FDA одобрява Kalydeco (VX-770) - първото лекарство, което е насочено към основната причина за муковисцидоза
January 31, 2012 31 януари, 2012 г.
Кистозна фиброза фондация днес
приветства одобрението на храните и лекарствата на Kalydeco ™
(ivacaftor-рано известен като VX-770), огромен напредък в търсенето на
лек за кистозна фиброза.
Медикаментът е разработен от Vertex
Pharmaceuticals Inc., С подкрепа на научни, клинични и значително
финансиране от кистозна фиброза фондация.
FDA одобрени Kalydeco
(kuh-луга-DEH-КОН) сегмент от населението CF, тези на възраст между 6 и
повече години с G551D мутация на кистозна фиброза. Лекарството се приема
в таблетна форма.
"Днес завършва важен етап в нашето пътешествие
към намиране на лек за кистозна фиброза," каза Робърт Дж. Beall, доктор
на науките, президент и главен изпълнителен директор на кистозна
фиброза фондация. "Kalydeco адреси основната причина за CF и науката зад
наркотици откри вълнуващи нови врати за научни изследвания и развитие,
което в крайна сметка може да доведе до допълнителни терапии, които ще
се възползват в по-голяма степен хората, живеещи с CF."
Скоро ще качим и преведена информацията.
http://www.cff.org/aboutCFFoundation/NewsEvents/1-31-FDA-Approves-Kalydeco.cfm
FDA Approves Ivacaftor For Cystic Fibrosis.
The CBS Evening News
(1/31, story 7, 0:30, Pelley) reported, "There is a break through
tonight in the treatment of cystic fibrosis. The FDA today approved a
drug called Kalydeco [ivacaftor]," which is "the first drug that treats
the genetic defect in CF, not just the symptoms."
The New York Times
(2/1, B2, Pollack, Subscription Publication) reports the medication's
approval came "more than 22 years after the gene responsible for the
disease was first identified." The treatment, "developed by Vertex
Pharmaceuticals, counters the effect of one specific mutation in the
gene that accounts for 4 percent - or about 1,200 - cystic fibrosis
cases in the United States. The drug is approved for patients age 6 and
older with the G551D mutation."
"The drug...helps a
defective protein that acts as a chloride-pump in cell membranes to
function properly, restoring the proper flow of water in the body," the
Los Angeles Times
(2/1, Khan) "Booster Shots" blog reports. "The FDA fast-tracked
Kalydeco and approved it in three months, given the drug's early signs
of promise." FDA Commissioner Dr. Margaret A. Hamburg, said in a news
release, "Kalydeco is an excellent example of the promise of
personalized medicine -- targeted drugs that treat patients with a
specific genetic makeup."
"Cystic fibrosis is the most-common fatal genetic disease in the Caucasian population, the FDA said," Bloomberg News
(2/1, Edney) adds. "The twice-daily" capsule "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." Nancy Wysenski,
executive vice president at Vertex, says that "about 200 of the 1,200
cystic fibrosis patients with the mutation are younger than 6" so it
"plans to study the drug in the younger patients beginning the middle of
this year."
The Boston Globe
(2/1, Kotz) reports, "The agency based its expedited approval -- which
took just three months -- on two clinical studies involving 213
patients aged 11 and over that lasted nearly a year." According to the
company, "in both studies, those treated with Kalydeco had improved
lung function, increased weight gain, and fewer acute problems that
often require hospital visits and treatment with antibiotics, compared
with those who took a placebo. The most common side effects of Kalydeco
include upper respiratory tract infection, headache, stomach ache,
rash, diarrhea, and dizziness."
The Boston Business Journal
(2/1, Donnelly, Subscription Publication) points out that "the drug was
discovered as part of a collaboration between the Cambridge,
Mass.-based biotechnology company and the Cystic Fibrosis Foundation
Therapeutics, Inc., the nonprofit drug discovery and development
affiliate of the Cystic Fibrosis Foundation." The company "said it would
start shipping Kalydeco to pharmacies this week."
The AP
(2/1, Perrone) reports that company executives say the treatment "would
cost $294,000 for a year's supply, placing it among the most expensive
prescription drugs sold in the US."
"Vertex said it would
provide the drug free of charge to patients with household income of
$150,000 or less who do not have insurance," MedPage Today
(2/1, Gever) reports. "For those with insurance, the company said,
'there will be a minimal out-of-pocket obligation after which Vertex
will help cover copay or coinsurance costs up to 30% of the list price
of the medicine. There is no income limit to be eligible for this
program.'" Also covering the story are Reuters (2/1, Yukhananov, Berkrot), the Wall Street Journal (2/1, Rockoff, Dooren, Subscription Publication), WebMD (2/1, DeNoon), and HealthDay (2/1, Preidt). |
“РЕДКИ, НО СИЛНИ ЗАЕДНО” е мотото, под което тази година ще премине честването на Международния ден на редките болести – 29 февруари 2012 г.
