Biogen receives accelerated approval for first-of-its-kind ALS treatment

Biogen has announced that they have received approval from the US FDA for the amyotrophic lateral sclerosis (ALS) treatment, QALSODY™ (tofersen). This is the first approval of a treatment that targets a genetic cause of ALS. 

Final results from the ATLAS Phase III trial studying the use of tofersen for people with presymptomatic SOD1-ALS will help to confirm the mid-trial results and be used to establish full approval. 

“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,” stated Christopher A. Viehbacher, President and Chief Executive Officer of Biogen. “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.”

The drug does come with the potential for some serious adverse reactions, including myelitis and/or radiculitis; papilledema and elevated intracranial pressure; and aseptic meningitis. The most common of these however occurred in less than 10% of patients treated with QALSODY. 

“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,” commented Jean Swidler, chair of Genetic ALS & FTD: End the Legacy. “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.”

The efficacy of the treatment was measured in a randomised, double-blind, placebo-controlled clinical trial, – VALOR – spanning 28 weeks. The study included 108 patients with 72 patients receiving 100 mg dose of QALSODY, and 36 patients receiving a placebo. Patients in the testing cohort demonstrated less decline in function according to the Revised Amyotrophic Lateral Sclerosis Functional Rating Scale, although the results were not considered significant against the control group. 

“I have observed the positive impact QALSODY has on slowing the progression of ALS in people with SOD1 mutations,” said Timothy M. Miller, MD, PhD, principal investigator of the QALSODY clinical trials and co-director of the ALS Center at Washington University School of Medicine in St. Louis. “The FDA’s approval of QALSODY gives me hope that people living with this rare form of ALS could experience a reduction in decline in strength, clinical function, and respiratory function.”