Biogen has picked up European marketing authorisation for Spinraza, its spinal muscular atrophy (SMA) treatment.
Spinraza is the first approved treatment in the European Union for SMA, a leading genetic cause of death in infants that is marked by progressive, debilitating muscle weakness.
The treatment was reviewed under the European Medicines Agency’s (EMA) accelerated assessment program, intended to expedite access to patients with unmet medical needs.
“We believe Spinraza will have a meaningful impact on infants, children and adults living with this devastating disease,” said Michel Vounatsos, CEO of Biogen.
The approval is primarily based on positive results from two pivotal multicenter, controlled studies.
“The overall clinical findings support the efficacy and safety of Spinraza in a broad range of individuals with SMA, including significant improvements in motor development and reduction in risk of death in infants,” said Dr Jan Kirschner from the Medical Centre University of Freiburg, Germany.
He added: “These unprecedented improvements bring new hope to a community where there previously were no approved treatments available to address the loss of motor function over time.
“We are now seeing motor improvements with Spinraza that are never seen in the natural course of the disease.”