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.
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.
The top 5 reasons to watch lumacaftor/ivacaftor are:
Orkambi will be available for shipment to specialty pharmacies in the United States this month.
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.
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.