Salanersen Receives FDA Breakthrough Status for Spinal Muscular Atrophy

Biogen has announced that the US Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation to salanersen, an investigational treatment for spinal muscular atrophy (SMA). The designation is intended to speed up the development and review of therapies that may offer significant advantages over existing treatments for serious diseases.

The decision was based on findings from a Phase 1b study in children with SMA who had previously received gene therapy. The study demonstrated clinically meaningful improvements in motor function and reductions in neurofilament light chain levels, a marker associated with neurodegeneration. Salanersen was also generally well tolerated.

Salanersen (BIIB115) is a novel antisense oligonucleotide designed to increase production of survival motor neuron (SMN) protein. Its high potency may enable once-yearly dosing.

Biogen is advancing salanersen through a global Phase 3 programme comprising three studies: STELLAR-1, SOLAR and STELLAR-2. These trials will evaluate the therapy across a broad range of SMA patients, including presymptomatic infants, adolescents and adults.

SMA is a rare genetic neuromuscular disorder that causes progressive muscle weakness and remains a leading genetic cause of infant mortality.