Vertex Pharmaceuticals, a relatively new biotech startup, has gained FDA approval for the first drug to target the underlying cause of a rare form of cystic fibrosis. Unlike other current cystic fibrosis medications, which treat only the symptoms of the disease, the new drug Kalydeco targets the disease at the level of a defective cell protein. And while this particular defect is the cause of cystic fibrosis in only about 1,200 people in the U.S., the new treatment suggests the potential of using the same drug development strategies for more typical cystic fibrosis cases, which Vertex is currently investigating.
Cystic fibrosis patients suffer from abnormally sticky mucus buildup in the lungs and other organs, leading to respiratory problems, infections, digestive problems, and death in young adulthood. Kalydeco is designed to correct for a genetic abnormality that s responsible for the mucus that clogs a patient s airways. And in clinical trials, it appeared to be doing just that: Patients taking the twice-a-day pill increased their lung strength by more than 10 percent, compared to those taking the placebo. The patients on Kalydeco also suffered fewer infections and were able to gain an average of seven pounds a significant amount, given that they typically struggle to put on any weight at all.
ACSH s Dr. Ruth Kava was excited to learn about the new drug and believes it surely bolsters the sense of possibility for treatment of a variety of other conditions.
In fact, Vertex evidently regards the success of Kalydeco as so beneficial to pharmaceutical research overall that the company will provide the expensive drug ($294,000 a year) for free or at a subsidized rate for patients meeting particular income and insurance criteria.
ACSH's Dr. Josh Bloom is greatly impressed with what Kalydeco represents. Vertex is not a company that only goes after blockbuster drugs, he notes. They spent 20 years on this project knowing that the chances of making a profit were slim to none. And the science involved was really impressive.
New hope for cystic fibrosis sufferers
Vertex Pharmaceuticals, a relatively new biotech startup, has gained FDA approval for the first drug to target the underlying cause of a rare form of cystic fibrosis. Unlike other current cystic fibrosis medications, which treat only the symptoms of the disease, the new drug Kalydeco targets the disease at the level of a defective cell protein.
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