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July 27, 2022

FDA grants priority review for Biogen’s tofersen to treat ALS

The NDA for tofersen comprised data from a Phase I trial, a Phase I/II trial, a Phase III VALOR trial and an OLE trial.

The US Food and Drug Administration (FDA) has granted priority review for Biogen’s New Drug Application (NDA) for investigational drug, tofersen, to treat superoxide dismutase 1 (SOD1) amyotrophic lateral sclerosis (ALS). 

A decision from the regulatory agency on approval for the drug is anticipated on 25 January next year. 

The FDA also plans to hold an advisory committee meeting for the NDA. 

The application for the drug comprised data from a Phase I clinical trial in healthy subjects, a Phase I/II trial analysing ascending dose levels, the Phase III VALOR trial and an open-label extension (OLE) trial. 

It also included the latest one-year combined data from VALOR and the OLE study.

The six-month, randomised Phase III VALOR trial failed to meet the primary endpoint of variation in the Revised Amyotrophic Lateral Sclerosis Functional Rating Scale from baseline to week 28.

But trends of lowered disease progression across various secondary and exploratory endpoints were reported. 

Headache, fall, back pain, procedural pain and pain in extremities were the most frequent adverse events (AEs) reported in subjects who received tofersen in VALOR and the OLE study.

Furthermore, 6.7% of subjects who received tofersen in VALOR and OLE studies had serious neurologic events including myelitis, aseptic meningitis, radiculitis and papilledema.

Currently, the open-label extension and Phase III ATLAS trials in presymptomatic people with a SOD1 genetic mutation are underway. 

An antisense drug, tofersen is being analysed to potentially treat SOD1-ALS. 

Under a development and licence agreement, Biogen obtained licence for tofersen from Ionis Pharmaceuticals.

Biogen Global Safety and Regulatory Sciences head and R&D interim head Priya Singhal said: “The available data show that tofersen has the potential to make a meaningful difference for people with SOD1-ALS. 

“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.”

In June, the company signed a licence and collaboration agreement with Alectos Therapeutics for developing and marketing AL01811 to treat Parkinson’s Disease.

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