Ivacaftor and Lumacaftor Create Positive Results for Common CF Mutation

Recently revealed results from a successful Phase 3 study combining Ivacaftor {Kalydeco} and Lumacaftor {VX-809} are extremely positive for patients with a common Cystic Fibrosis {CF} mutation. Patients who have the mutation of Cystic Fibrosis, which entails two copies of the F508del, have demonstrated in Phase 3 clinical trials an effective response to treatment with the Ivacaftor and Lumacaftor drug combo. Patients with CF who were administered with the dynamic duo Ivacaftor and Lumacaftor displayed significant improvements in their lung function and a sizable reduction in their rate of pulmonary exacerbations. This encouraging study data has led Vertex, the company who markets these potent agents, to prepare a New Drug Application {NDA} for the FDA by the close of 2014.

Ivacaftor and Lumacaftor CF Mutation

CFF chartThe Phase 3 study of Ivacaftor and Lumacaftor was comprehensive and included two 24 week trials which were conducted at 200 sites in Australia, Europe and North America. Over 1,100 people, ages twelve and older, who held two copies of the F508del mutation participated in these Phase 3 trials. There were two dosages of Ivacaftor and Lumacaftor administered to the participating patients, which were compared to a placebo arm of the study. The participants who received either of the Ivacaftor and Lumacaftor combination doses showed tangible improvement in lung function, as well as a lowered rate of pulmonary exacerbations as compared to the placebo group. Patients dosed with the Ivacaftor and Lumacaftor duo also experienced healthier outcomes and even weight gain as compared to the patients who were given a placebo. Both Ivacaftor and Lumacaftor have proven to work like a well-oiled machine together, as Lumacaftor works by moving the F508del CFTR protein to the cell surface and Ivacaftor improves its function and helps to increase the regular flow of fluids and salt in and out of the cell.

The innovations in research and development of drugs to treat patients with Cystic Fibrosis has been monumental, and the life expectancy for a child diagnosed with CF has more than doubled over the past three decades. Continued research and development is clearly needed, and Ivacaftor and Lumacaftor look to be at the forefront of these R&D projects. LGM Pharma is a provider of Ivacaftor, CAS# 873054-44-5 and Lumacaftor, CAS# 936727-05-8 for research and development purposes. Clients can be assured of quality API products and continuous support throughout the R&D process.


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