- Precision medicine decision based on in vitro data and supported by
more than five years of real-world clinical data that demonstrate
KALYDECO's strong safety and efficacy profile for eligible patients -
- Vertex working with FDA to obtain rapid approval for more than 600
additional people who have mutations responsive to KALYDECO, including
one of five 'splice mutations -
Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that the
U.S. Food and Drug Administration (FDA) has approved KALYDECO
(ivacaftor) for use in people with cystic fibrosis (CF) ages 2 and older
who have one of 23 residual function mutations in the cystic fibrosis
transmembrane conductance regulator (CFTR) gene. This precision
medicine decision is based on analyses of in vitro data and is
supported by more than five years of real-world clinical data that
demonstrate KALYDECO's strong safety and efficacy profile. More than 900
people ages 2 and older in the U.S. have one of these mutations. Based
on this approval, Vertex today increased its guidance for 2017 product
revenues of KALYDECO to a range of $740 million to $770 million.
In addition to the mutations added to the label today, Vertex is
continuing discussions with the FDA concerning the approval for
additional people who have mutations responsive to KALYDECO, including
one of five 'splice mutations. These five mutations were evaluated as
part of the previously disclosed Phase
3 EXPAND study in which the KALYDECO monotherapy arm met its primary
efficacy endpoint while being generally well tolerated. More than 600
people ages 2 and older in the U.S. have one of these mutations.
Five years ago, KALYDECO became the first medicine to treat the
underlying cause of CF. Since then, we have continued to invest in
studies to improve the understanding of how this important medicine may
benefit others with this serious and life-shortening disease, said
Jeffrey Chodakewitz, M.D., Executive Vice President and Chief Medical
Officer at Vertex. We are encouraged by the FDA's willingness to
explore innovative ways to make highly effective medicines like KALYDECO
with a well-established safety profile available to more people who are
in urgent need. We will continue to work closely with the FDA to bring
KALYDECO to more people with responsive mutations who are still in need
as rapidly as possible.
CF treatment has advanced rapidly, but there is need for broader access
to these important medicines and development of additional medicines
remains urgent, said Patrick Flume, M.D., Director of the Medical
University of South Carolina Cystic Fibrosis Center. The use of in
vitro data to support this approval is an important step forward in
making medicines like KALYDECO available to more patients, especially
those with rare mutations.
Based on clinical data from numerous prior studies and a demonstrated in
vitro response employing a well-established, analytically validated
method, KALYDECO is now approved to treat people with CF ages 2 and
older who have one of 33 mutations in the CFTR gene.
CF is caused by defective or missing cystic fibrosis transmembrane
conductance regulator (CFTR) proteins resulting from mutations in theCFTRgene.
The defective or missing proteins result in poor flow of salt (chloride)
and water into and out of the cell in a number of organs, including the
lungs. Chloride transport is a marker of the function of the CFTR
protein at the cell surface. As with the other mutations for which
KALYDECO is approved, mutations covered under today's approval are known
to have some CFTR protein at the cell surface and have shown in vitro
increases in chloride transport in response to KALYDECO by at least 10
percent of normal over baseline. Similar to the R117H mutation
for which KALYDECO was previously approved, these additional residual
function mutations result in a moderate loss of CFTR chloride transport,
and people with these mutations generally have progressive lung function
decline and other complications of CF.
Pipeline Progress for Residual Function Mutations
On March 28, 2017, Vertex announced positive results from a Phase 3
study that evaluated tezacaftor/ivacaftor combination treatment in
people who have one mutation that results in residual CFTR function and
one F508del mutation. The study met its primary endpoint and the
combination was generally well tolerated. Vertex plans to submit these
data as part of a New Drug Application (NDA) to the U.S. Food and Drug
Administration (FDA) and a Marketing Authorization Application (MAA) to
the European Medicines Agency (EMA) for the tezacaftor/ivacaftor
combination early in the third quarter of 2017.
About Cystic Fibrosis and KALYDECO(ivacaftor)
Cystic fibrosis is a rare, life-threatening genetic disease affecting
approximately 75,000 people inNorth America,EuropeandAustralia.
CF is caused by a defective or missing CFTR protein resulting from
mutations in theCFTRgene. Children must inherit two
defectiveCFTRgenes one from each parent to have
CF. There are approximately 2,000 known mutations in theCFTRgene.
Some of these mutations, which can be determined by a genetic test, lead
to CF by creating defective or too few CFTR proteins at the cell
surface. The defective or missing CFTR protein results in poor flow of
salt and water into or out of the cell in a number of organs, including
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. The median predicted age
of survival for a person born today with CF is 41 years, but the median
age of death is 27 years.
KALYDECO (ivacaftor) is the first medicine to treat the underlying cause
of CF in people with specific mutations in the CFTR gene. Known
as a CFTR potentiator, KALYDECO is an oral medicine designed to keep
CFTR proteins at the cell surface open longer to improve the transport
of salt and water across the cell membrane, which helps hydrate and
clear mucus from the airways. In adults and pediatric patients age 6
years and older one 150 mg tablet is taken orally every 12 hours with
fat-containing food. Pediatric patients ages 2 to less than 6 years who
weigh less than 14 kg take one 50 mg packet of oral granules mixed with
1 teaspoon (5 mL) of soft food or liquid administered orally every 12
hours with fat-containing food. Pediatric patients ages 2 to less than 6
years of age who weigh 14 kg or greater take one 75 mg packet of oral
granules with 1 teaspoon (5 mL) of soft food or liquid administered
orally every 12 hours with fat-containing food.
People with CF who have specific mutations in the CFTR gene are
currently benefiting from KALYDECO in 27 different countries across
North America, Europe and Australia.
KALYDECO (ivacaftor) INDICATION AND
IMPORTANT SAFETY INFORMATION
KALYDECO (ivacaftor) is a prescription medicine used for the treatment
of cystic fibrosis (CF) in patients age 2 years and older who have at
least one mutation in the CF gene that is responsive to KALYDECO.
Patients should talk to their doctor to learn if they have an indicated
CF gene mutation. It is not known if KALYDECO is safe and effective in
children under 2 years of age.
Patients should not take KALYDECO if they are taking certain
medicines or herbal supplements such as: the antibiotics
rifampin or rifabutin; seizure medications such as phenobarbital,
carbamazepine, or phenytoin; or St. John's wort.
Before taking KALYDECO, patients should tell their doctor if they:
have liver or kidney problems; drink grapefruit juice, or eat grapefruit
or Seville oranges; are pregnant or plan to become pregnant because it
is not known if KALYDECO will harm an unborn baby; and are breastfeeding
or planning to breastfeed because is not known if KALYDECO passes into
KALYDECO may affect the way other medicines work, and other medicines
may affect how KALYDECO works. Therefore the dose of KALYDECO may
need to be adjusted when taken with certain medications. Patients should
especially tell their doctor if they take antifungal medications such as
ketoconazole, itraconazole, posaconazole, voriconazole, or fluconazole;
or antibiotics such as telithromycin, clarithromycin, or erythromycin.
KALYDECO can cause dizziness in some people who take it. Patients should
not drive a car, use machinery, or do anything that needs them to be
alert until they know how KALYDECO affects them. Patients should avoid
food containing grapefruit or Seville oranges while taking KALYDECO.
KALYDECO can cause serious side effects including:
High liver enzymes in the blood have been reported in patients
receiving KALYDECO. The patient's doctor will do blood tests to
check their liver before starting KALYDECO, every 3 months during the
first year of taking KALYDECO, and every year while taking KALYDECO. For
patients who have had high liver enzymes in the past, the doctor may do
blood tests to check the liver more often. Patients should call their
doctor right away if they have any of the following symptoms of liver
problems: pain or discomfort in the upper right stomach (abdominal)
area; yellowing of their skin or the white part of their eyes; loss of
appetite; nausea or vomiting; or dark, amber-colored urine.
Abnormality of the eye lens (cataract) has been noted in some children
and adolescents receiving KALYDECO. The patient's doctor should perform
eye examinations prior to and during treatment with KALYDECO to look for
cataracts. The most common side effects include headache; upper
respiratory tract infection (common cold), which includes sore throat,
nasal or sinus congestion, and runny nose; stomach (abdominal) pain;
diarrhea; rash; nausea; and dizziness.
These are not all the possible side effects of KALYDECO.
here to see the full Prescribing Information for KALYDECO.
Collaborative History with Cystic Fibrosis Foundation Therapeutics,
Vertex initiated its CF research program in 2000 as part of a
collaboration with CFFT, the nonprofit drug discovery and development
affiliate of the Cystic Fibrosis Foundation. KALYDECO (ivacaftor) was
discovered by Vertex as part of this collaboration.
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 inCambridge, Mass., Vertex today has research and
development sites and commercial offices inthe United
States,Europe,Canadaand Australia. For seven years in a row,Sciencemagazine
has named Vertex one of its Top Employers in the life sciences. For
additional information and the latest updates from the company, please
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, the statements in the third and fourth paragraphs of the
press release and statements regarding (i) the potential approval of
KALYDECO for patients with 'splice mutations, (ii) Vertex's updated
guidance for KALYDECO 2017 net product revenues and (iii) Vertex's plans
to submit an NDA and MAA for tezacaftor/ivacaftor. While Vertex believes
the forward-looking statements contained in this press release are
accurate, these forward-looking statements represent the company's
beliefs only as of the date of this press release and 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 the
company's expectations regarding its 2017 KALYDECO net product revenues
may be incorrect (including because one or more of the company's
assumptions underlying its expectations may not be realized), that data
from the company's development programs may not support registration or
further development of its compounds due to safety, efficacy or other
reasons, and 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.
View source version on businesswire.com: http://www.businesswire.com/news/home/20170517006271/en/
Vertex Pharmaceuticals Incorporated
Eric Rojas, 617-961-7205
David Whitrap, +1 617-961-5093
Megan Goulart, +44 20 3204 5275
Source: Vertex Pharmaceuticals Incorporated