No stopping Vertex in CF with new FDA approval

Vertex Pharmaceuticals (Nasdaq: VRTX) is another step closer to realizing its ambitious goal of treating every person with cystic fibrosis (CF) and to ensuring healthy revenue figures for the year ahead.

The Boston-based biotech has received approval from the US Food and Drug Administration (FDA) for another of its CF drugs, the third that it can sell in the USA. Vertex already has approval for Kalydeco (ivacaftor) and Orkambi (lumacaftor/ivacaftor), and its newly-approved treatment, called Symdeko, is a combination of Kalydeco and a new medicine called tezacaftor.

Symdeko, which has been approved for treating the underlying cause of CF in people ages 12 and older who have two copies of the F508del mutation in the cystic fibrosis transmembrane conductance regulator gene or who have at least one mutation that is responsive to tezacaftor/ivacaftor, will have a list price of $292,000 per year, a level between Kalydeco and Orkambi.