Vertex Pharmaceuticals Receives FDA Approval For Combo Cystic Fibrosis Drug

Vertex Pharmaceuticals (VRTX) announced today that it has received FDA approval for its combo Cystic Fibrosis drug known as Orkambi. Vertex already has a drug already approved for a less common form of Cystic Fibrosis that targets a specific genetic makeup. The previous drug Kalydeco, already approved by the FDA, targeted about 1,200 patients with the G551D mutation. 

The new combo drug combines Kalydeco together with lumacaftor to form a combination drug used to treat patients. This new combo drug Orkambi achieved a 2% increased lung function for patients with a  type of Cystic Fibrosis mutation known as the F508del mutation. The previous drug Kalydeco achieved a 10% increased lung function for Cystic Fibrosis patients with the G551D mutation. 

Although 2% in the Orkambi trial is lower than 10% in the Kalydeco trial it seems the FDA wanted to approve Orkambi today anyway because these Cystic Fibrosis patients who are age 12 and older with the F508del mutation have no treatment options at all. They desperately need something that will help them and if Orkambi can at least improve lung function by 2% then that is better than nothing. 

This approval was imperative for Vertex, though, because it adds more stock value to its bottom line. That is because analysts estimate that Orkambi could potentially generate about $5 billion at peak sales. Orakambi is expected to sell with a price tag of $259,000 (wholesale) per patient per year. It is estimate there are 8,500 of these Cystic Fibrosis patients age 12 years and older.