Kalydeco™ is a new oral medication for the treatment of cystic fibrosis, approved by the U.S. Food and Drug Administration (FDA) in January 2012.
It is the first drug available that targets the underlying cause of CF — a faulty gene and its protein product, CFTR."
The drug description goes on to explain further about the effects of the drug. What this drug did is demonstrate that the gene that causes a lack of chloride secretion which thickens the mucous in the conducting airways can be reversed, thereby allowing CF patients to breathe normally.
Only on this MB are the differences among drugs ignored.
Arikace will be competing with other inhalation antibiotics to treat Pseudomonas aeruginosa in CF patients, then bronchiectasis patients, then other types of pneumonia and (hopefully) the big ticket item, NTM. But these approvals will take years to develop. Not next year, or necessarily 2014. But relatively soon by drug development timelines.
Till VRTX had to admit that they only could serve 4% of the CF market.... in NYT: The drug, called Kalydeco and developed by Vertex Pharmaceuticals, counters the effect of one specific mutation in the gene that accounts for 4 percent — or about 1,200 — cystic fibrosis cases in the United States.