Orkambi: New Treatment for Cystic Fibrosis Now Available

Roni Shye
Roni Shye, PharmD BCGP BCACP, is a licensed pharmacist in the states of Florida, Ohio, and Pennsylvania.
Posted on

Though there are other treatments available to help manage symptoms, cystic fibrosis does not currently have a cure. That’s part of what makes Orkambi (ivacaftor/lumacaftor) exciting—this new medication targets the two defective genes that are the underlying cause of the disease.

Now, after receiving FDA approval on July 2, 2015, Orkambi is available in pharmacies.

What is cystic fibrosis?

Cystic fibrosis (CF) is an inherited and life-threatening disease that affects the respiratory and digestive systems. It mainly affects the lungs, but can also involve the pancreas, liver, intestine, sinuses, and sex organs.

Symptoms can include coughing with thick mucus or blood, lung infections, and diarrhea, though these vary from person to person.

What causes cystic fibrosis?

Cystic fibrosis is caused by a mutation of the CFTR gene.

A child will develop cystic fibrosis if they inherit two copies of a mutated gene, one from each parent (neither of whom is likely to have the disease themselves).

How does the gene mutation affect someone with CF?

The mutation causes interference with the transportation of water and chloride in the body. That interference leads to the buildup of thick, sticky mucus that can damage many of the body’s important organs.

Who is Orkambi for?
Orkambi is used for the treatment of cystic fibrosis in patients 12 years of age and older, who have two copies of the F508del mutation in their CFTR gene.

What is unique about Orkambi?
Orkambi is the first medication for cystic fibrosis that is meant to treat the underlying cause of the disease, rather than just the symptoms.

How is Orkambi taken?

The recommended dosage of Orkambi is 2 tablets by mouth every 12 hours. Orkambi is a combination medication, with each tablet containing lumacaftor 200mg and ivacaftor 125mg.

What are the most common side effects associated with Orkambi?

Some common side effects include shortness of breath, upper respiratory symptoms, nausea, diarrhea, rash, and increased menstrual bleeding.

Will Orkambi be available at my pharmacy?

No. Orkambi will be available exclusively at specialty pharmacies—you won’t be able to go to your regular retail pharmacy to fill a prescription. You can see a list of the specialty pharmacies that will offer Orkambi here and here.

More worrisome? Orkambi comes with a very high price tag, at an estimated $259,000 per year. Vertex, Orkambi’s manufacturer, does offer support for getting coverage, and may offer assistance for patients in need. See their website for more information.

Want more information on Orkambi?

See the announcement from the FDA regarding Orkambi here.

Drugs featured in this story

Filed under