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E.U has approved Kalydeco to treat people with CF

On July 27, Vertex, the company who developed Kalydeco (ivacaftor, formerly known as VX 770), has announced that the EU has approved Kalydeco to treat people with CF, in ages 6 and older, who have at least one copy of the G551d mutation. Vertex is and will continue to work with appropriate local regulatory authorities over the next few weeks to make Kalydeco available in different countries.

On June 28, 2012, Vertex Pharmaceuticals announced the final results of a Phase 2 clinical trial of Kalydeco™ in combination with VX-809, showing significant improvements in lung function in people with the most common CF mutation, Delta F508.

Both Kalydeco (formerly called VX-770 and also known by its generic name, Ivacaftor and VX-809, a CF drug in development, are designed to treat the root cause of cystic fibrosis. Vertex released preliminary results from the Phase 2 trial earlier this year.  

The 56-day study enrolled 109 people, ages 18 and older, with one or two copies of Delta F508. People in the study with two copies of Delta F508 (the most common CF mutation) who received the highest dose of VX-809 combined with Kalydeco showed the greatest improvement in lung function. That means an absolute improvement in lung function of 6.1 percentage points within group (p < 0.001) and 8.6 percentage points compared to placebo (p < 0.001) in homozygous patients receiving combination treatment (Day 28 to 56) with highest study dose of VX-809 -600mg.

Based on the final results, Vertex plans to begin a pivotal trial of the combination treatment in people with two copies of Delta F508 in early 2013.

Participants with one copy of the Delta F508 mutation also showed improvements in lung function, compared with those who received a placebo — though smaller than the improvements seen in those with two copies of Delta F508. Vertex plans to conduct additional studies of Kalydeco and VX-809 in those with one copy of Delta F508.

Adverse events were similar between treatment and placebo groups; most events were mild to moderate.

In people with the G551D mutation of CF ages 6 and older, Kalydeco helps improve the function of a defective protein, called CFTR, which causes the sticky mucus and symptoms in CF. In people with the G551D mutation, the defective protein moves to the right place at the surface of the cell but does not function correctly. Instead, it acts like a locked gate, preventing the proper flow of salt and fluids in and out of the cell. Kalydeco helps unlock that gate and restore the function of the CFTR protein, allowing a proper flow of salt and fluids on the surface of the lungs. This helps to thin the thick, sticky mucus caused by CF that builds up in the lungs.

As we announced here before on this site, the European Medicines Agency (EMA) announced on May 25 that it has recommended the approval of the CF drug Kalydeco™ for people with this G551D mutation aged 6 years and older in the European Union. The recommendation is being sent to the European Commission, which has the authority to approve medicines in the European Union. The European Commission generally follows the recommendation of the EMA and typically makes a decision on approval of a drug within three to four months. The FDA approved Kalydeco in January 2012 for people with the G551D mutation of CF ages 6 and older. 

The DeltaF508 mutation is more complex and therefore a combined therapy is tried out. In people with the F508del mutation, CFTR proteins do not reach the cell surface in normal amounts. VX-809, known as a CFTR corrector, aims to increase CFTR function by increasing the trafficking, or movement, of CFTR to the cell surface. VX-770, known as a CFTR potentiator, aims to increase the function of defective CFTR proteins by increasing the gating activity, or ability to transport ions across the cell membrane, of CFTR at the cell surface.

Vertex developed Kalydeco and VX-809 with significant financial, clinical and scientific support from the US Cystic Fibrosisii Foundation.

Photo via CF Worldwide Blog

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