FDA OKs Drug That Targets Rare Form of Cystic
TUESDAY, Jan. 31 (HealthDay News) -- Kalydeco, the first drug
that targets the defective protein behind a rare form of the deadly
lung disorder cystic fibrosis, was approved Tuesday by the U.S.
Food and Drug Administration.
One patient advocate group applauded the decision.
"Today marks an important milestone in our journey to find a cure for cystic fibrosis," Robert Beall, president and CEO of the Cystic Fibrosis Foundation, said in a news release. "Kalydeco addresses the underlying cause of cystic fibrosis, and the science behind the drug has opened exciting new doors to research and development that may eventually lead to additional therapies that will benefit more people."
People with cystic fibrosis have mutations in a gene that
produces a protein called CFTR, which regulates the movement of
ions (such as chloride) and water in the body. This defect causes
sticky mucus to accumulate in the lungs and other organs, resulting
in infections, digestive problems and usually death in early
Cystic fibrosis affects an estimated 30,000 people in the United
States and is the most common fatal genetic disease in the white
population. About 1,200 (4 percent) of cystic fibrosis patients
have a rare form of the disease caused by a specific G551D mutation
in the cystic fibrosis transmembrane regulator (CFTR) gene, the FDA
explained in a news release.
The twice-a-day pill Kalydeco (ivacaftor) was approved for
patients with this rare form of cystic fibrosis. The pill, which is
taken with fat-containing food, helps the protein made by the CFTR
gene function better, the FDA explained.
Two 48-week clinical trials involving 213 patients -- one with
patients ages 12 and older and the other with patients ages 6 to 11
-- showed that the drug improved lung function and reduced other
symptoms of cystic fibrosis.
The most common side effects of the drug were upper respiratory
tract infection, headache, stomach ache, rash, diarrhea and
dizziness, said the FDA, which approved the drug under its priority
The drug will come with a hefty price tag, however: Kalydeco,
which is made by Massachusetts-based Vertex Pharmaceuticals Inc.,
costs almost $300,000 a year.
"Kalydeco is the first available treatment that targets the defective CFTR protein, which is the underlying cause of cystic fibrosis," Dr. Janet Woodcock, director of the FDA's Center for Drug Evaluation and Research, said in the news release. "This is a breakthrough therapy for the cystic fibrosis community because current therapies only treat the symptoms of this genetic disease."
Kalydeco is not effective in cystic fibrosis patients with two
copies of the F508 mutation in the CFTR gene, which is the most
common mutation that causes cystic fibrosis, the FDA stressed. If a
patient's mutation isn't known, an FDA-approved cystic fibrosis
mutation test should be used, the agency said.
The Cystic Fibrosis Foundation said there is an ongoing phase 2
trial for people with the more common CFTR gene mutation, using
Kalydeco alongside a second experimental drug, VX-809. Findings
from the first part of this trial have been encouraging, with the
second part still underway.
The U.S. National Heart, Lung, and Blood Institute has more
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