Amarantus BioScience Holdings, Inc., a biotechnology company focused on developing products for regenerative medicine, neurology and orphan diseases, announced that it has received orphan drug designation from the US FDA for Eltoprazine in the treatment of Parkinson's disease levodopa-induced dyskinesia (PD-LID).

Amarantus published positive results from a phase 2 initial proof-of-concept clinical study in February of 2015 in the journal Brain, and highlighted the publication of two independent peer-reviewed scientific publications describing the mechanism of action of eltoprazine for the treatment of PD LID in August of 2015 and December 2016.

"The grant of this orphan drug designation for eltoprazine in PD-LID squarely positions Amarantus as an orphan drug company, as each of our pipeline candidates in our therapeutics division has received such designations from the FDA for one or more indications," said Gerald E. Commissiong, president & CEO of Amarantus. "PD-LID is a tremendously debilitating disorder, and we will now begin evaluating expedited pathways to market for eltoprazine that may now be afforded by the orphan drug designation."

The FDA Orphan Drug Designation programme provides a special status to drugs and biologics intended to treat, diagnose or prevent so-called orphan diseases and disorders that affect fewer than 200,000 people in the US. 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.

Parkinson's disease is a chronic, progressive motor disorder that causes tremors, rigidity, slowed movements and postural instability. The Parkinson's Disease Foundation estimates that there were approximately one million people living with Parkinson's disease in the United States in 2011. The most commonly-prescribed treatments for Parkinson's disease are levodopa-based therapies. In the body, levodopa is converted to dopamine to replace the dopamine loss caused by the disease. The therapeutic efficacy of levodopa is gradually lost over time, and abnormal involuntary movements, dyskinesias, gradually emerge as a prominent side-effect in response to previously beneficial doses of the drug. Levodopa-induced dyskinesia can be severely disabling, rendering patients unable to perform routine daily tasks.

Eltoprazine is a small molecule 5HT1A/1B partial agonist in clinical development for the treatment of Parkinson's disease levodopa-induced dyskinesia (PD-LID), adult attention deficit hyperactivity disorder (ADHD) and Alzheimer's aggression. Eltoprazine has been evaluated in over 680 human subjects to date, and has a well-established safety profile. Eltoprazine was originally developed by Solvay Pharmaceuticals for the treatment of aggression. Upon Solvay's merger with Abbott Pharmaceuticals, the eltoprazine program was out-licensed to PsychoGenics. PsychoGenics licensed eltoprazine to Amarantus following successful proof-of-concept trials in PD-LID and adult ADHD.