Kalydeco Approved to Treat Rare Form of Cystic Fibrosis

TUESDAY, Jan. 31 (HealthDay News) — Kalydeco (ivacaftor) has been approved by the U.S. Food and Drug Administration to treat the root cause of a rare form of the inherited disease cystic fibrosis.

CF causes a deadly buildup of thick mucus in the lungs and other organs. Kalydeco was sanctioned to treat people 6 and older whose CF is caused by a mutation of the G551D gene, which oversees the distribution of water throughout the body, the FDA said in a news release.

CF affects about 30,000 people in the United States, and is the most common fatal genetic disease among whites, the agency said. Some 4 percent of cases, or approximately 1200 people, are thought to have the G551D mutation.

Kalydeco was given priority six-month review and was given orphan drug status, since the targeted form of CF affects fewer than 200,000 people in the United States, the FDA said.

The twice-daily pill was approved based on clinical studies involving 213 people with the G551D mutation. The most common side effects included upper respiratory tract infection, headache, stomach ache, diarrhea and dizziness.

Kalydeco is not effective in people with the most common genetic cause of CF, the FDA stressed. The drug is produced by Vertex Pharmaceuticals, based in Cambridge, Mass.

More information

To learn more about cystic fibrosis, visit Medline Plus.