The FDA defines a "rare pediatric disease" as a disease that affects fewer than 200,000 individuals in the U.S. primarily aged from birth to 18 years. Under the FDA's Rare Pediatric Disease Priority Review Voucher program, a sponsor who receives an approval of a new drug application (NDA) or biologics license application (BLA) for a rare pediatric disease may be eligible for a voucher, which can be redeemed to obtain expedited FDA review for any subsequent marketing application. Vouchers may be sold or transferred by the recipient; in the last 6 months, 2 priority review vouchers have been sold for a combined $595M in cash.

In December 2015, Amarantus submitted ODD and RPDD applications to the US FDA for engineered skin substitute in the treatment of Giant Congenital Hairy Nevus (GCMN) , in addition to its ODD application to the US FDA for eltoprazine in the treatment of Parkinson's disease levodopa-induced dyskinesia (PD-LID) submitted in October 2015.

The Company expects to receive responses regarding these designation applications in the first quarter of 2016.
http://www.amarantus.com/news/press-releases/...ation-from

The FDA Orphan Drug Designation program provides a special status to drugs and biologics intended to treat, diagnose or prevent diseases and disorders that affect fewer than 200,000 people in the U.S. This designation provides for a seven-year marketing exclusivity period against competition, as well as certain incentives, including federal grants, tax credits and a waiver of PDUFA filing fees.
http://www.amarantus.com/news/press-releases/...rphan-drug