Vertex Pharmaceuticals Incorporated VRTX today announced the
submission of a supplemental New Drug Application (sNDA) to the U.S. Food and
Drug Administration (FDA) for the approval of KALYDECO^TM (ivacaftor)
monotherapy for people with cystic fibrosis (CF) ages 6 and older who have at
least one non-G551D gating mutation in the cystic fibrosis transmembrane
conductance regulator (CFTR) gene. KALYDECO is currently approved for people
with CF ages 6 and older who have at least one copy of the G551D mutation. CF
is caused by a defective CFTR protein that results from mutations in the CFTR
gene. G551D is known as a gating mutation, and there are an additional nine
known gating mutations. Gating mutations prevent the CFTR protein from opening
or working (gating) properly at the cell surface. Worldwide, approximately
2,000 people with CF ages 6 and older have at least one copy of the G551D
mutation, and approximately 400 people with CF ages 6 and older have at least
one non-G551D gating mutation.
"Today's submission to the FDA is an important step toward our goal of helping
more people with CF," said Robert Kauffman, M.D., Ph.D., Senior Vice President
and Chief Medical Officer at Vertex. “The study in people with non-G551D
gating mutations is the first of multiple ongoing efforts to expand the number
of people who may benefit from ivacaftor, and we expect additional data from
these ongoing studies beginning later this year.”
The sNDA submission is based on previously announced data from a Phase 3 study
of ivacaftor monotherapy that showed statistically significant improvements in
lung function (FEV[1]). The mean absolute treatment difference in percent
predicted FEV[1] between treatment with ivacaftor and placebo was 10.7% (p <
0.0001) and the mean relative treatment difference in percent predicted
FEV[1] was 14.2% (p < 0.0001) through the 8-week treatment period. The safety
and tolerability results observed in this study were consistent with those
observed in prior Phase 3 studies of ivacaftor monotherapy in people with CF
who have the G551D mutation. The study in gating mutations is one of three
ongoing Phase 3 label-expansion studies designed to evaluate whether
additional people with CF may benefit from treatment with ivacaftor alone.
In addition to the sNDA submission, Vertex intends to submit a Marketing
Authorization Application (MAA) variation in Europe in October 2013 for people
with CF ages 6 and older who have at least one non-G551D gating mutation.
As part of the sNDA package, Vertex also submitted long-term data from the
96-week PERSIST open-label study of KALYDECO. PERSIST is an ongoing rollover
study of people with cystic fibrosis ages 6 and older with a G551D mutation
who took part in the 48-week Phase 3 STRIVE and ENVISON studies of KALYDECO.
The data from PERSIST submitted as part of the sNDA included efficacy and
safety results through 144 weeks of continuous treatment with KALYDECO.
The non-G551D gating data and the long-term PERSIST data will be presented at
the 27th Annual North American Cystic Fibrosis Conference (NACFC) in Salt Lake
City, Utah, October 17-19, 2013. Vertex expects the gating data to be
presented as an oral presentation during Symposium III, "CFTR: Matching CFTR
Mutations and Drugs," on October 19.
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