One of the biggest challenges of treating cystic fibrosis is the origin of the disease. The disorder is caused by a genetic abnormality that results in a thick buildup of mucus in the lungs, but that defect varies across the entirety of the CF patient population. A drug that addresses one segment of the patient population won’t work on another segment of CF patients, which has been a challenge for the biotechnology industry to overcome.
Biotech company Vertex recently scored Food and Drug Administration approval on a new drug designed to cover a broader spectrum of CF patients. The new Vertex drug, called Orkambi, is actually a combination of two of the company’s other drugs. The pill combines the active pharmaceutical ingredient of the previously approved Vertex drug Kalydeco with a second Vertex compound, called lumacaftor.
Kalydeco had been approved to treat patients who had a particular genetic defect that would respond to that treatment; lumacaftor targets a different genetic defect, Xconomy explains. In combining the two drugs in a pill that delivers a one, two punch, Vertex says it can address approximately half of the 70,000 cystic fibrosis patients worldwide. The FDA approved Orkambi for use in patients 12 years old and older, though Vertex expects to eventually expand the drug’s label to cover younger children, as well.
Orkambi won’t come cheap. Vertex is pricing the combination drug at $259,000 a year. That’s less than the price for Kalydeco, which costs $312,000 per year. TheStreet notes that Orkambi’s net price could end up being even lower as rebates and negotiated insurance discounts are factored in. But TheStreet notes that the launch of the drug will be watched closely to see if insurance companies and pharmacy benefit managers push back on the cost of the drug. To learn more about advances in treating rare diseases, such as CF, contact us.