The European Commission (EC) has granted marketing authorisation for US-based biotechnology company Biogen’s Spinraza (nusinersen) for the treatment of 5q spinal muscular atrophy (SMA).

As the most common form of the disease, 5q SMA accounts for approximately 95% of all SMA cases.

The disease is a leading genetic cause of death in infants and is characterised by loss of motor neurons in the spinal cord and lower brain stem, resulting in severe and progressive muscular atrophy and weakness.

Biogen’s Spinraza is the first approved treatment for SMA in the European Union (EU) and was reviewed under the European Medicines Agency’s (EMA) accelerated assessment programme, focused on expediting access to patients with unmet medical needs.

The EC approval is mainly based on results from two major multicentre, controlled studies, including end-of-study data from ENDEAR (infantile-onset SMA) and an interim analysis of CHERISH (later-onset SMA).

"We are now seeing motor improvements with Spinraza that are never seen in the natural course of the disease."

The two studies confirmed the clinically meaningful efficacy and favourable benefit-risk profile of the Biogen treatment demonstrating increased survival in infants with SMA.

Also, the approval was supported by an open-label data in pre-symptomatic and symptomatic individuals with, or likely to develop, Types 1, 2 and 3 SMA.

Medical Center University of Germany Professor Dr Jan Kirschner said: “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.

“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.”

The therapy requires administration via an intrathecal injection that helps deliver therapies directly to the cerebrospinal fluid (CSF) around the spinal cord, where motor neurons degenerate in individuals with SMA due to insufficient levels of survival motor neuron (SMN) protein.