Breakthrough In Fight Against Cystic Fibrosis

(RTTNews) - Treatment with a combination of two drugs could improve the quality of life of patients with cystic fibrosis.

Results of two late-stage trials published in the New England Journal of Medicine showed that treatment with the two drugs - lumacaftor and ivacaftor - produced significant improvements in lung function, and also lowered the speed of pulmonary exacerbation as compared to placebo.

Ivacaftor is known by the brand name Kalydeco and is from the stable of Vertex Pharmaceuticals Inc. (VRTX). Kalydeco is approved in the U.S., Europe, Canada, Australia and New Zealand to treat people with CF who have specific genetic mutations in the CFTR gene.

Cystic fibrosis is a rare, life-threatening genetic disease affecting about 75,000 people in North America, Europe and Australia. The disease is caused by defective or deficient cystic fibrosis transmembrane conductance regulator or CFTR protein activity. In this, the body overproduces thick mucus that leads to chronic lung infections and pancreatic problems.

People with two copies of the F508del gene mutation represent the largest group of patients with cystic fibrosis. There are about 8,500 people aged 12 and older with two copies of the F508del mutation in the U.S.

The randomized controlled trials that targeted the foremost common genetic reason for cystic fibrosis included a total of 1,108 people, aged 12 and older, who were treated for six months.

Pamela Davis, dean of the school of medicine at Case Western Reserve University School of Medicine, Cleveland, said in an accompanying editorial, "Nevertheless, this is the beginning of effective therapy for cystic fibrosis associated with the most common mutant form of CFTR. With further drug development to avoid drug-drug interactions, even the challenging Phe508del CFTR mutation will almost surely come under excellent therapeutic control."

Vertex Pharma said last Tuesday that the U.S. Food and Drug Administration or FDA's Pulmonary-Allergy Drugs Advisory Committee voted 12 to 1 to recommend approval of the company's drug Orkambi for use in people with cystic fibrosis aged 12 and older, who have two copies of the F508del mutation in the CFTR gene.

Orkambi is a combination of approved drug Kalydeco and Vertex Pharma's investigational compound Lumacaftor.

The FDA is expected to make a decision on the approval of Orkambi by July 5. If approved, Orkambi will be the first and only medicine to treat the underlying cause of cystic fibrosis for eligible people with cystic fibrosis aged 12 and older with two copies of the F508del mutation in the CFTR gene.