FDA grants Orphan Drug Designation to Medgenics’ INFRADURE Biopump

Medgenics, Inc. (NYSE MKT: MDGN and AIM: MEDU, MEDG), the developer of Biopump^TM, 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.

Source: Medgenics, Inc.