According to the US Food and Drug Administration (FDA), an...

According to the US Food and Drug Administration (FDA), an orphan drug is defined as one "intended for the treatment, prevention or diagnosis of a rare disease or condition, which is one that affects less than 200,000 persons in the United States."[4][5] In the European Union (EU), the European Medicines Agency (EMA) defined a drug as "orphan" if it is intended for the diagnosis, prevention or treatment of a life-threatening or chronically and seriously debilitating condition affecting not more up to 10,000 EU people.

My take is this is for only FSGS only..not related to the big dollars we talking about for our main trial...


DMX-200 for FSGS is currently in a Phase 2 clinical trial and has been granted Orphan Drug Designation by the FDA and EMA. Orphan Drug Designation is granted by the FDA and EMA in order to support the development of products for rare diseases. Orphan drug designation qualifies the sponsor of the drug for various development incentives by the FDA and EMA, including: seven years and ten years respectively of market exclusivity if regulatory approval is received, exemption from certain application fees, and an abbreviated regulatory pathway to approval.