FDA accepts new drug application and grants priority review for treatment of ALS


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Biogen Inc. announced that the FDA has accepted a new drug application for tofersen, an investigational drug to treat superoxide dismutase 1 ALS.

According to a press release from the company, ALS superoxide dismutase 1 (SOD1) is a rare genetic form of the disease. While the average life expectancy for people with ALS is 3-5 years from the onset of symptoms, some patients with SOD1 survive less than a year.

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Biogen’s application for tofersen, which would be the first treatment to target a genetic cause of ALS, has been granted priority review and an action date under the Prescription Drug User Fee Act of January 25, 2023, according to the release. . The FDA plans to hold an advisory committee meeting for the application at a date yet to be determined.

“Available data show that tofersen has the potential to make a significant difference for people 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 statement. “Pursuing the FDA’s accelerated approval pathway provides the opportunity to make tofersen available to people living with this deadly neurodegenerative disease as quickly as possible.”

With no other treatments currently available for SOD1-ALS, Biogen said it is seeking approval under the FDA’s fast track, based on the neurofilament’s use as a biomarker of substitution that is reasonably likely to predict clinical benefit.

Results from a Phase 1 study in healthy volunteers, a Phase 1/2 study evaluating escalating dose levels, the Phase 3 VALOR study and the open-label extension study (OLE ) have been included in the NDA for tofersen. Also included are the most recent 12-month integrated results from VALOR and the OLE study, which were recently presented at the annual meeting of the European Network to Cure ALS.

According to the release, study results suggest reductions in neurofilament preceded and predicted slower declines in measures of clinical and respiratory function, strength and quality of life.

“The integrated 12-month results confirm the trends observed during the initial reading”, Toby Ferguson, MD, PhD, vice president and head of the neuromuscular development unit at Biogen, Healio told Healio. “These are essential measures for people living with this devastating disease. The disease is progressive, involves loss of daily functions and is uniformly fatal.

During the FDA review period, Biogen is expected to maintain its early access program for tofersen, which includes participants in more than a dozen countries. Phase 3 OLE and ATLAS studies in pre-symptomatic individuals with a SOD1 genetic mutation are ongoing, the company said.


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