Vertex Announces CF Combination Drug Milestones and Progress

Patrícia Silva, PhD avatar

by Patrícia Silva, PhD |

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One of the approaches to developing novel treatments for difficult-to-manage conditions such as cystic fibrosis (CF) is taking FDA-approved, well-studied therapies and combining them with other drugs to produce more effective formulations. Along these lines, Vertex Pharmaceuticals has just announced several of their upcoming research and development milestones for combination drugs indicated for CF. These announcements were made during the 28th Annual North American Cystic Fibrosis Conference (NACFC) in Atlanta.

Their updates include new combinations with Kalydeco (ivacaftor), which by itself is currently approved in North America, Australia, and Europe for the treatment of nearly 3,000 CF patients, 6 years old and older. Kalydeco has of course made headlines in 2014 after proving its efficacy in landmark clinical trials and receiving FDA approval. Now, thanks to recent trials, when combined with Lumacraftor, it can be used to treat CF patients 12 years old and above who have the gene mutation F508del. This particular mutation is present in at least 22,000 people in the US, Europe, and Australia.

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Another update from Vertex covers a combination of ivacaftor and the company’s second CFTR corrector, VX-661, also indicated for those carrying the F508del mutation. Vertex has announced that the 12-week Phase 2 study for this combination is already fully enrolled, and that the community can expect conclusive findings early in 2015. As of now, the company is planning for a potential landmark Phase 3 program, and will open subject enrollment within the first half of 2015.

Additionally, the company is working on an experimental triple combination of VX-661, ivacaftor, and one of its next-generation correctors, which Vertex plans to launch into clinical testing next year. This follows highly promising in vitro results.