FDA's approval of Kalydeco ivacaftor for a small subset of cystic fibrosis patients - three months ahead of the PDUFA goal and five and a half years after Vertex Pharmaceuticals Inc. started the first clinical trial of the molecule - is powerful proof that the alignment of venture philanthropy; smart, aggressive drug development; and efficient regulatory oversight can accelerate the creation of a breakthrough therapy.

Some aspects of the story are unique to Kalydeco, but many of the lessons learned could be broadly applied, according to FDA officials, senior managers at Vertex and the Cystic Fibrosis Foundation (CFF).