The drug, a twice-daily oral tablet for patients 12 years and older, expands the company’s reach in the treatment of cystic fibrosis, the progressive lung disease that often kills patients in their 20s. Vertex said the new drug would be available within days. Orkambi is aimed at treating patients with a genetic mutation that is the leading cause of cystic fibrosis.

Boston-based Vertex already sells Kalydeco, which treats a different genetic type of cystic fibrosis and has an annual price of $311,000 per patient in the U.S. Orkambi is expected to reach a broader patient population than Kalydeco. The company estimated the new drug might treat as many as 12,000 patients in Europe, with England and Germany being the largest potential markets.

On a conference call Thursday with Wall Street analysts, company officials estimated that about 35% to 40% of the patients who are candidates for Orkambi will be on Medicaid, the government program for lower-income people.

The drug is the latest to carry a potentially budget-busting price tag. Others are for cancer and hepatitis C. Kalydeco’s high price has put pressure on government insurance programs, such as in Arkansas.

The 8,500-patient population in the U.S. 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.


Write to Maria Armental at [email protected] and Thomas M. Burton at [email protected]

Source: Wall Street Journal Health