Kalydeco: The most important new drug of 2012
Kalydeco, a drug for cystic fibrosis, is the most important new drug of 2012 according to Forbes magazine and was developed by Vertex pharmaceuticals with seed funding from the Cystic Fibrosis Foundation.
Cystic Fibrosis is a genetic disorder that results in scarring (fibrosis) and cyst formation within the pancreas, lungs, liver, and intestines.
Kalydeco, given alone, will only help a few thousand patients the world over. Like other drugs for very rare diseases, its price is very high: $294,000 per patient per year.
Though its chemical structure could be routinely made by a synthetic chemist, it is covered by a patent so it is illegal to make in a lab.
The efforts to cure cystic fibrosis were spearheaded by a discovery from Francis Collins, later famous for heading the Human Genome Project and then the National Institutes of Health, who discovered the gene that, when mutated, causes cystic fibrosis 23 years ago. Kalydeco is the first drug to directly affect the defects caused by these mutations, leading to improvements in patients’ lung function.