Medgenics Receives Orphan Drug Designation from FDA for INFRADURE Biopump to Treat Hepatitis D
June 20 2012 - 10:31AM
Business Wire
Medgenics, Inc. (NYSE MKT: MDGN and AIM: MEDU, MEDG), the
developer of BiopumpTM, a novel technology for the sustained
production and delivery of therapeutic proteins in patients using
their own tissue, today announced that its INFRADURE™ Biopump has
received Orphan Drug Designation from the U.S. Food and Drug
Administration (FDA) Office of Orphan Products Development (OOPD)
for the treatment of hepatitis D. INFRADURE is the version of
Medgenics’ Biopump technology platform which produces interferon
alpha, commonly used to treat hepatitis. This marks the first
Orphan Drug Designation granted for treatment of a clinical
indication using the Biopump.
Orphan Drug Designation carries multiple benefits, including the
availability of grant money, certain tax credits and seven years of
market exclusivity, as well as the possibility of an expedited
regulatory process.
Marlene Haffner, M.D. MPH, former Director of Orphan Products
Development at the FDA, and regulatory advisor to Medgenics said,
“I am excited by this grant of Orphan Drug Designation of INFRADURE
in hepatitis D. The novel Biopump platform potentially offers
significant advance over current treatment not just for an orphan
disease, but also for other diseases where protein therapy is a
potential treatment. Designated Orphan Products have frequent
access for advice to both FDA’s OOPD and to the FDA review
divisions. They are also frequently approved by FDA via an
accelerated pathway of Priority Review, which is granted for drugs
offering a significant advance in the treatment of a serious and
life threatening disease, or when there is no approved treatment.
The INFRADURE Biopump seems to meet these criteria, particularly
since hepatitis D is a serious disease with no approved
treatment.”
Bruce R. Bacon, M.D., past President of the American Association
for the Study of Liver Disease, a recognized global expert in
hepatitis and a member of Medgenics’ Strategic Advisory Board,
commented, “INFRADURE offers the potential for a superior treatment
for the 15 million people suffering from hepatitis D worldwide. The
current treatment for hepatitis D requires years of weekly
injections of interferon alpha, which leads to patient discomfort
and substantial compliance challenges. Oral antiviral treatments
have proven to be ineffective in treating hepatitis D. INFRADURE is
intended to be implanted infrequently, with a single administration
potentially replacing many months of weekly injections. This could
offer a safe and efficacious treatment that could greatly improve
patient compliance. The treatment also has potential for efficacy
with greater patient compliance for other forms of hepatitis
including the 170 million people infected with hepatitis C and the
over 350 million people infected with hepatitis B.”
“The FDA’s timely approval of our Orphan Drug Designation for
INFRADURE to treat hepatitis D is a key milestone in our broader
hepatitis program as data gathered through clinical trials for
INFRADURE in hepatitis D may serve as relevant support for other
clinical uses of INFRADURE including hepatitis C and hepatitis B,”
stated Andrew L. Pearlman, Ph.D., President and Chief Executive
Officer of Medgenics. “Following on the FDA’s recent clearance to
proceed with our Phase IIb study of the EPODURE™ Biopump technology
to treat anemia, and the positive comments of the National
Institutes of Health Recombinant DNA Advisory Committee, we see
this Orphan Drug Designation from the FDA as another important step
in the regulatory pathway for our Biopump platform. As Dr. Haffner
has indicated, we are hopeful that this will lead to a streamlined
regulatory pathway to approval of INFRADURE in hepatitis D and
believe that it could also help advance the regulatory pathway for
other applications of our Biopump technology in the U.S.”
“We look forward to updating our clinical trial plans in view of
the Orphan Drug Designation. We anticipate that hepatitis D
clinical studies will use INFRADURE Biopumps that are fundamentally
identical to those which will be used for the treatment of
hepatitis C in our two Phase I/II studies in Israel which are now
awaiting final approval from the Israeli Ministry of Health.” added
Pearlman.
About Hepatitis D
According to the U.S. Centers for Disease Control and
Prevention, hepatitis D, also known as "delta hepatitis," is a
serious liver disease caused by infection with the hepatitis D
virus (HDV), which is an RNA virus structurally unrelated to the
hepatitis A, B or C viruses. Hepatitis D, which can be acute or
chronic, is not common in the United States. HDV is an incomplete
virus that requires the helper function of the hepatitis B virus
(HBV) to replicate and only occurs among people who are infected
with HBV. HDV is transmitted through percutaneous or mucosal
contact with infectious blood and can be acquired either as a
co-infection with HBV or as a super-infection in persons with HBV
infection. There is no vaccine for hepatitis D, but it can be
prevented in persons who are not already HBV-infected by
administrative of the hepatitis B vaccination. Hepatitis D infects
about 15 million people worldwide.
About Medgenics
Medgenics is developing and commercializing Biopump™, a
proprietary tissue-based platform technology for the sustained
production and delivery of therapeutic proteins using the patient's
own skin biopsy for the treatment of a range of chronic diseases
including anemia, hepatitis, and hemophilia, among others.
Medgenics believes this approach has multiple benefits compared
with current treatments, which include regular and costly
injections of therapeutic proteins.
Medgenics has three long-acting protein therapy products in
development based on this technology:
- EPODURETM to produce and deliver
erythropoietin for many months from a single administration, which
has demonstrated elevation and stabilization of hemoglobin levels
in anemic patients for periods of six months to more than 36 months
in a Phase I/II dose-ranging trial in Israel and has received
approval for a Phase IIa trial in dialysis patients due to launch
in Q2 2012 in Israel. An Investigational New Drug application has
been cleared by the FDA to initiate a Phase IIb study to evaluate
the safety and efficacy of EPODURE in the treatment of anemia in
dialysis patients in the U.S.
- INFRADURETM for sustained production
and delivery of interferon-alpha for use in the treatment of
hepatitis is awaiting final approval of the Israeli Ministry of
Health of two Phase I/II trials in Israel in hepatitis C, slated to
commence Q3 2012.
- HEMODURETM for sustained production and
delivery of clotting Factor VIII therapy for the sustained
prophylactic treatment of hemophilia is now in development.
Medgenics is focused on the development and manufacturing of its
innovative Biopumps, aiming to bring them to market via strategic
partnerships with major pharmaceutical and/or medical device
companies.
In addition to treatments for anemia, hepatitis and hemophilia,
Medgenics plans to develop and/or out-license a pipeline of future
Biopump products targeting the large and rapidly growing global
protein therapy market, which is forecast to reach $132 billion in
2013. Other potential applications for Biopumps include multiple
sclerosis, arthritis, pediatric growth hormone deficiency, obesity
and diabetes.
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