Orphan drug designation

Orphan drugs

What is “Orphan Drug Designation”? If our drug receives an orphan drug designation from a regulatory agency, such as US FDA, it is easier to gain marketing approval, and there may be other financial incentives. This usually applies to a drug for a rare disease. I have just found out that Amarantus BioScience has received an orphan drug designation for MANF from FDA for treatment of retinitis pigmentosa. Because we are actively working on MANF for developing treatment for Wolfram syndrome, this is good news. If we get positive preclinical data, we have a chance to get an orphan drug designation for MANF for Wolfram syndrome. So I was quite encouraged by the news.

Thank you for reading this. I always appreciate your encouragement. I feel that something wonderful is going to happen to us soon.

Take care,

Fumi Urano