European regulators have assigned orphan drug status to an experimental therapy for Amyotrophic Lateral Sclerosis (ALS) called masitinib, indicating that it has the potential to offer a significant benefit over existing treatment options.
ALS, also referred to as Lou Gehrig's disease, is a neuromuscular condition that attacks nerve cells in the brain and spinal cord responsible for controlling voluntary muscles, such as those needed to move, speak, eat and breathe.
The condition is inherited in 5 percent to 10 percent of cases, but the cause for the majority of cases is not well understood, and there is currently no cure.
Masitinib, which is being developed by French group AB Science, is an orally administered tyrosine kinase inhibitor targeting mast cells and macrophages, important cells for immunity, by inhibiting a limited number of kinases.
According to the firm, winning orphan status in the EU is a significant milestone because it means that the Committee for Orphan Medicinal Products considered the drug in combination with Riluzole generated significant benefit over Riluzole alone, based on the results of the interim analysis of the on-going Phase II/III trial.