FDA grants accelerated approval for QALSODY (tofersen) as ALS treatment

pharmafile | April 26, 2023 | News story | Medical Communications  

Biogen has announced that the US Food and Drug Administration (FDA) has approved QALSODY (tofersen) 100mg/15ml injection for the treatment of amyotrophic lateral sclerosis (ALS) in adults with a mutation in the superoxide dismutase 1 (SOD1) gene.

The drug has been approved under the accelerated approval pathway, based on a reduction in plasma neurofilament light chain (NfL) that was demonstrated in patients treated with QALSODY. Confirmatory trials may be required to continue the approval of the drug for this indication.

Christopher A Viehbacher, president and chief executive officer of Biogen, commented: “For more than a decade, Biogen has been steadfast in our commitment to pursuing treatments for ALS, and I want to thank the scientists as well as the entire ALS community who have all worked tirelessly to bring this first-of-its-kind treatment to people with SOD1-ALS. Today also marks a pivotal moment in ALS research as we gained, for the first time, consensus that neurofilament can be used as a surrogate marker reasonably likely to predict clinical benefit in SOD1-ALS. We believe this important scientific advancement will further accelerate innovative drug development for ALS.”

Jean Widler, chair of Genetic ALS & FTD, added: “Since SOD1 mutations were first identified as a cause of ALS 30 years ago, the familial ALS community has been searching for genetically targeted treatments. QALSODY offers families who have lost generation after generation in the prime of their life to this devastating disease a therapy targeting the underlying cause of SOD1-ALS. Today marks an important moment in ALS research as QALSODY is the first ALS treatment approved based on a biomarker. We are excited to see what future therapies are developed now that it is understood that lowering levels of neurofilament provides important evidence that a treatment is affecting the neurodegenerative process.”


Betsy Goodfellow

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