MassBiologics receives orphan drug status from FDA for hepatitis C treatment

December 04, 2013

BOSTON, MA - MassBiologics of the University of Massachusetts Medical School (UMMS) has received an orphan drug designation from the U.S. Food and Drug Administration (FDA) for MBL-HCV1, a monoclonal antibody developed to prevent hepatitis C virus (HCV) recurrence in patients receiving a liver transplant.

Complications from chronic HCV infection are the most common indications for liver transplantation today. For patients with end-stage liver disease or hepatocellular carcinoma resulting from HCV infection, liver transplantation is often the only treatment option, but it is not a cure for the disease. In almost all cases, the new donor liver becomes infected with HCV soon after transplantation.

MassBiologics' monocloncal antibody, currently in a Phase 2 clinical trial, is intended to prevent HCV from damaging the transplanted liver.

"Being granted orphan drug status facilitates the goal of bringing this investigational product to patients," says Deborah C Molrine, MD, deputy director of clinical affairs at MassBiologics and professor of pediatrics at University of Massachusetts Medical School. "The economic incentives available to MassBiologics and potential commercial partners through the Orphan Drug Act will contribute greatly to bringing this monoclonal antibody to market as a treatment option for patients receiving liver transplants as a result of HCV infection."

The Orphan Drug Act was established by Congress in 1983 to aid the development of new therapies for rare medical conditions or diseases that affect less than 200,000 patients annually. To help stimulate new drug development for these less common conditions, the FDA provides financial benefits to companies that achieve orphan drug designation, including market exclusivity for 7 years, tax incentives, fee waivers and potential grant support.

Developed by MassBiologics, MBL-HCV1 is a fully human monoclonal antibody that targets a region of the hepatitis C virus on its surface envelope, preventing it from infecting liver cells. MBL-HCV1 has been shown to be safe in healthy human subjects and is currently being studied in patients with chronic hepatitis C infection undergoing liver transplantation.

"Infusions of the monoclonal antibody have been well-tolerated in transplant patients and allow for delivery of the targeted HCV treatment to begin just before the removal of the diseased liver and to continue through the early post-transplant period," said Dr. Molrine. "A Phase 2 study is underway in liver transplant patients that combines the monoclonal antibody with one of the first two oral HCV direct acting anti-virals to be licensed by the FDA. We anticipate having data to present soon on the effect of this treatment on HCV detection after liver transplantation."
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About MassBiologics

MassBiologics, formerly known as the Massachusetts Biologic Laboratories, is the only non-profit FDA- licensed manufacturer of vaccines and other biologic products in the United States. MassBiologics produces 20-30 percent of the US tetanus/diphtheria vaccine supply. In addition to the HCV monoclonal antibody program, MassBiologics has discovered and developed human monoclonal antibodies to severe acute respiratory syndrome (SARS), Clostridium difficile and to rabies virus. MassBiologics traces its roots to 1894, and since then has maintained a mission to improve public health through applied research, development and production of biologic products. MassBiologics has been a part of the University of Massachusetts Medical School since 1997.

About the University of Massachusetts Medical School

The University of Massachusetts Medical School (UMMS), one of five campuses of the University system, is comprised of the School of Medicine, the Graduate School of Biomedical Sciences, the Graduate School of Nursing, a thriving research enterprise and an innovative public service initiative, Commonwealth Medicine. Its mission is to advance the health of the people of the Commonwealth through pioneering education, research, public service and health care delivery with its clinical partner, UMass Memorial Health Care. In doing so, it has built a reputation as a world-class research institution and as a leader in primary care education. The Medical School attracts more than $240 million annually in research funding, placing it among the top 50 medical schools in the nation. In 2006, UMMS's Craig C. Mello, PhD, Howard Hughes Medical Institute Investigator and the Blais University Chair in Molecular Medicine, was awarded the Nobel Prize in Physiology or Medicine, along with colleague Andrew Z. Fire, PhD, of Stanford University, for their discoveries related to RNA interference (RNAi). The 2013 opening of the Albert Sherman Center ushered in a new era of biomedical research and education on campus. Designed to maximize collaboration across fields, the Sherman Center is home to scientists pursuing novel research in emerging scientific fields with the goal of translating new discoveries into innovative therapies for human diseases.

University of Massachusetts Medical School

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