EXTON, Pa., June 11, 2013 /PRNewswire/ -- ViroPharma
Incorporated (Nasdaq: VPHM) today announced that the European
Commission has granted orphan drug designation for maribavir for
treatment of cytomegaloviral (CMV) disease in patients with
impaired cell mediated immunity. The "Orphan Medicinal
Product Designation" is designed to encourage the development of
drugs which may provide significant benefit to patients suffering
from rare diseases identified as "life-threatening or chronically
debilitating" conditions. ViroPharma has previously received
orphan drug designation for maribavir in the United States for treatment of clinically
significant cytomegalovirus viremia and disease in at-risk
patients.
Under EMA guidelines, Orphan Medicinal Product Designation
provides 10 years of potential market exclusivity if the product
candidate is approved for marketing in the European Union and the
orphan designation is maintained. Orphan status also permits
EMA assistance in optimizing the candidate's clinical development
through participation in designing the clinical protocol and
preparing the marketing application. Additionally, a drug
candidate designated by the EMA as an Orphan Medicinal Product may
qualify for a reduction in regulatory fees as well as a European
Union-funded research grant. Finally, if a Pediatric Investigation
Plan is completed, an additional two years of exclusivity could be
granted for a product with Orphan Medicinal Product
designation.
"We are very pleased to receive orphan designation for maribavir
from the European Commission," commented John Watson, ViroPharma's senior director,
regulatory affairs, Europe. "This
designation recognizes the potential of maribavir to address great
unmet medical needs. We commend the European Commission for
providing incentives such as this for the development of drugs for
rare and life threatening diseases."
ViroPharma is currently conducting two Phase 2 dose ranging
studies of oral maribavir at one of three doses (400mg, 800mg or
1200mg BID) in transplant recipients. The first is a randomized,
active (valganciclovir) controlled maribavir dose blinded
multicenter Phase 2 study in up to 160 European hematopoietic stem
cell or solid organ transplant recipients who have demonstrated CMV
viremia but do not have CMV organ disease. ViroPharma is also
conducting a randomized, dose blinded multicenter Phase 2
study intended to enroll up to 120 hematopoietic stem cell or solid
organ transplant recipients who have resistant or refractory CMV
viremia with or without CMV organ disease. This study is currently
being conducted in the US with plans to extend to include European
sites.
ViroPharma Incorporated is based in Exton, Pennsylvania, US, and also has offices
in Canada and in eight European
countries including Belgium,
France, Germany, Italy, Spain,
Sweden, Switzerland and the United Kingdom. ViroPharma is continuing to
grow its footprint and presence in Europe, and to support this growth,
ViroPharma's European operations were initiated in 2007,
leveraging in Europe the company's
expertise in the development and commercialisation of biotechnology
products, and in business development.
About maribavir
Maribavir, a member of a new class of
drugs called benzimidazole ribosides, is a potent and selective,
orally bioavailable antiviral drug with a unique mechanism of
action against cytomegalovirus and a favorable clinical safety
profile. Unlike currently available anti-CMV agents that inhibit
CMV DNA polymerase, maribavir inhibits viral DNA assembly and
inhibits egress of viral capsids from the nucleus of infected
cells. Maribavir is active in vitro against strains of CMV that are
resistant to commonly used anti-CMV drugs. Maribavir does not
affect the maturation of viral DNA or affect the viral DNA
polymerase. The previous focus of clinical development of maribavir
as an anti-CMV agent was on the prevention of CMV disease in
transplant patients. Results from Phase 3 studies indicated
that maribavir at a dose of 100mg BID failed to meet its efficacy
endpoints. However, maribavir has demonstrated a favorable
safety and tolerability profile; across all clinical studies to
date, the most notable adverse event associated with maribavir was
taste disturbance. While Phase 3 studies of CMV prophylaxis
at the 100mg BID dose did not show sufficient activity to prevent
CMV disease, the overall safety profile of maribavir and limited
data from cases in which open-label maribavir was used as CMV
treatment suggest that higher doses may provide clinical activity
and clinical studies are ongoing. The U.S. Food and Drug
Administration (FDA) and the European Commission have granted
orphan drug designation to maribavir for treatment of clinically
significant cytomegalovirus viremia and disease in at-risk
patients, and treatment of cytomegalovirus disease in patients with
impaired cell mediated immunity, respectively.
About Cytomegalovirus
CMV is a member of the
herpes virus group, which includes the viruses that cause
chicken pox, mononucleosis, herpes labialis (cold sores), and
herpes genitalis (genital herpes). Like other herpesviruses,
CMV has the ability to remain dormant in the body for long periods
of time. Human CMV infection rates average between 50 percent
and 85 percent of adults in the U.S. by 40 years of age, but in
healthy adults causes little to no apparent illness. However, in
immunocompromised individuals including cancer patients, HIV
patients, and transplant patients, and in children born with
primary CMV infection, CMV can lead to serious disease or
death. Patients who are immunosuppressed following
hematopoietic stem cell (bone marrow) or solid organ
transplantation are at high risk of CMV infection. In these
patients, CMV can lead to severe conditions such as pneumonitis or
hepatitis, or to complications such as acute or chronic rejection
of a transplanted organ. While currently available systemic
anti-CMV agents are effective against the virus, their use is
limited by toxicities, most notably bone marrow suppression and
renal impairment.
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,
including C1 esterase inhibitor deficiency, treatment of seizures
in children and adolescents, adrenal insufficiency, and 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 healthcare
professionals we serve.
ViroPharma routinely posts information, including press
releases, which may be important to investors in the investor
relations and media sections of our company's website,
http://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.
Various known and unknown risks, uncertainties and other factors
could lead to material differences between the actual future
results, financial situation, development or performance of the
company and the estimates given here. Forward-looking statements in
this press release include statements regarding potential
therapeutic indication and use of maribavir and regarding
maribavir's potential to address unmet medical needs. The
development and commercialization of pharmaceutical products is
subject to risks and uncertainties. There can be no assurance that
studies with maribavir will generate positive results or that
maribavir will receive regulatory approvals. The EMA may view data
regarding maribavir as insufficient or inconclusive, request
additional data, require additional clinical studies, limit any
approved indications, deny the approval of maribavir or deny orphan
drug market exclusivity at the time of any future approval. A full
list and description of risks and uncertainties can be found in
ViroPharma's Annual Report on Form 10-K for the fiscal year ended
December 31, 2012, and 10-Q for the
quarter ended March 31, 2013 filed
with the Securities and Exchange Commission. 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