Vertex Pharmaceuticals wins US Approval for Orkambi to treat cystic fibrosis

Vertex Pharmaceuticals has received green signal from the FDA for its combination drug Orkambi for the treatment of most common form of cystic fibrosis. As per the analysts, the approval will prove extremely beneficial for the company.

If the circumstances are considered of May then it was not sure that that Vertex Pharmaceuticals' combination therapy for the most common form of cystic fibrosis will get approval. But after few days, the drug Orkambi got approved.

The drug has been made by combining Kalydeco with lumacaftor. It is expected that the FDA will widen its approval area for the drug for patients having cystic fibrosis and are six years or older. By expanding the approval, more number of people will be benefitted.

Jeffrey Leiden, Vertex's chairman, president and CEO, said, "The approval of ORKAMBI represents a fundamental change in the treatment of the most common form of CF, marking significant progress for us and for the entire CF community".

Leiden said that in clinical trials, Orkambi has shown a 2.5 to 3% improvement in lung function when compared with placebo. The medication's annual cost will be $259,000. It is bit higher than the predicted gross price of the drug, which was in the range of $230,000 to $250,000 per year.

Cystic fibrosis is serious genetic disease that leads to lung infections and early death. Talking about the two drugs from which the drug has been made are Kalydeco and lumacaftor. The former one was developed for patients having a problem with a protein on their cell walls.

The second drug, lumacaftor, is said to be the first drug meant to treat the main cause of disease in such people.