Vertex Pharmaceuticals Inc. announced today that it submitted a New Drug Application to the U.S. Food and Drug Administration for the approval of the first triple-combination therapy for cystic fibrosis.
Yesterday, President Donald J. Trump signed the Right to Try bill into law. The House approved the bill last week and the Senate passed it last summer.
Corbus Pharmaceuticals reported promising results Thursday from an early stage clinical study of a potential anti-inflammatory drug for people with cystic fibrosis.
CF Foundation's $1 Million Investment Helped Drug Known as Cayston® Become a Reality
Legislation Would Increase Patient and Expert Participation in FDA Review of Rare Disease Treatments
The U.S. Food and Drug Administration (FDA) approved the use of ivacaftor (Kalydeco®) today for people ages 2 and older with cystic fibrosis who have at least one of the following five splice mutations: 3849+10kbC->T, 2789+5G->A, 3272-26A->G, 711+3A->G, E831X.
The U.S. Food and Drug Administration (FDA) today approved ivacaftor (Kalydeco®) for people ages 2 and older who have at least one of 23 residual function mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.
Galapagos and Cystic Fibrosis Foundation Therapeutics Sign €1.3 million* Agreement
Cystic Fibrosis Foundation Therapeutics to Provide Alnylam with $1.5 Million in Funding for Discovery Efforts