The NurOwn technology processes adult human mesenchymal stem cells present in bone marrow which can self-renew and differentiate into many other cell types.
The orphan status entitles BrainStorm to seven years of marketing exclusivity for NurOwn after regulatory approval, in addition to the opportunity of applying for grant funding from the US government to defray costs of clinical trial expenses, tax credits for clinical research expenses and potential exemption from the FDA’s application user fee.
BrainStorm president Chaim Lebovits said the orphan drug designation will help the company advance NurOwn as a potential new treatment for ALS, for which human clinical trials are expected to start during the first half of 2011.