New Drug Advances Cystic Fibrosis Care

New Drug Advances Cystic Fibrosis Care 150 150 Kevin Mayhood

Orkambi helps reduce infections and slows loss of lung function in some patients.

Matt Hennessey knew he was on the experimental medicine and not a placebo just days after he began participating in a drug trial. He felt better.

The addition of Orkambi pills to his daily routine was just another part of his maintenance care for cystic fibrosis. Twice each day he donned and inflated the vest that shakes the mucus coating his lungs and stomach to prevent it from becoming stagnant. And he continued swallowing other medicines and inhaling aerosols for the same reason.

“Every other month for 13 years, I would get an infection and go on oral antibiotics and more intensive aerosols,” says Hennessey, 23, of Columbus, Ohio. “Since starting Orkambi, I’ve been on oral antibiotics one time in two years. It’s a remarkable shift, a tangible improvement in my life.”

The Food and Drug Administration approved Orkambi in July. Patients who received the drug during the trials improved slightly. Compared to patients who received placebos, the treatment group’s lung function was 4.3 to 6.7 percent better. They also had 30-39 percent fewer pulmonary exacerbations — infections — and significantly fewer exacerbations that required hospitalizations and intravenous antibiotics.

Maintenance is important with a debilitating disease, says Karen McCoy, MD, chief of the Section of Pulmonary Medicine at Nationwide Children’s Hospital. “This may help some people while we develop an even better drug.”

Dr. McCoy, who led the drug trial in central Ohio, is principal investigator for the Cystic Fibrosis Therapeutic Development Center and the director of the Cystic Fibrosis Center at the hospital.

According to the Cystic Fibrosis Foundation, more than 1,800 mutations affect the cystic fibrosis gene. Orkambi is the first drug approved for the form of cystic fibrosis affecting half of the 30,000 U.S. patients.

Orkambi is actually two drugs. Ivacaftor, sold as Kalydeco, dramatically improves the lives of people with a gene defect that affects less than 10 percent of the cystic fibrosis population, says Dr. McCoy. Ivacaftor alone can’t do much for the larger group of cystic fibrosis patients, but combined with lumacaftor, the drugs help epithelial cells function better.

“The combined drugs do improve the quality of life for some patients,” Dr. McCoy says. “With each infection, a patient may not return to the same lung function he or she had before. Treatment for acute exacerbations takes time and the drugs used are very harsh and can be damaging to other organs, and expensive. For all those reasons, going longer between treatments is a neat marker a patient is better than before the drug.”

For Hennessey, the decreased number of infections and improved quality of life has made Orkambi a valuable part of his maintenance program.


Wainright CE, Elborn JS, Ramsey BW, Marigowda G, Huang X, Cipolli M, Davies JC, De Boeck K, Flume PA, Konstan MW, McColley SA, McCoy K, McKone EF, Munck A, Ratjen F, Rowe SM, Waltz D, Boyle MP; TRAFFIC Study Group; TRANSPORT Study Group. Lumacaftor-Ivacaftor in patients with cystic fibrosis homozygous for Phe508del CFTRNew England Journal of Medicine. 2015 Jul 16;373(3):220-231.

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