FDA Grants breakthrough therapy designation to Biogen’s salanersen for SMA

Biogen (NASDAQ:BIIB) announced today that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation to its investigational antisense oligonucleotide, salanersen, for the treatment of spinal muscular atrophy (SMA).

The designation is intended to expedite the development and regulatory review of drugs that show preliminary clinical evidence of substantial improvement over existing therapies for serious conditions.

The FDA’s decision is supported by data from a Phase 1b study involving children diagnosed with SMA who had shown a suboptimal response to prior gene therapy.

In the trial, treatment with salanersen resulted in meaningful motor function gains and a reduction in neurofilament levels—a biomarker often used to indicate nerve cell damage and disease progression.

Furthermore, the drug—which is administered on a once-yearly dosing schedule—was reported to be generally well-tolerated by participants.

Biogen is now positioning salanersen for a broad global Phase 3 clinical program.