Amarantus Gets FDA Orphan Drug Status For Retinitis Pigmentosa Treatment MANF

(RTTNews) - Amarantus BioScience Holdings, Inc. (AMBS) said Tuesday that the U.S. Food and Drug Administration has granted the company's investigational drug MANF orphan drug designation for the treatment of Retinitis Pigmentosa.

Retinitis Pigmentosa refers to a group of inherited diseases causing retinal degeneration often leading to blindness.

MANF, a naturally-occurring protein that reduces and prevents apoptosis in response to injury or disease, was discovered utilizing Amarantus' proprietary PhenoGuard Protein Discovery Engine. Pre-clinical data showed that MANF provided protective functional effects in an animal model of Retinitis Pigmentosa. Moreover, toxicology studies have demonstrated that MANF was well-tolerated following a single intravitreal administration of a therapeutically relevant dose.

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. The 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.