SAN FRANCISCO and GENEVA, Dec. 19, 2014 (GLOBE NEWSWIRE) -- Amarantus BioScience Holdings, Inc. (OTCQB:AMBS), a biotechnology company focused on the development of diagnostics in Alzheimer's disease and therapeutic products in the areas of neurology, psychiatry, ophthalmology and regenerative medicine, announced that it has applied to the U.S. Food & Drug Administration (FDA) for Orphan Drug Designation for its investigational drug MANF (mesencephalic-astrocyte-derived neurotrophic factor) as a treatment for retinal artery occlusion (RAO). RAO is a blockage of the blood supply to the retina which causes severe and sudden loss of vision.
MANF, a naturally-occurring protein that reduces and prevents apoptosis (programmed cell death) in response to injury or disease, was discovered utilizing Amarantus' proprietary PhenoGuardTM Protein Discovery Engine. Pre-clinical data showed that MANF provided protective functional effects in an animal model of RAO. Moreover, toxicology studies have demonstrated that MANF was well-tolerated following a single intravitreal administration of a therapeutically relevant dose.
"Filing our second Orphan Drug Designation for MANF is an important component of our regulatory strategy for the program," said Gerald E. Commissiong, President & CEO of Amarantus. "We believe that our pre-clinical efficacy data with MANF supports its use in ophthalmologic disorders such as RAO and retinitis pigmentosa (orphan status applied for in October 2014) where there are currently limited or no treatment options available. We are extremely encouraged by our data and believe MANF has the potential to provide meaningful changes to the quality of life of patients and their families."
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.
MANF, a naturally-occurring protein that reduces and prevents apoptosis (programmed cell death) in response to injury or disease, was discovered utilizing Amarantus' proprietary PhenoGuardTM Protein Discovery Engine. Pre-clinical data showed that MANF provided protective functional effects in an animal model of RAO. Moreover, toxicology studies have demonstrated that MANF was well-tolerated following a single intravitreal administration of a therapeutically relevant dose.
"Filing our second Orphan Drug Designation for MANF is an important component of our regulatory strategy for the program," said Gerald E. Commissiong, President & CEO of Amarantus. "We believe that our pre-clinical efficacy data with MANF supports its use in ophthalmologic disorders such as RAO and retinitis pigmentosa (orphan status applied for in October 2014) where there are currently limited or no treatment options available. We are extremely encouraged by our data and believe MANF has the potential to provide meaningful changes to the quality of life of patients and their families."
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.
