Amarantus Applies for Orphan Drug Designation for MANF in treatment of Retinitis Pigmentosa

Amarantus Bioscience Holdings, Inc., a biotechnology company focused on the development of diagnostics and therapeutics for Alzheimer's disease, Parkinson's disease and ophthalmological disorders, announced that it has applied to the Food & Drug Administration in the US for Orphan Drug Designation for MANF (mesencephalic-astrocyte-derived neurotrophic factor) in retinitis pigmentosa (RP).


MANF, a naturally-occurring protein that reduces and prevents apoptosis (programmed cell death) in response to injury or disease, 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.

The FDA 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.

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