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.

How well do you really know your competitors?

Access the most comprehensive Company Profiles on the market, powered by GlobalData. Save hours of research. Gain competitive edge.

Company Profile – free sample

Thank you!

Your download email will arrive shortly

Not ready to buy yet? Download a free sample

We are confident about the unique quality of our Company Profiles. However, we want you to make the most beneficial decision for your business, so we offer a free sample that you can download by submitting the below form

By GlobalData
Visit our Privacy Policy for more information about our services, how we may use, process and share your personal data, including information of your rights in respect of your personal data and how you can unsubscribe from future marketing communications. Our services are intended for corporate subscribers and you warrant that the email address submitted is your corporate email address.

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.