UPDATED: Vertex nabs expedited FDA OK on breakthrough CF drug

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Vertex Pharmaceuticals ($VRTX) is no longer a one-trick biotech company. The FDA issued an early approval today for Kalydeco (ivacaftor), a new drug that promises to strengthen the lungs and ease symptoms for a slice of the patient population sharing a genetic malfunction.

Vertex, which rose to prominence on the successful development of the groundbreaking hepatitis C drug Incivek (telaprevir), billed the new drug as the first to treat the underlying causes of the disease. And the biotech is promising to expand on its work in the field with new therapies that can help a broader range of patients. After the announcement came out, Vertex officials spread the word that Kalydeco will be sold for $294,000 a year, not unusual for a tiny disease population like this.

Drug developers note: This is the second early FDA action on a targeted therapeutic in as many days. The agency wants to send the message loud and clear that when developers deliver new meds tailored for specific patient populations, regulators will respond with alacrity.

"Kalydeco is an excellent example of the promise of personalized medicine--targeted drugs that treat patients with a specific genetic makeup," said FDA Commissioner Margaret A. Hamburg, M.D. "The unique and mutually beneficial partnership that led to the approval of Kalydeco serves as a great model for what companies and patient groups can achieve if they collaborate on drug development."

About 4% of CF patients have the G551D mutation, some 1,200 people in the U.S. "Kalydeco also provides us with a roadmap for exploring additional targeted approaches to treatment for all people with cystic fibrosis," says Robert Beall, CEO of the Cystic Fibrosis Foundation, which is backing R&D work for CF.

"Even though this drug isn't for the majority of people, it proves that you can look at the mistake in the genes and design a drug in a rational way that will fix the problem," Dr. Drucy Borowitz of the State University of New York at Buffalo tells the AP.

- get the release from the FDA
- read the release from Vertex
- here's the AP report

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