"This transaction supercharges our efforts to develop lifesaving therapies in order to pursue daring, new opportunities that one day may lead to a permanent, lifelong cure for this disease.”
Robert Beall, PhD, President and CEO, Cystic Fibrosis Foundation.
KALYDECO is an FDA-approved therapy for the treatment of cystic fibrosis (CF).
KALYDECO™ is a cystic fibrosis transmembrane conductance regulator (CFTR) potentiator which corrects the mutated genes in patients with cystic fibrosis – a novel therapeutic approach designed to treat the root cause of cystic fibrosis instead of treating the severity of symptoms. KALYDECO has been approved for people with CF ages 2 and older who have at least one of the following rare CF mutations: G551D, G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, G1349D and R117H.
APPLICATIONS IN DEVELOPMENT
1) Kalydeco + tezacaftor and 2) Kalydeco + tezacaftor + 2nd. generation corrector (VX-440, VX-152, VX-659 or VX-445)
Cystic Fibrosis (CF)