Kalydeco Approved to Treat Rare Form of Cystic
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
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
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.
To learn more about
cystic fibrosis, visit Medline Plus.
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