Souce: Vertex Pharmaceuticals press release
Vertex Pharmaceuticals says it plans to begin additional studies of KALYDECO™ in children with cystic fibrosis as young as two years of age and in people with CF who have certain mutations that were not evaluated in the previous Phase 3 studies. Pending final feedback from regulatory agencies, the company plans to begin three clinical studies of KALYDECO in mid-2012.
In December, Vertex announced that the U.S. Food and Drug Administration (FDA) accepted the New Drug Application for KALYDECO and granted the company's request for six-month Priority Review. A target review date of April 18, 2012, is set under the Prescription Drug User Fee Act for the FDA's approval decision. Vertex's marketing authorization application for KALYDECO has also been validated by the European Medicines Agency, which accepted Vertex's request for accelerated assessment in Europe.
As Vertex prepares for the potential launch of KALYDECO for people with the G551D mutation, the company is also planning to begin additional studies of KALYDECO in children with CF as young as two years of age and in people with CF who have certain mutations that were not evaluated in the previous Phase 3 studies.
Pending final feedback from regulatory agencies, the company plans to begin three clinical studies of KALYDECO in mid-2012:
- Pediatric study: A study of KALYDECO in children ages 2 through 5 with gating mutations, including G551D, is expected to evaluate the safety, tolerability and effect on sweat chloride and other measures of clinical activity using a pediatric formulation of KALYDECO.
- Study in people with the R117H mutation: Vertex plans to begin the first clinical study of KALYDECO in people who have at least one copy of the R117H mutation in the CF gene. The R117H mutation causes abnormal function of the CFTR protein at the cell surface. Approximately 3 percent of people with CF in the U.S. have the R117H mutation.
- Study in other gating mutations: Vertex also plans to begin the first clinical study of KALYDECO in other gating mutations where CFTR proteins are present at the cell surface but do not function properly. G551D is the most common gating mutation, present in approximately 4 percent of people with CF in the U.S., and was the focus of previous Phase 3 KALYDECO studies. The remaining gating mutations to be evaluated in this study account for an additional approximately 1 percent of people with CF in the U.S.
Additional studies of KALYDECO and Vertex's pipeline medicines in development are ongoing or planned for 2012, including:
- Two CFTR correctors in development for people with the most common CF mutation, F508del: Enrollment is ongoing in the second part of a Phase 2 clinical trial of combination regimens of KALYDECO, a CFTR potentiator, and VX-809, a CFTR corrector, in people with the most common mutation in CF, known as F508del. In addition, Vertex plans to begin Phase 2 development of VX-661, a second CFTR corrector, in the first quarter of 2012. Data from the study with VX-809 is expected mid-year, followed by data from the study with VX-661 later in 2012.