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Kalydeco

Vertex Pharmaceuticals Incorporated

Drug Names(s): VX-770, VX 770, ivacaftor

Description: VX-770 is a potentiator that may act to restore the function of the cystic fibrosis transmembrane conductance regulator (CFTR) protein, the defective cell membrane protein responsible for the progression of CF. Defects in the CFTR protein affect the transport of chloride and other ions across cells, and lead to the accumulation of thick, sticky mucus in the lungs of patients with CF. This mucus fosters chronic infection and inflammation, and results in irreversible lung damage. VX-770 is designed to increase the probability that the CFTR channel is open, which could result in an increase in chloride transport across the cell surface in some patients. In laboratory experiments, using cells from patients with CF where CFTR proteins are present on the cell surface, VX-770 has restored the function of defective CFTR channels.

Deal Structure: Vertex reports US sales for Kalydeco as approximate sales

Vertex and Cystic Fibrosis Foundation
In May 2004, Vertex entered into a collaboration agreement with Cystic Fibrosis Foundation Therapeutics (CFFT), the non-profit drug discovery and development affiliate of the Cystic Fibrosis Foundation, pursuant to which CFFT provided Vertex with funding for its CF research and development programs. Two drug candidates currently in clinical development for CF, VX-770 and VX-809, were discovered by Vertex under this research collaboration. Vertex retains the right to develop and commercialize any compounds discovered in the course of the research collaboration, including VX-770 and VX-809, and will pay a royalty to CFFT on the net sales of any approved drugs discovered in the collaboration.

In April 2011, Vertex and the Cystic Fibrosis Foundation expanded their collaboration to include VX-661. As part of the collaboration, CFFT will provide Vertex with up...See full deal structure in Biomedtracker

Partners: Royalty Pharma AG


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