5 reasons to watch the new cystic fibrosis drug

FDA’s approval last week of Vertex Pharmaceutical’s groundbreaking drug lumacaftor/ivacaftor (Orkambi) for cystic fibrosis is certainly good news, but formulary managers need to be aware of the high cost of the drug and other factors. Orkambi is the first medicine to treat the underlying cause of cystic fibrosis (CF) in people aged 12 years and older with 2 copies of the F508del mutation.

Related: FDA approves Orkambi for treatment of cystic fibrosis

The top 5 reasons to watch lumacaftor/ivacaftor are:

• While the medication can treat as many as 8,500 patients in the United States, the cost is an estimated $259,000 per patient annually. That might not fly with legislators and PBMs, which are already protesting the high cost of medications such as Harvoni for hepatitis C.

• Between 35% and 40%of the candidates for Orkambi will be on Medicaid, according to a Vertex conference call with Wall Street analysts.

• While an FDA advisory committee recommended that Orkambi be approved, several physicians on the committee said the drug had modest effectiveness, according to The Wall Street Journal. In addition, some patients experienced worsened lung function while taking the drug.

• Vertex already has success with Kalydeco, which treats a different genetic type of cystic fibrosis, but carries an annual price tag of $311,000 per patient. Orkambi will reach a broader patient population, but FDA officials at the advisory committee meeting questioned whether Orkambi was superior to Kalydeco alone, according to The Wall Street Journal.

• Orkambi will likely only treat 28% of the 30,000 people in the United States with the disease, but may treat as many as 12,000 patients in Europe. England and Germany are the largest potential markets.

Orkambi will be available for shipment to specialty pharmacies in the United States this month.

 

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