The Most Important New Drug Of 2012: Kalydeco?

Matthew Herper, a science and medicine blogger for Forbes, says that Kalydeco, a new drug which treats cystic fibrosis, "is a triumph of genetics and drug development." The pharmaceutical concern is Vertex, which also makes Incivek for hepatitis C.

He points out that Kalydeco itself will only help about 2,800 patients worldwide. As such, it is not the numbers this one drug will serve which make it significant. Rather, Mr. Herper claims "it’s probably the most exciting as a harbinger of drugs to come."

Herper gives three reasons why he believes Kalydeco is important. The first portends a future for drugs which fix genetic abnormalities, for which there is great need far beyond one type of cystic fibrosis:

•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.

The second has to do with how the R&D was funded:

•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.

The third suggests drugs which help very few people can be profitable:

•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.

I defer to Mr. Herper's expertise to evaluate the importance of any new drug. I do think, however, it is worth noting that, despite a vast fortune spent trying to develop new drugs which take advantage of advances in genetics research, little or nothing before Kalydeco has borne fruit. So if this drug is the first of many more that help people as a result of the human genome project, it is, I would think, as promising a development as Mr. Herper says.