Vertex Cystic Fibrosis Treatment Gets Green Light From FDA


A federal advisory committee to the Food and Drug Administration (FDA) has submitted a formal recommendation to approve a drug developed by Vertex Pharmaceuticals that could possibly treat patients diagnosed with the most common form of cystic fibrosis disease.

The advisory panel to the FDA voted twelve to one, in favor of the twice-a-day drug, which Vertex plans to sell under brand name Orkambi, based on the safety and effectiveness on data submitted. The FDA officials are expected to decide by July 5 whether or not to give the go-ahead signal for the sale of the drug in the US. The FDA's decision is not guaranteed by the advisory panel's vote, but often follows its recommendation.

The drug Orkambi is the second cystic fibrosis drug under Vertex and would be the breakthrough follow-up treatment in the world that acts directly to neutralize the genetic abnormality that induces the disease, instead of just treating the symptoms. The company trusts Orkambi will boost the business to steady profitability after 26 years in the pharmaceutical industry.

Vertex has been the known distributor of Kalydeco, which became the first drug that treats the primary genetic defect of cystic fibrosis back in 2012, but it has been applicable to an exclusive group of only 2,000 out of the 30,000 patients with cystic fibrosis in the US.

Orkambi is a combination of ivacaftor, the ingredient in Kalydeco, and a new compound called lumacaftor. An FDA official suggested that Orkambi's effect on lung function did not appear more distinguished than that of ivacaftor alone for the test patients, creating doubts whether the second drug, lumacaftor, contributed to anything.

Usually, a combination drug like Orkambi must be presented to be better than either compound alone to merit an approval.

However, previous studies had recommended the two components individually did not work, and the study of ivacaftor individually left some uncertainty to how effective it could be.

But most of the panel agreed that in the end it won't matter. The Orkambi combination drug has treated the disease and there are no obvious safety concerns with the mixture compared with ivacaftor alone.

The brand new drug, Orkambi, is projected for patients who have two copies of F508del, the most frequent mutation of cystic fibrosis. About 14,000 patients are in this group, though the drug would be approved initially only for the approximately 8,500 of them who are at least twelve years old. This accounts for around 28 percent of those in the US who suffer from cystic fibrosis. Vertex is now testing the drug in children as young as six years old.

Cystic fibrosis is an inherited disease caused by genetic mutations. Sticky mucus develops in the lungs, resulting to a gradual decline in the capability to breathe. Death from respiratory failure often happens when patients are in their 20s, 30s or 40s.

Photo: frankieleon | Flickr

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