Vertex Report Positive Results for Cystic Fibrosis Combo

Vertex have announced positive late-stage clinical trial results for their drug combo to treat cystic fibrosis (CF), taking a leap closer to regulatory filing.

Vertex confirmed that the combination of lumacaftor and Kalydeco, resulted in statistically significant improvements in lung function compared to placebo, for patients aged 12 years and over with two copies of the gene mutation F508del.

Mean absolute improvements in lung function (percent predicted forced expiratory volume in one second, or ppFEV1) of between 2.6 and 4.0 percentage points from baseline compared to placebo were observed in the phase III clinical trial, with mean relative improvements of 4.3% to 6.7%.

If this effect is sustained long-term it could make a big difference to patents with cystic fibrosis, as on average, patients with two copies of the F508del can expect to lose almost 2% of their lung function yearly.

Results for the combo also showed improvement across a number of key secondary endpoints, with noteworthy reductions in the rates of pulmonary exacerbations and improvements in both body mass index, and the proportion of patients with at least a 5% relative improvement in lung function.

Lead investigator, Bonnie Ramsey, noted that “the significant improvements in pulmonary exacerbations are particularly important given the potential for these events to result in hospitalisations, permanent lung damage and the need for additional treatment with antibiotics and other medicines.”

The cystic fibrosis drug combo is the “first regimen designed to address the underlying cause of CF for people with the most common form of the disease” noted Jeffrey Chodakewitz, Chief Medical Officer at Vertex, adding that based on the results, Vertex “plan to move as fast as possible to submit applications for approval of this combination regimen in countries around the world.”

After the announcement, shares in Vertex jumped over 50% in pre-market trading on Tuesday.


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