EMA grants PRIME status to Roche’s risdiplam for SMA

The European Medicines Agency has granted PRIority Medicines status to Roche’s investigational oral medicine risdiplam, for the treatment of the rare and debilitating genetic disease most commonly diagnosed in children, spinal muscular atrophy, and could conceivably become the first oral medicine for the treatment of spinal muscular atrophy types 1, 2 and 3. It is currently being investigated in three global, multicentre clinical trials in all types of the disease.
Sandra Horning, MD, Roche’s Chief Medical Officer and Head of Global Product Development, said: “SMA is the leading genetic cause of death in young children, and families and clinicians continue to seek alternative treatment options for this progressively debilitating and life-threatening disease. The EMA’s decision to grant PRIME designation recognises the potential of the oral systemic agent risdiplam to deliver clinically meaningful results for patients and address a continuing medical need in SMA.”