Orkambi Officially Receives FDA Approval

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Last month, PBIRx introduced our readers to a new drug known as Orkambi, that was expected to receive FDA approval in early July. Today, we are following up to share the news that the approval has officially gone through!

As explained by the FDA, this drug was designed to treat cystic fibrosis in individuals that have two copies of a specific mutation. The mutation, known as the F508del mutation, "causes the production of an abnormal protein that disrupts how water and chloride are transported in the body." As of now, this drug will solely be used to treat the disease in patients who are 12 years of age or older, who have two copies of the specific genetic mutation which the drug treats. That represents 28% of cystic fibrosis patients.

Upon hitting the market, Orkambi is expected to cost $259,000 per patient, according to the Wall Street Journal. It's manufacturer, Vertex Pharmaceuticals, anticipates that the drug will be available rather quickly.

Things To Remember:

• Orkambi is taken orally twice a day. 
• The drug is a combination of Kalydeco (ivacaftor) and lumacaftor.
• The effectiveness of Orkambi has not been proven in Cystic Fibrosis patients that do not have two copies the F508del mutation.
• Side effects of Orkambi include, but are not limited to, shortness of breath, nausea, rash and upper respiratory tract infections.

For more information about Orkambi, please click here.

Here at PBIRx, we realize that the approval of this new drug could affect many pharmacy benefit plans. That's why our team is working closely on analyzing its impact and developing solutions for each of our many clients.

To learn how your pharmacy benefit plan may be affected, please give us a call at 1 (888) Rx-PBIRx (797-2479).