Biotechnology company Amarantus Bioscience Holdings, Inc. (AMBS-OTC) reported this morning that it had applied to the U.S. Food & Drug Administration (FDA) for Orphan Drug Designation for MANF (mesencephalic-astrocyte-derived neurotrophic factor) in retinitis pigmentosa. The company's stock appeared to be responding well to the news, and was up over 9% as of 11 am (ET).
What is MANF?
Discovered by Amarantus's chief scientific officer, Dr. John Commissiong, Mesencephalic Astrocyte-derived Neurotrophic Factor (MANF) is a naturally occurring protein that reduces and prevents apoptosis (programmed cell death) in response to injury or disease. It is in development to treat several apoptosis-related disorders, including retinitis pigmentosa as well as Parkinson's disease, Wolfram’s Syndrome, diabetes, ischemic heart disease, and others. Today's news focuses on advancing the development of MANF for retinitis pigmentosa, which is an inherited, degenerative eye disease that causes severe vision impairment and often blindness by age 40.
MANF has demonstrated efficacy in three animal models of retinitis pigmentosa. Amarantus is collaborating with the University of Miami's Bascom Palmer Eye Institute and the Buck Institute for Research on Aging in its retinitis pigmentosa program. Additionally, the company has completed toxicology experiments, concluding that MANF was well-tolerated following intravitreal administration at therapeutically relevant doses.
Initially, orphan indications such as retinitis pigmentosa could provide Amarantus with an opportunity to reach the market in a more expeditious timeframe. The FDA's Orphan Drug Designation program provides a special status to drugs and biologics intended to treat, diagnose, or prevent so-called orphan diseases and disorders that affect fewer than 200,000 people in the U.S. This designation provides for a seven-year marketing exclusivity period against competition, as well as certain incentives, including federal grants, tax credits and a waiver of PDUFA filing fees.