drug dramatically improves lung function in CF patients and guards against future lung damage.

Philip was three weeks old when he was diagnosed with cystic fibrosis. His mother, Catherine Hopkins, said her son must undergo extensive breathing treatments at least twice a day to prevent thick mucus from forming in his lungs and digestive track.

Hopkins said her family has been waiting on FDA approval for this breakthrough drug since Philip was born.

“I told him one day he wasn’t going to have to do all those therapies,” Hopkins said. “He was going to get up and leave the house and play.”

The initial drug before Orkambi, Kalydeco, was considered a breakthrough drug because of its ability to improve lung function. Unfortunately, the drug only showed promise in 4 percent of CF patients. Only 2,000 people in the United States have a certain mutation that responds to the drug.

Orkambi, manufactured by Vertex Pharmaceuticals, extended the treatment to thousands more CF patients because it targeted patients who have two copies of the most common mutation, called F508del.

Dr. Joel Schmidt, a pediatric pulmonologist at VCU Medical Center, said VCU was one of two Virginia hospitals that participated in clinical trials for the new drug.

Schmidt said his team of researchers became emotional after seeing the remarkable results in some patients. The loss of lung function is what leads to chronic infections, and eventually death in most CF patients before their 40th birthday.

“For the younger patients who have not had years and years of fighting the progression of lung disease, it’s probably going to have the biggest impact,” Schmidt said. “It will keep their lung function stable, hopefully.”

While FDA approval for Orkambi has only been granted for patients 12 years and older, Schmidt said the drug will eventually be extended to patients as young as two years of age.

For Philip Hopkins family, it’s a first giant step toward a cure.

“He won’t have to go to the doctor and be poked and prodded,” Catherine Hopkins said. “It’s just going to be a normal life.”