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MANF receives orphan drug designation for retinitis pigmentosa

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MANF, an investigational drug, has received orphan drug designation for the treatment of retinitis pigmentosa from the U.S. Food and Drug Administration, Amarantus BioScience Holdings announced in a press release.

Preclinical data showed that MANF (mesencephalic astrocyte-derived neurotrophic factor) had a protective functional effect in an animal model of retinitis pigmentosa, and toxicology studies showed a single intravitreal administration of a therapeutically relevant dose was well-tolerated.

“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,” Gerald E. Commissiong, president and CEO of Amarantus, said in the release.

The designation will give Amarantus 7 years of marketing exclusivity against competition and other incentives, according to the release.