Medgenics Inc., a developer of a novel technology for the sustained production and delivery of therapeutic proteins in patients using their own tissue, has filed for Orphan Drug Designation with the US Food and Drug Administration (FDA) for Infradure for the treatment of hepatitis D.

Infradure is based on Medgenics' proprietary tissue-based Biopump platform technology, which uses the patient's own tissue to continuously produce and deliver therapeutic proteins, such as interferon-alpha for use in the treatment of hepatitis.

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.

This application for Orphan Drug Designation follows Medgenics' recent submission of an Investigational New Drug (IND) application to the FDA for a phase II b anaemia trial in dialysis patients using Epodure, a different implementation of the same Biopump platform that produces erythropoietin (EPO).

Marlene Haffner, MD MPH, former Director of Orphan Products Development at the FDA, and regulatory advisor to Medgenics said, “Infradure's application for the treatment of hepatitis D appears to meet the key criteria required for Orphan Drug Designation by the FDA, as the number of US patients with this disease is estimated to be considerably fewer than 200,000. Furthermore, preclinical data support that Infradure has a reasonable rationale for treatment of the disease based on its potential ability to continuously delivery interferon alpha, the current standard of care for hepatitis D that is administered by years of repeat injections. Should Orphan Drug Designation be granted, the regulatory approval route for Infradure for the treatment of hepatitis D could be significantly expedited.”

This Orphan Drug Designation filing marks the first application for Medgenics' Biopump technology seeking an expedited regulatory pathway in the US. Obtaining Orphan Drug Designation could potentially allow for substantially smaller pivotal clinical trials, as compared to a more wide-spread disease. This could lead to more immediate access of the treatment to a patient population in need. Medgenics expects to receive the FDA's initial response to the filing before the end of this quarter, and believes Orphan Designation could be confirmed during the third quarter.

Bruce Bacon, MD, past president of the American Association for the Study of Liver Disease and a recognized global expert in hepatitis who serves on Medgenics' Strategic Advisory Board, commented, “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. 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. Medgenics' treatment is potentially a game changer not only as a treatment for hepatitis D, but also as a key element in the treatment of various other forms of hepatitis worldwide.”

“Our application for Orphan Drug Designation for hepatitis D demonstrates Medgenics' commitment to the treatment of hepatitis. Obtaining Orphan Drug Designation could be the most rapid route for us to bring our Biopump technology to the US,” stated Andrew L Pearlman, PhD, president and CEO of Medgenics. “We recently submitted an IND application with the FDA for a phase IIb anaemia trial for EPODURE, a version of the Biopump which produces a different protein, EPO, and have received approval in Israel to commence a phase IIa trial for that same indication. We are very encouraged with the momentum we are building in 2012 as these important clinical advancements reflect the progress that various applications of our platform technology are making through regulatory approval processes as we continue to build shareholder value.”

“We are eager to pursue this niche opportunity in hepatitis D, which would use a similar Infradure approach to that we will employ in treating hepatitis C in a phase I/II study in Israel that is scheduled to commence in the third quarter of 2012 pending final regulatory approval. Receiving Orphan Drug Designation for our Infradure Biopump in the treatment of hepatitis D could lead not only to an expedited approval route based on clinical studies of moderate size for this specific rare indication, but could also represent a significant advancement of our entire portfolio of treatments. Such an approval may serve to pave the way for a more rapid pace at which our platform protein delivery technology can move through the FDA approval process for other multibillion-dollar clinical indications,” added Dr Pearlman.

According to the US 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.

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.