The Food and Drug Administration looks set for a great 2012. With a few days left to go, it has approved 40 new drugs and vaccines, one of the most impressive totals ever, according to data from Pharmaceutical Approvals Monthly and FDA press releases. In this haul, one medicine stands out for its scientific and medical importance.
Kalydeco, for cystic fibrosis, is a triumph of genetics and drug development, the first medicine to directly affect the genetic defect that causes the disease. It will only help four percent of the 70,000 people who suffer from declining lung function, damaged pancreases and shortened lives due to CF worldwide, but in those few it has a dramatic effect. It makes medical history for three reasons:
• It’s a genomics triumph: Francis Collins, later famous for heading the Human Genome Project and then the National Institutes of Health, 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.
• A patient group powered its development: Kalydeco would probably not exist were it not for the Cystic Fibrosis Foundation, which funded its early development at Vertex and gets a royalty on the drug. This success paved the way for other disease foundations including the Michael J. Fox Foundation, Myelin Repair, and the Multiple Myeloma Research Foundation.
• Its price: 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. Vertex shares have fallen 37% from their high earlier this year because of doubts by investors that Vertex will succeed in its attempts to dramatically expand Kalydeco’s use by combining it with a second drug that will make it work in CF patients whose disease is caused by other, more common, mutations. Initial results were very promising, but then Vertex had to restate them. Sales of its best-seller, Incivek for hepatitis C, are dropping. But whatever you think of Vertex shares, Kalydeco is already a success, with $113 million in sales in the first nine months of 2012.
Kalydeco was not the only important drug this year, in which the FDA also approved the first flu vaccine made in cells, not chicken eggs (that’s a Novartis product) and several important cancer drugs including Onyx’s Kyprolis, Medivation’s Xtandi, and Roche’s Perjeta. Nor is it the most commercially important — that honor goes to Gilead’s Stribild combination pill for HIV, which could help preserve that company’s HIV franchise through patent expirations. But it’s probably the most exciting as a harbinger of drugs to come.
Reposted from Forbes. Written by Matthew Herper.