Retinitis pigmentosa drug granted orphan drug status

Amarantus BioScience Holdings, Inc., announced that it has received orphan drug designation from the U.S. FDA for the company's investigational drug MANF (mesencephalic-astrocyte-derived neurotrophic factor) for the treatment of retinitis pigmentosa.

"This represents an important milestone for the company as well as a significant step forward for our clinical and regulatory strategy," said Gerald E. Commissiong, president and CEO of Amarantus. "Our goal is to continue to identify ways to build value into our MANF program and advance this promising product candidate in multiple therapeutic areas with significant unmet need."

MANF is believed to have broad potential because it is a naturally-occurring protein produced by the body for the purpose of reducing and preventing apoptosis in response to injury or disease. Preclinical data showed that MANF provided protective functional effects in an animal model of retinitis pigmentosa. Toxicology studies have demonstrated that MANF was well-tolerated following a single intravitreal administration of a therapeutically relevant dose.