Kalydeco approval
February 8, 2012
THE US FDA has approved Kalydeco
(ivacaftor) for the treatment of a
rare form of cystic fibrosis in
patients aged 6 years and older
who have the specific G551D
mutation in the cystic fibrosis
Transmembrane Regulator gene.
Cystic fibrosis affects around
30,000 people in the US, and is the
most common fatal genetic
disease in the Caucasian population.
In patients with the G551D
mutation, Kalydeco, a pill taken
two times a day with fat-containing
food, helps the protein made by
the CFTR gene function better and
as a result, improves lung function
and other aspects of CF such as
increasing weight gain.
MEANWHILE the FDA has also
granted Gleevec (imatinib) regular
approval for use in adult patients
following surgical removal of
CD117-positive gastrointestinal
stromal tumors (GIST).
GIST is a rare form of cancer that
originates in cells found in the wall
of the GI tract.
“The development of Gleevec
over the past decade highlights the
need to further study drugs after
approval to truly characterise their
benefits,” said Dr Richard Pazdur,
Director of the Office of Hematology
and Oncology Products in the FDA’s
Center for Drug Evaluation and
Research.
“Although originally approved in
the metastatic disease setting, this
subsequent trial has demonstrated
that longer use of Gleevec can
prolong patient’s lives in earlier
disease settings,” he added.
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