New anti-ALS drug receives Orphan Drug Designation

A new gene therapy for treating an inherited form of Amyotrophic Lateral Sclerosis has been given an Orphan Drug Designation by the European Medicines Agency. The therapy is already in preclinical development at EPFL. "We are thrilled that the EMA endorses the soundness of our new therapy against this devastating rare disease," says Bernard Schneider, the EPFL scientist who leads the development of a new drug aimed at Amyotrophic Lateral Sclerosis (ALS). "We are highly motivated to bring our patented approach to the clinic and address this unmet patient need. We are grateful to the Swiss ALS Foundation and Catalyze4Life for helping us on this path." Amyotrophic lateral sclerosis (ALS), often referred to as "Lou Gehrig's Disease", is a rare neurodegenerative disorder that causes progressive muscle wasting and weakness, leading to death within 3-5 years. Although its exact causes are still unclear, we know that the disease involves a complex array of factors that affect different molecular pathways along the motor neural axis (brain-spinal cord-muscles). The European Medicines Agency (EMA) assigns Orphan Drug Designation to medicines that can be used for the diagnosis, prevention or treatment of a rare but life-threatening or chronically debilitating condition that affects no more than five in 10,000 people in the European Union.