FDA accepts new drug application, grants priority review for ALS treatment
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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 company press release, superoxide dismutase 1 (SOD1) ALS is a rare, genetic form of the disease. While average life expectancy for people with ALS is 3 to 5 years from symptom onset, some patients with SOD1 ALS survive less than a year.
Biogen’s application for tofersen, which would be the first treatment to target a genetic cause of ALS, was granted priority review and given a Prescription Drug User Fee Act action date of Jan. 25, 2023, according to the release. The FDA plans to hold an advisory committee meeting for the application on a yet-to-be determined date.
“The available data show that tofersen has the potential to make a meaningful difference for people with SOD1-ALS,” Priya Singhal, MD, MPH, head of global safety and regulatory sciences and interim head of research and development at Biogen, said in the release. “Pursuing the FDA’s accelerated approval pathway offers the potential to make tofersen available to people living with this fatal, 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 accelerated approval pathway, based on the use of neurofilament as a surrogate biomarker that is reasonably likely to predict clinical benefit.
Results from a phase 1 study in healthy volunteers, a phase 1/2 study evaluating ascending dose levels, the phase 3 VALOR study and the open-label extension (OLE) study were included in the NDA for tofersen. Also included were the most current 12-month integrated results from VALOR and the OLE study, which were recently presented at the European Network to Cure ALS annual meeting.
According to the release, study results suggest reductions in neurofilament preceded and predicted slowing of decline in measures of clinical and respiratory function, strength and quality of life.
“The 12-month integrated results support the trends seen at the initial readout,” Toby Ferguson, MD, PhD, vice president and head of the Neuromuscular Development Unit at Biogen, told Healio. “These are critical measures for people living with this devastating disease. The disease is progressive, leads to the loss of everyday 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. The OLE and phase 3 ATLAS studies in pre-symptomatic individuals with a SOD1 genetic mutation are ongoing, the company stated.