FDA Announces Orphan Drug Designation for HDV Drug

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The Orphan Drug Designation Program offers orphan status to biologics and drugs that are safe and effective for rare diseases; also for prevention use.

The US Food and Drug Administration (FDA) has just announced orphan drug designation for a drug to treat chronic hepatitis delta virus (HDV) infection, the most severe form of viral hepatitis. The drug, pegylated interferon lambda 1a (Lambda), was developed by Eiger BioPharmaceuticals, Inc.

The FDA’s Orphan Drug Designation Program grants orphan status to drugs that are intended to treat, diagnose, or prevent rare diseases — sometimes called “orphan diseases”  — meaning that those diseases that affect fewer than 200,000 people in the United States.

Worldwide, however, HDV infection is more prevalent and has a significant impact on global health.  As a coinfection of hepatitis B virus (HBV), HDV leads to more severe liver disease than infection with just HBV, and to more rapidly progressing liver fibrosis, liver cancer, and liver failure. As many as 15 to 20 million people worldwide, including 4.3% to 5.7% of individuals chronically infected with HBV, may be infected with HDV. The percentage of patients with HBV who also have HDV varies by region — in some parts of the world it is as high as 60%.

Lambda is a type III interferon that stimulates critical immune responses during viral infections that help the body develop protection against the disease. The drug has been administered to more than 3000 patients in HBV/HCV clinical trials, but has not been approved for any indication.

Eiger, a biopharmaceutical company that focuses on therapies for orphan diseases, has licensed worldwide rights to Lambda.

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Eiger announces FDA Orphan Drug Designation granted to pegylated interferon lambda in hepatitis delta virus infection [press release].  Palo Alto, California: Eiger Bio, Inc. Published online September 5, 2017. Accessed September 13, 2017.