Vertex gains FDA approval for cystic fibrosis treatment

First US approval for Sanofi’s rare blood disorders franchise
Credit: Castleski

The US Food and Drug Administration has granted approval for Vertex Pharmaceuticals’ Symdeko for treating the underlying cause of cystic fibrosis in people aged 12 and over.

Symdeko is Vertex’s third medicine approved to treat the underlying cause of CF. The company says it is “ready to launch” the therapy and will begin shipping to pharmacies in the US this week.

“The approval of Symdeko, our third disease-modifying CF medicine, offers many patients an important new treatment option,” said Jeffrey Leiden, Chairman, President and CEO of Vertex.

“This approval is an important milestone in our journey to treat every person with CF, and we remain committed to urgently advancing our efforts to develop new medicines that treat the underlying cause of CF for the many people still waiting.”