The European Commission has granted orphan drug designation to BrainStorm Cell Therapeutics’ stem cell therapy NurOwn to treat Amyotrophic Lateral Sclerosis (ALS), also known as Lou Gehrig's Disease.
NurOwn consists of autologous bone marrow-derived mesenchymal stromal cells secreting neurotrophic factors for the treatment of ALS and had earlier received orphan drug designation in the US.
At present, a Phase IIa dose-escalating trial with 12 ALS patients is being conducted at the Hadassah Medical Center in Jerusalem, Israel, with projected launch of a Phase II multi-center trial at three US institutions towards the end of 2013, pending FDA approval.
Source:
http://regulatoryaffairs.pharmaceutical-business-review.com/news/brainstorms-nurown-wins-orphan-drug-designation-in-europe-300713
