Recently, the New York Times posted an article stating that a new cystic fibrosis drug is available on the market. Kalydeco, the drug developed by Vertex Pharmaceuticals, is capable of correcting a mutation that occurs on the G551D gene. The G551D mutation is one of the rare forms of mutations in patients with cystic fibrosis, and therefore the drug will cost an estimated amount of about $294,000 annually.The article stated that ivacaftor, Kalydeco's generic name, took years to develop because the researchers could not figure out how the mutations evetually led to the actual disease. The pharmaceutical company has two other drugs in the clinical trial phase, and plans to finish trials and get approval as soon as possible.
This was a great post, and the article itself is an interesting read. However, I'm not entirely sure from the article itself how the drug "counters the effect of one specific mutation" that causes CF. Is it gene therapy, where the affected genomic sequence is corrected, or does the drug act on metabolic intermediates that have causation on CF? Very interesting nonetheless.
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