Blogs

Vertex Pharmaceuticals this week submitted its new cystic fibrosis (CF) drug, Kalydeco, to the US Food and Drug Administration (FDA), seeking expedited review. The drug is designed to treat a rare form of CF affecting just 4% of the approximately 30,000 CF patients in the US, who have a mutation of a gene known as G551D. It was developed as a result of a unique collaboration between Vertex and the Cystic Fibrosis Foundation, and will be the subject of a special closing session at 2011 RAPS: The Regulatory Convergence. Panelists from CFF and Vertex, and other regulatory experts will discuss the implications for drug development, personalized medicine and increasing involvement of patient groups.

Read more:

• Vertex Seeks FDA Green Light for Cystic Fibrosis Drug, Second Potential Hit of Big Year (Xconomy)
• Gene Discovery Was Only First Step (Wall Street Journal)
• Vertex Submits Application for Priority Review and Approval of KALYDECO™ (VX-770, ivacaftor) in the U.S. as First Potential Medicine to Target the Underlying Cause of Cystic Fibrosis (Vertex press release)