Vertex Pharmaceuticals Inc. (VRTX) won approval of the first drug to treat the underlying cause of cystic fibrosis.
The Food and Drug Administration cleared Kalydeco for a rare form of cystic fibrosis in patients ages 6 and older who have a specific gene mutation, the agency said today in a statement. Cystic fibrosis is the most-common fatal genetic disease in the Caucasian population, the FDA said.
Cystic fibrosis, which creates a thick mucus in the lungs and digestive tract, affects about 30,000 in the U.S. and about 4 percent, or 1,200, have the mutation, the agency said. The FDA approved the drug three months ahead of the expected deadline.
“Kalydeco is an excellent example of the promise of personalized medicine -- targeted drugs that treat patients with a specific genetic makeup,” FDA Commissioner Margaret Hamburg said in the statement.
Vertex rose 6.4 percent to $36.95 at 4 p.m. New York time.
The twice-daily pill also is designated as an orphan drug because it treats few people and Cambridge, Massachusetts-based Vertex will be able to sell the medicine seven additional years without competition from generics.
Kalydeco will cost $294,000 a year per patient, Nancy Wysenski, executive vice president and chief commercial officer at Vertex, said on a conference call today with analysts and media.
She said about 200 of the 1,200 cystic fibrosis patients with the mutation are younger than 6. The company plans to study the drug in the younger patients beginning the middle of this year. Vertex also is studying Kalydeco in combination with another drug candidate for a more common cystic fibrosis mutation and expects initial data in the middle of this year.
Cystic fibrosis is life-threatening and has no cure, according to the National Institutes of Health. Kalydeco thins the mucus to keep airways from getting clogged and infected. Genetic testing should be done before the medicine is taken.
Francis Collins, now director of the National Institutes of Health, and a team of researchers, discovered the gene involved with cystic fibrosis in 1989, Janet Woodcock, director of the FDA’s Center for Drug Evaluation and Research, said in a blog post. Vertex worked 13 years to target the underlying cause of the disease, Jeffrey Leiden, Vertex’s incoming president and chief executive officer said in a statement.
Vertex joined with the Cystic Fibrosis Foundation to develop the drug through “venture philanthropy,” Woodcock said.
The foundation put up $75 million for the research, according to a statement from the patient group. It will receive tiered royalties the company didn’t disclose.
The most-common side effects of Kalydeco include upper respiratory tract infection, headache, stomach ache, rash, diarrhea and dizziness, the FDA said.
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