FDA officials support Vertex’s drug

An experimental combination drug’s approval has been supported by federal health advisers; the drug is aimed at treating cystic fibrosis. They are recommending the approval in the face of unsolved questions regarding the effect of one of the pill's ingredients.

Vertex Pharmaceuticals is seeking approval of its drug for patients with the most common form of cystic fibrosis, which is a deadly inherited disease that leads to sticky mucus buildup in the lungs. A panel of the Food and Drug Administration expert advisers voted 12-1 supporting approval of the drug, on the basis of safety and effectiveness data put forward by Vertex. Cystic fibrosis troubles approximately 30,000 people in the United States.

The new drug of Vertex would be sold with brand name Orkambi. The drug is Vertex’s follow-up to its advanced pill Kalydeco. In 2012, Kalydeco became the first drug to treat the primary cause of cystic fibrosis.

Vertex Chief Medical Officer Jeffrey Chodakewitz, in a statement, “The positive recommendation brings the cystic-fibrosis community one step closer to potential approval of the first medicine to treat the underlying cause of this disease for many more people”.

It is expected that by July 5, officials will decide whether or not the drug will be approved for sale in the United States. The Vertex stock had been stopped during the panel meeting. When the trading started again, individual shares rose 7% to $132.80.

The new drug is expected to be of great significance. According to the company, in the beginning, Orkambi would be used to treat nearly 8,500 patients with the F508del mutation. This accounts for around 28% of those in the US who have been attacked by the disease.