Replies to post #67280 on Biotech Values

[rkrw]

Its not surprising it got pulled. The fda had disagreed with them on stats. fda switches a few things around and they just missed significance. So I think the fda as much as told them it wouldn't be approved for acute without more work. FDA may even be gaming it to make sure there's two sources of supply. In reality it won't make much difference commercially, people will use it as they need it and not stick to the label. I do expect CSL to get approval for the acute indication some time in 2009. Both will essentially be interchangable. My total guess levp (vphm) ends up with about 80% share C1 acute+prevention, first mover advantage, good relations with the hae association and more nimble than the large CSL where C1 probably doesn't move their needle a ton.

[Lewis R Goudy]

ViroPharma Submits Supplemental Biologics License Application for
Cinryze(TM) to Treat Acute Attacks of Hereditary Angioedema


EXTON, Pa., Dec 01, 2008 /PRNewswire-FirstCall via COMTEX/ -- ViroPharma
Incorporated (Nasdaq: VPHM) today announced that the company submitted a
supplemental Biologics License Application (sBLA) to the U.S. Food and Drug
Administration (FDA) for Cinryze(TM) C1 Inhibitor (human) as a treatment for
acute attacks of hereditary angioedema (HAE).

The sBLA is based on a re-analysis and resubmission of data from a pivotal Phase
3 acute treatment study of Cinryze and interim data from an ongoing open label
acute study of the drug. The administration of Cinryze was effective in treating
acute HAE attacks in these studies. The safety profile was similar to that
observed with the use of Cinryze for routine prophylaxis of angioedema attacks
in patients with HAE, the currently approved claim. Overall, more than 9,000
doses of Cinryze have been administered to over 180 patients in all controlled
and open label clinical studies of Cinryze for both acute treatment and routine
prophylaxis against angioedema attacks.

The Phase 3 acute treatment study was a randomized, double blind, placebo
controlled multi-center trial in 71 patients evaluating the safety and efficacy
of Cinryze for treatment of HAE attacks. The primary efficacy measure in the
pivotal Phase 3 acute treatment study was the time from initial treatment to the
start of unequivocal relief of the defining symptom. Based on the primary
efficacy variable, in the All Randomized (ITT) Dataset, the likelihood of a
patient having the start of unequivocal relief of the defining symptom was 2.048
times greater in the Cinryze treatment group than in the placebo treatment group
(p=0.048). The median time to the start of unequivocal relief of the defining
symptom was shorter in subjects in the Cinryze treatment group (two hours) than
in subjects in the placebo treatment group (greater than four hours).

In the open label study of Cinryze as treatment for acute attacks of HAE, no
patients who had acute laryngeal edema attacks required hospitalization or
intubation. Cinryze was generally well tolerated. There were no deaths or
serious adverse reactions related to Cinryze administration, or discontinuations
due to treatment-emergent adverse events. In the analysis of 447 acute attacks
in 82 patients, open label Cinryze administration provided substantial relief of
the defining symptom in 93.4 percent of the attacks within four hours of
injection, with a median time to onset of relief of 30 minutes. There was no
observed loss of effectiveness over multiple administrations of Cinryze for
subsequent HAE attacks.

"Routine prophylaxis against angioedema attacks is beneficial for many HAE
patients, and an FDA-approved medication to treat acute attacks also would be
important in managing this devastating disease," said Robert Pietrusko,
Pharm.D., ViroPharma's vice president, global regulatory affairs and quality.
"We've made great strides in recent weeks with the approval of Cinryze and the
launch of our patient access program CINRYZESolutions(TM) to assure its
availability for all patients who are in need of this critical therapy. We are
also pleased to have made this acute treatment submission this year."

About Cinryze C1 Inhibitor (human)

Cinryze is a highly purified, pasteurized and nanofiltered plasma-derived C1
inhibitor product that has been approved by FDA for routine prophylaxis against
angioedema attacks in adolescent and adult patients with HAE. C1 inhibitor
therapy has been used acutely for more than 30 years in Europe to treat patients
with C1 inhibitor deficiency. Cinryze has not been approved for acute treatment
in the United States or any other jurisdiction.

Cinryze has been well tolerated. The most common adverse reactions observed have
been upper respiratory infection, sinusitis, rash and headache. No drug-related
serious adverse events (SAEs) have been observed in clinical trials. Severe
hypersensitivity reactions may occur. Thrombotic events have occurred in
patients receiving high dose off-label C1 inhibitor therapy well above the
approved treatment dosage regimen. 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 patients
for prior exposure to certain virus infections and by manufacturing steps to
reduce the risk of viral transmission including pasteurization and
nanofiltration.

Cinryze is for intravenous use only. A dose of 1000 Units Cinryze can be
administered every 3 or 4 days for routine prophylaxis against angioedema
attacks in HAE patients. Cinryze is administered at an injection rate of 1 mL
per minute.

About Hereditary Angioedema

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 unrestricted, 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 4,600 people with
HAE in the United States.

For more information on HAE, visit the U.S. HAE Association's website at:
www.haea.org.

About ViroPharma Incorporated

ViroPharma Incorporated is a biopharmaceutical company dedicated to the
development and commercialization of products that address serious diseases
treated by physician specialists and in hospital settings. ViroPharma
commercializes Vancocin(R) (vancomycin hydrochloride capsules, USP), approved
for oral administration for treatment of antibiotic-associated pseudomembranous
colitis caused by Clostridium difficile and enterocolitis caused by
Staphylococcus aureus, including methicillin-resistant strains, and Cinryze(TM)
(C1 inhibitor (human)) for routine prophylaxis against angioedema attacks in
adolescent and adult patients with hereditary angioedema (HAE), also known as C1
inhibitor deficiency (for prescribing information on ViroPharma's commercial
products, please download the package inserts at
http://www.viropharma.com/Products.aspx). ViroPharma currently focuses its drug
development activities in diseases including cytomegalovirus (CMV), HAE and C.
difficile.

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
the Company's current expectations or forecasts of future events.
Forward-looking statements in this press release include statements regarding
ViroPharma's clinical development programs. Our actual results could differ
materially from those results expressed in, or implied by, these forward-looking
statements. The development and commercialization of pharmaceutical products is
subject to risks and uncertainties. The data that were submitted to the U.S.
Food and Drug Administration includes data from two separate studies including
the pivotal Phase 3 study of Cinryze in acute HAE attacks and the ongoing
open-label study of Cinryze for acute treatment of HAE, which includes partial
data from an ongoing open label study. There can be no assurance that the
complete data from the open label study will demonstrate that Cinryze
successfully treats all types of acute hereditary angioedema (HAE) attacks and
may not be predictive of the results of any future testing. The FDA may view the
data regarding the use of Cinryze for acute treatment of HAE we have submitted
as a supplemental BLA as insufficient or inconclusive, not accept our
submission, request additional data, require additional clinical studies, delay
any decision past the time frames anticipated by us, limit any approved
indications, deny the approval of Cinryze for acute treatment of HAE or approve
a competing product which has been granted orphan drug designation thereby
preventing Cinryze from reaching the market for acute treatment of HAE. These
factors, and other factors, including, but not limited to those described in
ViroPharma's annual report on Form 10-K and quarterly reports on Form 10-Q filed
with the Securities and Exchange Commission during 2008, could cause future
results to differ materially from the expectations expressed in this press
release. The forward- looking statements contained in this press release may
become outdated over time. ViroPharma does not assume any responsibility for
updating any forward- looking statements.

SOURCE ViroPharma Incorporated



URL: http://www.viropharma.com

http://www.haea.org

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