Vertex seeks FDA approval stamp for breakthrough CF drug

Vertex has hit another big milestone of 2011. The Cambridge, MA-based drug company ($VRTX), which began marketing its potential blockbuster HepC drug Incivek this year, has given the FDA its application for approval of a game-changing cystic fibrosis therapy that produced some remarkable results in late-stage trials. And potential approvals of the drug--which has the generic moniker ivacaftor and is code named VX-770--could give the standout developer a second big cash cow.

The company has asked U.S. regulators for a priority review of the drug--which it would like to market under the name Kalydeco--that could shorten the review time from 10 months to 6 months. The FDA does expedited reviews for breakthrough drugs, and Vertex made a compelling case for VX-770 with pivotal data revealed earlier this year. Results of one Phase III study of the drug showed a 10.6% mean improvement in lung function in patients on the treatment, more than twice the level of efficacy needed to achieve statistical significance and garnering high marks for the program from analysts and physicians.

This has been a closely watched program among biotech investors and those advocating for better drugs to treat CF, a genetic disease that is triggered by mutated CFTR genes and leads to chronic lung infections and damage, resulting in premature deaths of patients with the illness. The 13-year project to advance VX-770, which has received support from the CF Foundation, could lead the first FDA-approved medicine that treats the root cause of the disease. Still, the company is seeking approval for the drug to treat CF patients with at least one copy of the G551D mutation to the CFTR gene, and the patients who fit that bill represent only 4% of the CF population and about 1,200 patients in the U.S.

"The CF Foundation is thrilled that our collaboration with Vertex has contributed to an important potential new medicine for the treatment of some people with CF," said Robert Beall, president and CEO of the CF Foundation, in a statement. "The results of the [VX-770] studies have opened the door to a new way of treating CF and we hope that this approach will lead to the development of other targeted medicines for all people with this disease."

Expect more news on this program. Vertex aims to file its application for approval of VX-770 with the European Medicines Agency by the end of this month. The EMA has already granted the company an expedited review for the drug to reduce the time for agency's assessment of the application from about 7 months to roughly 5 months.

- here's Vertex's release
- get more in Xconomy's article

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