The company, which on Thursday won approval for a second drug to treat the underlying cause of cystic fibrosis, hopes to have treatments for roughly 90 percent of patients with the disease by around 2020. The safety and effectiveness of Orkambi was assessed in two double-blind, placebo-controlled clinical trials of 1,108 participants with CF who were 12 years and older with the F508del mutation.
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TheStreet.com, a business news and analysis site, reported earlier this week that sales of the drug “are also expected to transform Vertex into a sustainably profitable company for the first time since its founding in 1989″.
The new drug, to be sold as Orkambi (or-KAM-bee), is Vertex’s follow-up to its breakthrough pill Kalydeco (kuh-LYE-deh-koh), which became the first drug to treat the underlying cause of cystic fibrosis in 2012.
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. Beall, Ph.D., President and CEO of the Cystic Fibrosis Foundation. It can be a fatal disease that causes patients’ lungs and other organs to clog with thick, sticky mucus.
The increased number of patients eligible to take Orkambi and the lower efficacy are likely the reason tat Vertex priced Orkambi at $259,000 per year, a sizable discount to Kalydeco. Vertex has downplayed the ability to match the remarkable Kalyedco launch (80% 1yr) because of a larger population, but we believe the combination of a knowledgeable patient group and strong advocacy of the CF Foundation will lead to a comparatively steep penetration curve.
Shares, temporarily halted during the regular trading session Thursday, rose 4% to $131.26 over Wednesday’s closing price.
In May its approval looked more doubtful after a review questioned whether the combination was actually more effective than ivacaftor alone – but the FDA panel backed the drug a few days later. The company, which relocated from Cambridge, Massachusetts to a new $800 million campus in Boston past year, has a market value of over $30 billion. At the FDA advisory committee meeting held in May, some experts criticized Orkambi for offering only modest improvement in lung function.
“We applaud the FDA for its swift approval of Orkambi”, notes Robert J.
Orkambi combines lumacaftor and ivacaftor to treat these problems with a two-step approach.
Orkambi has also been submitted for approval in Europe, and a decision by the EMA is expected before the end of the year.
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In the 1950s, children with cystic fibrosis seldom survived long enough to complete elementary school.
