Sub-Group Analysis Of Data From Pediatric Patients With Hereditary Angioedema Published In The Journal Of Pediatrics

Sub-Group Analysis Of Data From Pediatric Patients With Hereditary Angioedema
                    Published In The Journal Of Pediatrics

- Publication Describes Safety and Efficacy Profile of Cinryze® (C1 esterase
inhibitor [human]) in Children and Adolescents Receiving 2,237 Infusions -

PR Newswire

EXTON, Pa., Jan. 16, 2013

EXTON, Pa., Jan. 16, 2013 /PRNewswire/ -- ViroPharma Incorporated (Nasdaq:
VPHM) today announced the publication of data demonstrating that use of
Cinryze^® (C1 esterase inhibitor [human]) in pediatric patients provided
relief from symptoms of hereditary angioedema (HAE) attacks and reduced the
rate of attacks. HAE is a rare, severely debilitating, life-threatening
genetic disorder caused by a deficiency of a human plasma protein called C1
inhibitor. The paper entitled Nanofiltered C1-Esterase Inhibitor for The Acute
Management and Prevention of Hereditary Angioedema Attacks Due to C1-Inhibitor
Deficiency in Children by Dr. William Lumry et al. describes the efficacy and
the safety profile of Cinryze in prevention and treatment of HAE attacks in
the largest analysis of pediatric data from prospective studies of patients
with HAE ever reported in medical literature. The article is in press at The
Journal of Pediatrics and was posted online on January 14, 2013.

Cinryze is approved by the U.S. Food and Drug Administration (FDA) for routine
prophylaxis against angioedema attacks in adolescent and adult patients with
HAE; Cinryze is not approved to treat HAE attacks or for procedural
prophylaxis. Cinryze is not approved for use in children.

According to published literature, in most patients with HAE, clinical
symptoms manifest in childhood, typically between the ages of four and 11
years and may worsen during puberty. Symptoms and frequency of attacks
increase during periods of intense physiologic development, such as between
the ages of three and 6 years and with onset of puberty. Subcutaneous edema
and recurrent abdominal pain caused by gastrointestinal edema are the most
common manifestations in children. Common attack triggers include infections,
emotional stress, and tissue trauma. Asphyxia is possible when angioedema
involves the upper airway and can occur rapidly in children because of narrow
airway diameter. Despite this, the diagnosis of HAE is often delayed until
late adolescence or adulthood.

Data from the Routine Prophylaxis Placebo-Controlled Study
Four pediatric patients (ages 9 to 17 years) enrolled in and completed the
pivotal prophylaxis trial. Children had a nearly 2‑fold reduction in number
of HAE attacks while receiving Cinryze for prophylaxis compared with the time
period during which they received placebo (mean number of attacks: 7.0 vs 13.0
over 12 weeks), consistent with the published data from the study population
as a whole (6.26 vs. 12.73).

Data from the Open-Label Extension of the Routine Prophylaxis Study
Twenty-three children received open-label Cinryze for routine prophylaxis.
Prior to enrollment, the median monthly attack rate was 3.0 and decreased to
0.39 while the patients were receiving Cinryze for routine prophylaxis. The
majority of patients (20/23, 87 percent) experienced less than or equal to one
attack per month, and 22 percent reported no attacks during the study period.

Pre-procedural Prophylaxis
Eight children received Cinryze prior to 40 procedures; 90 percent of which
were dental procedures. A single 1000 U dose of Cinryze was administered prior
to 39 procedures, and two 1000 U doses were administered over a 48-hour period
for one procedure per investigator's discretion. Across all procedures, only
one HAE attack was reported within 72hours after pre-procedural dosing; no
attacks occurred during or within 2 days of the administration of Cinryze.

Data from the Placebo-Controlled Study on Treating HAE Attacks
Twelve pediatric patients were treated for an attack (7Cinryze, 5placebo) in
the placebo-controlled acute-attack treatment study. Another 3children
received open-label Cinryze for treatment of laryngeal angioedema and/or prior
to emergency surgical procedures. Unequivocal relief of the defining symptom
began within 4 hours after initial treatment in 71 percent of patients (5 of
7) receiving Cinryze, consistent with the rate observed in the study
population as a whole (60 percent), compared with 2 of 5 patients receiving
placebo. For those children who achieved unequivocal relief, the median time
to the beginning of unequivocal relief was 30 minutes with Cinryze compared
with 2 hours with placebo.

Data from the Open-Label Study on Treating Acute Attacks
In the open-label acute extension, 22 pediatric patients experienced 121
attacks. Eighty-eight attacks were treated with one dose of Cinryze and 33
attacks with two doses. Unequivocal relief started within 1 hour after the
initial dose of Cinryze in 79 percent of attacks and within 4 hours after the
initial dose in 89 percent of attacks. In the majority of laryngeal attacks,
unequivocal relief started within 1 hour, and no child receiving Cinryze
required intubation or hospitalization for a laryngeal attack. Response rate
within 4hours and time to beginning of relief remained consistent
irrespective of attack number or location. In addition, one hundred seventeen
attacks were evaluated for clinical relief (discharged to home prior to
obtaining all assessments required for unequivocal relief); 113 of 117 (97
percent) achieved clinical relief within 4 hours.

Safety Data
In the pivotal prophylaxis study, one patient experienced pyrexia that was
considered by the investigator to be possibly related to study drug. In the
open-label prophylaxis extension, 17 of 23 patients reported adverse events;
two patients reported a total of three adverse events that were considered by
the investigator to be related to Cinryze: One patient had headache and
nausea, and the other had infusion-site erythema. All 3 of these events were
mild in severity.

No adverse events were reported in the acute-attack treatment trial. In the
open-label treatment extension, 9of 24 subjects reported adverse events. No
adverse events in the open-label treatment extension were considered by the
principal investigator to be related to Cinryze.

No serious or severe adverse events were considered by the investigator to be
related to Cinryze, and no adverse events led to treatment discontinuation.
There was no evidence of human immunodeficiency virus (HIV) or viral hepatitis
transmission or development of clinically relevant anti-C1 INH antibodies in
these studies.

Design of Sub-Group Analysis of Prospective Data
Forty-six children and adolescents ranging in age from two to 17 years
received a total of 2,237 infusions of Cinryze across two randomized,
placebo-controlled studies and their open-label extensions. One of the studies
and its extension evaluated the use of Cinryze in acute HAE attacks, and the
other evaluated its use in prophylaxis. In the pivotal acute-attack treatment
trial, patients who presented to the study site within 4 hours after onset of
a moderate or severe acute attack of the abdomen, face, or external genitalia
were randomly assigned to intravenous infusions of placebo or Cinryze 1000 U.
The pivotal prophylaxis trial consisted of 2 consecutive 12-week treatment
periods during which patients received study medication for preventing HAE
attacks. Patients were randomly assigned to receive intravenous infusions of
1000 U Cinryze or placebo every 3 to 4 days.

"Through an analysis of the largest compilation of pediatric patient data from
prospective HAE clinical studies, we were able to determine that the safety
and efficacy profile of Cinryze in this pediatric subpopulation were similar
to those seen in the overall trial population," commented Dr. William Lumry,
Medical Director, Asthma and Allergy Research Associates in Dallas, Tx.
"Though no drugs are currently approved for children with HAE, taken together,
these data offer a substantive body of evidence supporting the clinical
utility of Cinryze in pediatric patients with hereditary angioedema."

About Cinryze^® (C1 esterase inhibitor [human])
Cinryze is a highly purified, pasteurized and nanofiltered plasma-derived C1
esterase inhibitor product. In the U.S., Cinryze is approved by the FDA for
routine prophylaxis against angioedema attacks in adolescent and adult
patients with HAE. In the E.U., the product is approved by the EMA 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 inthe United Statesand at least 10,000 people in theEuropean Union.

For more information on HAE, visit the U.S. HAE Association's website
atwww.haea.organd the HAEi's (International Patient Organization for C1
Inhibitor Deficiencies) website atwww.haei.org.

AboutViroPharma 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, Friedreich's Ataxia, and adrenal insufficiency,
cytomegalovirus (CMV); and recurrent C. difficile infection (CDI). 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, 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
provide our current expectations or forecasts of future events.
Forward-looking statements in this press release include statements regarding
the therapeutic use, safety, efficacy, tolerability and potential of Cinryze
in children and adolescents. Our actual results could differ materially from
those results expressed in, or implied by, these forward-looking statements.
The commercialization of pharmaceutical products is subject to risks and
uncertainties. The data that were discussed in The Journal of Pediatrics
article are subject to different interpretations and may not be predictive of
the results of any individual's results or of how Cinryze performs in
commercial usage. 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 September 30, 2012
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

Website: http://www.viropharma.com
Contact: ViroPharma Incorporated, Kristina M. Broadbelt (Media), Associate
Director, PR & Advocacy, +1-610-321-2358, or Robert A. Doody Jr. (Investors),
Assistant Director, Investor Relations, +1-610-321-6290
 
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