Cystic Fibrosis Cure & Treatment: Vertex's Orkambi Gets FDA Approval

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Aa new combination drug developed by Vertex Pharmaceuticals Inc. may potentially cure underlying cause of the most common form of cystic fibrosis.

Cystic fibrosis is a genetic and life-threatening disorder that affects the lungs and digestive system by causing infection and inflammation brought about by thick mucus buildup. There is no known cure for this condition, notes the Cystic Fibrosis Foundation, and medications in the market are only treating related symptoms, causing patients to live up to their mid-20s only.

Reuters reports that Orkambi, a combination of Vertex's Kalydeco and a new compound called lumacaftor, was approved by the Food and Drug Administration (FDA) on Thursday. Its aims to treat the root cause of cystic fibrosis in patients aged 12 and above.

The pharmaceutical company stated that Orkambi will cost $259,000 per year in the United States, $41,000 less than the price of Kalydeco. Analysts have expected the cheaper price, believing that the move emulates the immediate need for treatment at an affordable rate.

Analysts predict that Orkambi will generate estimated annual sales of $5.9 billion worldwide, following the recent 25 percent rise in the net sales of Kalydeco in 2014.

Fierce Biotech reports that Orkambi is designed to attack the F508del gene mutation, the most common form of cystic fibrosis found in roughly 8,500 patients, according to Vertex. Kalydeco fights a different CFTR mutation and is used by about 2,000 patients.

During its initial test, Orkambi showed increased lung function improvement compared to a control test subject. Although the FDA has initially pointed out that the trial did not prove the addition of lumacaflor is better than the usage of Kalydeco alone, it has not stopped its approval of Orkambi. 

With the FDA's decision, Vertex broadened its lead against competitors in cystic fibrosis treatment technology. The approval could further help the company increase its net income for the next few years.

CNBC News writes that according to Vertex's CEO, Dr. Jeffrey Leiden, the approval of Orkambi is the start of significant progress for both the company and the affected families in finding a cure for the most common form of cystic fibrosis.

Half of the 30,000 Americans suffering from the disease are expected to benefit from Orkambi, which may further decrease the estimated 70,000 affected individuals worldwide.