FDA clears drug for leading form of cystic fibrosis

The doctor added that he expects the FDA will expand its approval for Orkambi to patients with CF who are six years or older to increase the amount of people who would benefit from the new treatment.


Orkambi (lumacaftor 200 mg/ivacaftor 125 mg) is now approved to treat cystic fibrosis (CF) in patients 12 years and older, who have the F508del mutation, which causes the production of an abnormal protein that disrupts how water and chloride are transported in the body.

The long-expected approval of Vertex Pharmaceuticals’ new combination drug for cystic fibrosis is finally here. This particular form occurs when a child takes on two genetic mutation copies-one from each of the parent. It causes sticky mucus to form in the lungs as well as other organs, leading to infections, digestive problems and an early death.

Vertex has an assistance program in place, called Vertex Global Positioning System, to help ensure that patients will be able to afford this pricey medicine; Chief Commercial Officer Stuart Arbuckle says that most Kalydeco patients pay less than $50 a month out of pocket, and Vertex plans to continue the strategy with Orkambi.

Orkambi will carry a wholesale price tag of $259,000 per year in the United States, the company said in a regulatory filing.

Vertex Pharmaceuticals Inc. just received approval from the Food and Drug Administration for a new medicine that could provide treatment to up to half of the estimated 30,000 Americans suffering from the deadly respiratory disease cystic fibrosis.

On the same day, The Scientific Advisory Committee for FDA, a group of individual specialists, attended the presentation from FDA staff, Vertex councils, and patients while recommending Orkambi beforehand for approval. Orkambi is designed for the roughly 8,500 patients whose CF is caused by the so-called F508del mutation in their CFTR genes, making up the disease’s largest population. “It is our hope that everyone who is prescribed this drug will have immediate access to it so they can begin to live longer, healthier lives”, said Cystic Fibrosis Foundation CEO Robert Beall, in a statement. It is the second drug approved by the FDA that targets the underlying cause of CF. Ivacaftor (as a single therapy) was the first.


Orkambi improves lung function and significantly reduces the rate of pulmonary exacerbations, which can lead to frequent hospitalizations and accelerated lung disease. Officials said the drug appeared safe and that even small improvements could help fill a significant unmet need for a disease that has no cure.

FDA clears drug for leading form of cystic fibrosis | News OK