FDA accepts new drug application for Tofersen as treatment for SOD1-ALS


Currently, there is no targeted treatment for superoxide dismutase 1 amyotrophic lateral sclerosis, which has a life expectancy of 3 to 5 years from the onset of symptoms.

The FDA has accepted a new drug application (NDA) for tofersen (Biogen), an investigational drug that treats amyotrophic lateral sclerosis (ALS) with superoxide dismutase 1 (SOD1).

The application was granted priority review status and a January 25, 2023 Prescription Drug User Fee Act date. The FDA said it currently plans to hold an advisory committee meeting for the application, but the date has not yet been determined.

Currently, there is no targeted treatment for SOD1-ALS, which has a life expectancy of 3 to 5 years from the onset of symptoms, according to a statement released by Biogen.

“The available data shows that tofersen has the potential to make a significant difference for [patients] with SOD1-ALS,” Priya Singhal, MD, MPH, head of global safety and regulatory science and interim head of research and development at Biogen, said in the release. “Pursuing the FDA’s accelerated approval pathway provides the opportunity to make tofersen available to [individuals] live as quickly as possible with this fatal neurodegenerative disease. If approved, tofersen will be the first treatment to target a genetic cause of ALS and we hope this will pave the way for further advances in this relentless disease.

Biogen is seeking approval under the FDA’s Accelerated Approval Process due to the use of neurofilament as a surrogate biomarker that is reasonably likely to predict clinical benefit.

Neurofilaments are a normal protein in healthy neurons that are increased in blood and cerebrospinal fluid when damage has been done to neurons or their axons. They are a marker of neurodegeneration, and in ASL, higher levels of neurofilament shaving have been shown to predict more rapid decline in measures of clinical and respiratory function, strength, and quality of life.

Biogen will commit to generating ongoing data and finalizing the confirmatory dataset with the FDA.

“The 12-month results showed that people with SOD1-ALS who started tofersen earlier experienced a slower decline in clinical and respiratory function, strength and quality of life. These are essential steps to [individuals] living with this devastating disease,” Timothy Miller, MD, PhD, VALOR principal investigator and co-director of the ALS Center at Washington University School of Medicine, St. Louis, said in a statement. “For [patients] in my clinic living with SOD1-ALS, tofersen can significantly slow the rapid progression of their disease and the impact it has on their lives.

The NDA for tofersen included results from a Phase 1 study in healthy individuals, a Phase 1/2 study evaluating escalating dose levels, the Phase 3 VALOR study and the extension study in open. Additionally, researchers have included the most recent 12-month integrated results from VALOR and the open-label extension study, which were recently presented at the annual meeting of the European Network to Cure ASL.

Although the VALOR study did not meet the primary endpoint of change from baseline to week 28 in the Amyotrophic Lateral Sclerosis Revised Functional Rating Scale, trends of reduced disease progression on several secondary and exploratory endpoints were observed.

In the 12-month data, the researchers found that earlier initiation of tofersen resulted in long-lasting neurofilament reduction. Adverse events in the VALOR study and the open-label extension study were headache, procedural pain, falls, back pain, and pain in extremity, which were generally mild to moderate in severity .

Reference

FDA accepts Biogen’s New Drug Application and grants priority review of tofersen for a rare genetic form of ALS. Press release. Biogenic. July 26, 2022. Accessed July 26, 2022. http://media.biogen.com/news-releases/news-release-details/fda-accepts-biogens-new-drug-application-and-grants-priority

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