Amarantus Gets FDA Orphan Drug Status For Retinitis Pigmentosa Treatment MANF

By RTT News, December 23, 2014, 04:22:00 PM EDT

(RTTNews.com) - 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.

For comments and feedback: contact editorial@rttnews.com

http://www.rttnews.com

More:
Amarantus Gets FDA Orphan Drug Status For Retinitis Pigmentosa Treatment MANF