Vertex Receives CHMP Positive Opinions for ORKAMBI™ (lumacaftor/ivacaftor) and KALYDECO® (ivacaftor) in the European Union
September 25 2015 - 7:17AM
Business Wire
-CHMP recommends Marketing Authorization for
lumacaftor in combination with ivacaftor for people with cystic
fibrosis ages 12 and older with two copies of the F508del
mutation-
-CHMP recommends Marketing Authorization for
ivacaftor for children with cystic fibrosis ages 2 to 5 with 9
gating mutations & for people with cystic fibrosis ages 18 and
older with the R117H mutation-
Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today
announced that the European Union Committee for Medicinal Products
for Human Use (CHMP) issued a positive Opinion recommending
Marketing Authorization of ORKAMBI™ (lumacaftor/ivacaftor), the
first medicine to treat the underlying cause of cystic fibrosis
(CF) in people ages 12 and older with two copies of the F508del
mutation. In Europe, approximately 12,000 people with CF ages 12
and older have two copies of this mutation.
The CHMP also issued a positive Opinion recommending the
indication of KALYDECO® (ivacaftor) be expanded to include children
ages 2 to 5 with CF, in addition to the current approved use in
people ages 6 and older, who have one of nine gating mutations in
the cystic fibrosis transmembrane conductance regulator (CFTR) gene
(G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N and
S549R). A new weight-based oral granule formulation of ivacaftor
(50 mg and 75 mg) that is mixed in soft foods or liquids was
created for these younger children. The CHMP Opinion for the use of
ivacaftor in children ages 2 to 5 included a recommendation to
extend weight-based dosing of ivacaftor to children ages 6 to 11
who weigh less than 25 kg using the new oral granule formulation.
In addition, the CHMP issued a positive Opinion recommending
ivacaftor for use in people with CF ages 18 and older who have the
R117H mutation. In Europe, approximately 125 children ages 2 to 5
have one of the nine gating mutations included in today’s positive
Opinion, and approximately 250 adults have the R117H mutation.
“Today’s recommendations bring us closer to being able to help
many more people with cystic fibrosis who currently do not have a
medicine to treat the underlying cause of this chronic progressive
disease,” said Jeffrey Chodakewitz, M.D., Executive Vice President
and Chief Medical Officer at Vertex. “However, we know that there
are many more people with CF who are still waiting for treatment,
and we remain committed to continuing to work toward our goal of
creating new medicines for the vast majority of people with cystic
fibrosis.”
These positive CHMP Opinions are recommendations for approval to
the European Commission, which then has the authority to approve
medicines for the European Union. The European Commission generally
follows the recommendations of the CHMP and typically issues
Marketing Authorization within three months. If the CHMP’s
recommendations are approved, Vertex would then begin the
country-by-country reimbursement approval process for each new
medicine and indication.
About Cystic Fibrosis
Cystic fibrosis is a rare, life-threatening genetic disease
affecting approximately 75,000 people in North
America, Europe and Australia.
CF is caused by a defective or missing CFTR protein resulting
from mutations in the CFTR gene. Children must inherit
two defective CFTR genes — one from each parent — to have
CF. There are approximately 2,000 known mutations in
the CFTR gene. Some of these mutations, which can be
determined by a genetic, or genotyping test, lead to CF by creating
non-working or too few CFTR protein at the cell surface. The
defective function or absence of CFTR proteins in people with CF
results in poor flow of salt and water into and out of the cell in
a number of organs. In the lungs, this leads to the buildup of
abnormally thick, sticky mucus that can cause chronic lung
infections and progressive lung damage in many patients that
eventually leads to death. Today, the median predicted age of
survival for a person with CF is between 34 and 47 years, but the
median age of death remains in the mid-20s.
Collaborative History with Cystic Fibrosis Foundation
Therapeutics, Inc. (CFFT)
Vertex initiated its CF research program in 1998 as part of a
collaboration with CFFT, the nonprofit drug discovery and
development affiliate of the Cystic Fibrosis Foundation.
Lumacaftor and ivacaftor were discovered by Vertex as part of this
collaboration.
About Vertex
Vertex is a global biotechnology company that aims to discover,
develop and commercialize innovative medicines so people with
serious diseases can lead better lives. In addition to our clinical
development programs focused on cystic fibrosis, Vertex has more
than a dozen ongoing research programs aimed at other serious and
life-threatening diseases.
Founded in 1989 in Cambridge, Mass., Vertex today has
research and development sites and commercial offices in the
United States, Europe, Canada and Australia. For
five years in a row, Science magazine has named Vertex
one of its Top Employers in the life sciences. For additional
information and the latest updates from the company, please
visit www.vrtx.com.
Special Note Regarding Forward-looking Statements
This press release contains forward-looking statements as
defined in the Private Securities Litigation Reform Act of 1995,
including, without limitation, statements regarding the European
Commission generally following the recommendations of the CHMP and
typically issuing marketing approval within three months. While
Vertex believes the forward-looking statements contained in this
press release are accurate, there are a number of factors that
could cause actual events or results to differ materially from
those indicated by such forward-looking statements. Those risks and
uncertainties include, among other things, that Vertex could
experience unforeseen delays in obtaining marketing approval from
the European Commission and the other risks listed under Risk
Factors in Vertex's annual report and quarterly reports filed with
the Securities and Exchange Commission and available through the
company's website at www.vrtx.com. Vertex disclaims any obligation
to update the information contained in this press release as new
information becomes available.
(VRTX-GEN)
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Vertex Pharmaceuticals IncorporatedInvestors:Michael Partridge,
+1 617-341-6108orKelly Lewis, +1 617-961-7530orEric Rojas, +1
617-961-7205orMedia:mediainfo@vrtx.comorEurope & Australia: +44
20 3204 5275orNorth America: +1 617-341-6992
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