It causes the formation of sticky mucus in the lungs and other organs of the patient, leading to digestive problems, infections and early death.
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Vertex Pharmaceuticals has bagged its first regulatory approval for Orkambi, a combination therapy for the genetic disease cystic fibrosis. Even if you want to argue that efficacy should be closer to what was seen in the short Orkambi phase 2 trial (809-102), it’s still not as impressive as what Kalydeco can do in the patient populations the drug can treat. The Orkambi drug will be administered to patients who are at least 12 years old. According to a report from the Boston Globe, the drug, called Orkambi, would be priced at $259,000 per patient annually.
The Food and Drug Administration cleared the twice-a-day pill from Vertex Pharmaceuticals Inc. for a variation of cystic fibrosis that affects about 8,500 people in the USA who are 12 years and older. This F508del mutation, which is inherited from the parent of the patient, is the common cause of CF. Cystic Fibrosis has affected about 30, 000 people in America alone. In U.S, a number of 30,000 people are diagnosed with cystic fibrosis and approximately 70,000 are writhe with the deadly disease.
Given that the average survival age for CF patients is around 37, Orkambi was given breakthrough status by the FDA in 2012.
Robert Beall, president and CEO of the Cystic Fibrosis Foundation, says: “We applaud the FDA for its swift approval of Orkambi”.
Two pivotal trials that form the basis of Vertex’ marketing application for Orkambi in the U.S. showed that the drug was able to improve lung function by 2.6%-4.0% over 23 weeks, and cut the rate of pulmonary infections, hospitalisations and antibiotic use.
“Today’s approval significantly broadens the availability of targeted treatments for the specific defects that cause cystic fibrosis”, Dr. John Jenkins, director of the Office of New Drugs at the FDA’s Center for Drug Evaluation and Research, said in an agency news release.
Shares of Vertex rose $5.07, or 4 percent, to close at $131.26 in trading Thursday.
But the company’s cystic fibrosis medicines have also drawn criticism for their high costs as a national debate about pricey prescription drugs intensifies.
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Vertex describes its drug Lumacaftor, one of the components of Orkambi, as helping to position a kind of door that regulates the amount of fluid on the surface of cells in the lungs and digestive system of patients with the defective protein. Kalydeco cost $294,000 a year when it came to market in 2012 and last year had sales of $464 million.
