Release Date: 03-Sep-2021
Seelos Therapeutics receives orphan drug designation from The European Medicines Agency (EMA) for SLS-005 for the treatment of amyotrophic lateral sclerosis (ALS). In ALS, the accumulation of proteins such as TDP-43 and SOD1 in nerve cells can add to the development of the disease. Preclinical studies have showed that SLS-005 increases the clearance of these proteins, delaying the progression of ALS and preserving motor neurons and muscle fiber size.
SLS-005 contains of a natural sugar molecule, called trehalose, found in fungi, plants and bacteria. According to the studies SLS-005 helps autophagy, a cellular procedure that breaks down and recycles old and damaged proteins before they gather and become toxic. Also, SLS-005 can cross the blood-brain barrier andmdash; the semi-permeable protective layer that usually stops molecules from traveling to the central nervous system and causing damage and reach impaired nerve cells.
It is given to investigational therapies that have the power to treat rare and life-threatening diseases that affect less than five in 10,000 patients in the European Union. It provides some benefits to companies, like clinical protocol advice, fee reductions, and ten years of protection from competition following regulatory approval.