In the span of just over 24 hours earlier this month, the FDA approved two major new medications, both fruits of the past twenty years’ worth of genomic research. One, Kalydeco, is for cystic fibrosis; the other, Erivedge, is for metastatic, basal-cell skin cancer.
Both of these drugs are emblematic of avant-garde, precise, individualized medicine in which a specific genetic mutation in a patient is matched up with the action of the drug. Kalydeco is effective in the 3 to 4 percent of CF-positive children who carry a specific mutation known as G551D—very effective: a small trial of 140 children with the mutation, comparing Kalydeco with a placebo, showed marked improvement in breathing status, weight gain, and every other parameter that was assessed.
Read the full op-ed on the Forbes website.