EXTON, Pa., Nov. 12, 2013 /PRNewswire/ -- ViroPharma
Incorporated (Nasdaq: VPHM), an international biopharmaceutical
company committed to developing and commercializing innovative
products that address unmet medical needs and rare diseases, today
announced results of a new quality of life data analysis from the
randomized, placebo-controlled pivotal prophylaxis study of
Cinryze® (C1 esterase inhibitor [human]), the first and only C1
esterase inhibitor therapy approved for routine prevention of
angioedema attacks in patients with HAE. The release of these data
marks the first prospective evaluation of the impact of routine
preventative treatment on the quality of life of patients with HAE.
Quality of life data were specified in the protocol to be collected
at the pre-treatment baseline and at the end of each of the two
treatment periods but is not included in the prescribing
information for Cinryze.
These data were presented in a poster entitled, Quality of
Life in Patients with Hereditary Angioedema Receiving Nanofiltered
C1 Inhibitor for Prophylaxis: Results of a Randomized,
Placebo-Controlled, Crossover Study, by William Lumry, MD, FAAAI, FACAAI, et al.
While the burden of HAE and the impact on patients'
health-related quality of life (HRQoL) has been previously
described, the effect of routine prophylaxis on quality of life has
not been evaluated. The objective of this analysis was to evaluate
the HRQoL of patients with HAE while they were receiving Cinryze
either as routine prophylaxis or for the acute treatment of
individual attacks in the absence of prophylaxis during a
randomized, placebo-controlled, crossover study. HRQoL was measured
by the Short Form-36 V 1.0 (SF-36) questionnaire, the standard
patient-reported survey for patient health and quality of life.
Cinryze is not approved by the FDA to treat HAE attacks.
"These data demonstrate that patients who received prophylaxis
with C1 inhibitor had significantly better quality of life compared
to acute therapy with C1 inhibitor while on placebo," commented Dr.
William Lumry, lead author and
Medical Director of Asthma and Allergy Research Associates in
Dallas, TX. "The analysis
underscores the importance of educating patients on the potential
benefits of prevention, and that prevention should be considered by
physicians among the therapeutic options for patients with
hereditary angioedema."
In the randomized, placebo-controlled trial, patients received
intravenous injections of 1,000 U Cinryze or placebo every 3 to 4
days for 12 weeks and then crossed over to the other treatment for
a second 12-week period. Patients could receive open-label
Cinryze for acute treatment of breakthrough angioedema attacks
during the Cinryze period, or for acute attacks in the placebo
period. Sixteen of the 22 patients who were evaluated for efficacy
completed SF-36 questionnaires for both treatment periods.
Results of the study included the following:
- The baseline mean physical component summary score (36.4) was
well below the mean for the US general population's 'normal' HRQoL
score (50), highlighting the physical toll of experiencing frequent
and unpredictable angioedema attacks;
- Mean SF-36 scores at the end of the placebo period (plus acute
therapy with C1 inhibitor when angioedema attacks occurred) were
generally lower than or equal to baseline, indicating worsening of,
or no improvement in, HRQoL;
- Mean SF-36 scores at the end of the Cinryze prophylaxis period
were generally greater than placebo (plus acute therapy with C1
inhibitor) and baseline, indicating improvement in HRQoL over
both;
- Least-square mean differences between Cinryze prophylaxis and
placebo (plus acute therapy with C1 inhibitor) in norm-based
SF-36 scores were 6.55 (p = .015) for the physical component
summary score and 8.70 (p = .019) for the mental component summary
score;
- Statistically significant differences (p < .05) between
Cinryze prophylaxis and placebo (plus acute therapy with C1
inhibitor) were also observed for all individual SF-36 domains
(physical function, role physical, bodily pain, social function,
mental health, role emotional, vitality, and general health);
and
- The magnitude of the difference between treatment groups was
similar to or greater than those observed in other chronic diseases
that are characterized by intermittent attacks/episodes, such as
epilepsy, asthma, and migraine, when these are successfully
managed.
About Cinryze® (C1 esterase inhibitor
[human])
Cinryze is a highly purified, pasteurized and
nanofiltered plasma-derived C1 esterase inhibitor product. In
the U.S. and Canada, Cinryze is
approved for routine prophylaxis (prevention) against angioedema
attacks in adolescent and adult patients with HAE. In the EU,
the product is approved for the treatment and pre-procedure
prevention of angioedema attacks in adults and adolescents with
hereditary angioedema (HAE), and routine prevention of angioedema
attacks in adults and adolescents with severe and recurrent attacks
of hereditary angioedema (HAE), who are intolerant to or
insufficiently protected by oral prevention treatments or patients
who are inadequately managed with repeated acute treatment.
Cinryze is for intravenous use only.
Severe hypersensitivity reactions to Cinryze may occur.
Thrombotic events have occurred in patients receiving Cinryze, and
in patients receiving off-label high dose C1 inhibitor
therapy. Monitor patients with known risk factors for
thrombotic events. With any blood or plasma derived product,
there may be a risk of transmission of infectious agents, e.g.
viruses and, theoretically, the CJD agent. The risk has been
reduced by screening donors for prior exposure to certain virus
infections and by manufacturing steps to reduce the risk of viral
transmission including pasteurization and nanofiltration.
The most common adverse reactions in clinical trials associated
with Cinryze were rash, headache, nausea, erythema, phlebitis and
local reactions at the injection site. Adverse events of
sinusitis and upper respiratory infection also were observed in
clinical trials. No drug-related serious adverse events (SAEs)
were reported in clinical trials.
Please visit http://www.viropharma.com/products/cinryze.aspx for
the full U.S. Prescribing Information; the prescribing information
for other countries can be found at www.viropharma.com.
About Hereditary Angioedema (HAE)
HAE is a
rare, severely debilitating, life-threatening genetic disorder
caused by a deficiency of C1 inhibitor, a human plasma protein.
This condition is the result of a defect in the gene controlling
the synthesis of C1 inhibitor. C1 inhibitor maintains the natural
regulation of the contact, complement, and fibrinolytic systems,
that when left unregulated, can initiate or perpetuate an attack by
consuming the already low levels of endogenous C1 inhibitor in HAE
patients. Patients with C1 inhibitor deficiency experience
recurrent, unpredictable, debilitating, and potentially life
threatening attacks of inflammation affecting the larynx, abdomen,
face, extremities and urogenital tract. Patients with HAE
experience approximately 20 to 100 days of incapacitation per year.
There are estimated to be at least 6,500 people with HAE
in the United States and at least 10,000 people in
the European Union.
For more information on HAE, visit the U.S. HAE Association's
website at www.haea.org and the HAEi's (International
Patient Organization for C1 Inhibitor Deficiencies) website
at www.haei.org.
About ViroPharma Incorporated
ViroPharma Incorporated
is an international biopharmaceutical company committed to
developing and commercializing novel solutions for physician
specialists to address unmet medical needs of patients living with
diseases that have few if any clinical therapeutic
options. ViroPharma is developing a portfolio of therapeutics
for rare and Orphan diseases including C1 esterase inhibitor
deficiency, cytomegalovirus (CMV), Friedreich's Ataxia,
eosinophilic esophagitis (EoE) and adrenal insufficiency. Our goal
is to provide rewarding careers to employees, to create new
standards of care in the way serious diseases are treated, and to
build international partnerships with the patients, advocates, and
health care professionals we serve. ViroPharma's commercial
products address diseases including hereditary angioedema (HAE),
seizures in children and adolescents, adrenal insufficiency and
C. difficile-associated diarrhea (CDAD). For full U.S.
prescribing information on our products, please download the
package inserts at http://www.viropharma.com/Products.aspx; the
prescribing information for other countries can be found at
www.viropharma.com.
ViroPharma routinely posts information, including press
releases, which may be important to investors in the investor
relations and media sections of our company's web site,
www.viropharma.com. The company encourages investors to consult
these sections for more information on ViroPharma and our
business.
Forward Looking Statements
Certain statements in this
press release contain forward-looking statements that involve a
number of risks and uncertainties. Forward-looking statements in
this press release include statements regarding the quality of life
in patients taking Cinryze. There can be no assurance that
the data presented during the 70th American College of Allergy,
Asthma and Immunology (ACAAI) Annual Scientific Meeting regarding
Cinryze is predictive of how Cinryze will perform in commercial
usage. In addition, the data that were discussed in this
abstract and presentation are subject to different
interpretations. The commercialization of pharmaceutical
products is subject to risks and uncertainties. Our actual
results could differ materially from those results expressed in, or
implied by, these forward-looking statements. These factors,
and other factors, including, but not limited to those described in
our annual report on Form 10-K for the year ended December 31, 2012 and 10-Q for the quarter ended
March 31, 2013, June 30, 2013, and September 30, 2013 filed with the Securities and
Exchange Commission, could cause future results to differ
materially from the expectations expressed in this press release.
The forward-looking statements contained in this press release are
made as of the date hereof and may become outdated over time.
ViroPharma does not assume any responsibility for updating any
forward-looking statements. These forward looking statements should
not be relied upon as representing our assessments as of any date
subsequent to the date of this press release.
SOURCE ViroPharma Incorporated