FDA grants Breakthrough Therapy status to Biogen salanersen for spinal muscular atrophy
Children with spinal muscular atrophy who responded poorly to gene therapy may have a new option. The FDA has granted Breakthrough Therapy status to Biogen $BIIB salanersen, an experimental once-yearly injection that improved motor function and slowed nerve loss in early data. An
Children with spinal muscular atrophy who have responded poorly to gene therapy may have a new treatment option on the horizon. On June 4 the US Food and Drug Administration granted Breakthrough Therapy Designation to salanersen, an investigational drug from Biogen $BIIB. Salanersen is an antisense oligonucleotide, a type of therapy that adjusts how a gene is read, and it is designed to be given just once a year. That dosing schedule stands out in a field where existing treatments often require more frequent administration. Spinal muscular atrophy is a genetic disorder that damages the nerve cells controlling muscle movement, and in its more severe forms it appears in infancy. According to Biogen, children with SMA who had a suboptimal response to earlier gene therapy showed clinically meaningful improvements in motor function after starting once-yearly salanersen, along with slower neurodegeneration as measured by reduced neurofilament levels, a marker of nerve damage. The Breakthrough Therapy pathway is used by the FDA to speed development of drugs that target serious conditions and show early promise over available options. Salanersen is now in a Phase 3 program of three global studies, with the third expected to begin enrolling patients in June. Reported by Biogen and the FDA.