The FDA granted orphan drug status to MassBiologics for MBL-HCV1, a monoclonal antibody designed to prevent recurrence of hepatitis C in patients receiving liver transplantation, according to a news release.
“Being granted orphan drug status facilitates the goal of bringing this investigational product to patients,” Deborah C. Molrine, MD, deputy director of clinical affairs at MassBiologics and professor of pediatrics at University of Massachusetts Medical School, said in the release. “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.”
While liver transplant in HCV patients almost always leads to the new liver quickly becoming infected with HCV, MBL-HCV1 targets a portion of the surface envelope of the hepatitis C virus, reducing its ability to infect liver cells, the release said. It has been found to be safe in healthy patients and is in the midst of a phase 2 trial involving patients with chronic hepatitis C undergoing liver transplant.
“Infusions … 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,” Molrine said. Further data are expected shortly, she said.
The FDA’s orphan drug program offers incentives to drugmakers working on therapies affecting fewer than 200,000 people annually in the United States and those affecting more than 200,000 people but not expected to allow for recovery of the costs of developing and marketing the treatment.