This article is more than 5 years old. Information may no longer be current.

FDA gives orphan drug designation to MANF for retinal artery occlusion

Add topic to email alerts

Receive an email when new articles are posted on

Please provide your email address to receive an email when new articles are posted on .

Subscribe

Added to email alerts

You've successfully added to your alerts. You will receive an email when new content is published.

Click Here to Manage Email Alerts

You've successfully added to your alerts. You will receive an email when new content is published.

Click Here to Manage Email Alerts

Back to Healio

We were unable to process your request. Please try again later. If you continue to have this issue please contact customerservice@slackinc.com.

Back to Healio

The FDA granted orphan drug designation to Amarantus BioScience Holdings for its investigational retinal artery occlusion treatment drug MANF, according to a press release.

Mesencephalic-astrocyte-derived neurotrophic factor (MANF) is a natural protein that prevents apoptosis in response to disease or injury. The protective effects were demonstrated in animal models of retinal artery occlusion (RAO), and toxicology studies demonstrated that single intravitreal doses were well-tolerated, according to the press release.

“[T]he toxicology data generated thus far directly supports the translational potential for MANF in this indication,” Amarantus President and CEO Gerald E. Commissiong said in the release, adding that MANF received previous orphan drug designation for treatment of retinitis pigmentosa.

The orphan drug designation provides for a 7-year marketing exclusivity period against competition, as well as certain incentives, the release said.