Cystic Fibrosis Drug Orkambi Wins Approval

A new drug that is designed to help people who have a specific type of cystic fibrosis has won approval from a federal advisory committee to the Food and Drug Administration. Cystic fibrosis is a inherited disease that has to do with mutations in a gene.

Vertex is the name of a company that created a drug that is called Orkambi. It is the second drug the company has made that works on the underlying genetic defect of cystic fibrosis. The first drug was Kalydeco.

There are differences between the two drugs. Kalydeco has already been approved by the Food and Drug Administration, while Orkambi is awaiting full approval. Kalydeco includes an ingredient called ivacaftor. Orkambi includes both ivacaftor and lumacaftor.

Kalydeco is a medicine used for the treatment of cystic fibrosis in patients who are 2 years or older and who have one of the following mutations in their CF gene: G551D, G1244E, G1249D, G178R, G551S, S1251N, S1255P, S549N, or S549R.

Orkambi is for cystic fibrosis patients who have two copies of F508del (which is the most common mutation related to cystic fibrosis). In short, more cystic fibrosis patients will be able to use Orkambi than will be able to use Kalydeco. Orkambi is currently intended for use by cystic fibrosis patients who are at least 12 years old. That could change, since Vertex is testing the drug on children who have cystic fibrosis and are as young as 6 years old.

Cystic fibrosis is a life-threatening genetic disorder that causes severe damage to the lungs and digestive system. It is an inherited condition. In other words, the reason a person has cystic fibrosis is because he or she inherited certain mutations on their CF gene from their parents.

Cystic fibrosis affects the cells that produce mucus, sweat, and digestive juices. Those fluids are supposed to be thin and slippery. People who have cystic fibrosis end up with those fluids becoming thick and sticky. As a result, the fluids can plug up tubes, ducts, and passageways in the body (especially in the lungs and pancreas).

There is no cure for cystic fibrosis. Kalydeco, and now, Orkambi, are designed to treat the underlying causes of the disease (instead of focusing on the symptoms). If the Food and Drug Administration chooses to approve Orkambi, it could help about 2,000 of the 30,000 cystic fibrosis patients in the United States.

To be clear, an independent federal advisory to the Food and Drug Administration has approved Orkambi. The Food and Drug Administration itself is still making a decision on whether or not to approve it.

Image by Hey Paul Studios on Flickr.

Related Articles at FamilyTree.com:

* Blood Sample from Mom Reveals Genome of Baby

* Karyomapping Can Detect Genetic Conditions

* How to Find Out What’s In Your Genes

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