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.

NEXT: Price difference between Orkambi and Kalydeco

The 8,500-patient population in the United States would be about 28% of the 30,000 people in the U.S. with the disease. Janney Capital Markets, in a research note earlier this week, had predicted that Orkambi’s price would likely be less than that of Kalydeco because, it said, it is a less effective therapy that serves a larger patient population.

In clinical studies, the drug showed improved lung function in patients treated with the drug compared with those who received a placebo.

Related: New cystic fibrosis drugs may signficantly increase pharmacy benefit costs

Orkambi is a combination of Kalydeco, generically call ivacaftor, with another compound known as lumacaftor.

A decision on Vertex’s European application is expected in the fourth quarter. J.P. Morgan Chase estimated global drug sales could reach $1.6 billion next year and $4.2 billion by 2020.

Shares, temporarily halted during the regular trading session Thursday, rose 4% to $131.26 over Wednesday’s closing price.

An FDA advisory committee of 13 independent doctors, scientists and consumer and patient representatives recommended approval in May. The vote was 12 to 1, but several doctors on the panel said they regarded the drug as having modest effectiveness.

Some also noted that some patients actually experienced worsened lung function while taking the drug. FDA officials at the advisory committee meeting questioned whether Orkambi was superior to Kalydeco alone.

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