Biogen Gets FDA Priority Review of Tofersen in Rare Form of ALS

By Colin Kellaher

Biogen Inc. on Tuesday said the U.S. Food and Drug Administration granted priority review to its new drug application for tofersen for a rare, genetic form of amyotrophic lateral sclerosis, the fast-moving, paralyzing condition commonly known as Lou Gehrig's disease.

The Cambridge, Mass., drugmaker said the application covers tofersen in superoxide dismutase 1, or SOD1, ALS, for which there is currently no approved targeted treatment.

The FDA grants priority review to medicines that have the potential to provide significant improvements in the treatment of a serious disease, and the designation shortens the review period.

Biogen said that the FDA has set a target action date of Jan. 25, 2023, for the application, adding that the agency plans to hold an advisory committee meeting on date to be determined.

Biogen licensed tofersen from Carlsbad, Calif., pharmaceutical company Ionis Pharmaceuticals Inc. in 2018. The two companies have collaborated on neurology drugs since 2012.

Biogen said that mutations in the SOD1 gene are responsible for roughly 2% of the estimated 168,000 people who have ALS around the world, adding that while the average life expectancy for people with ALS is three to five years from time of symptom onset, patients with some SOD1 mutations have an even shorter life expectancy.

The company said it will maintain its early-access program for tofersen, which has participants in more than a dozen countries, during the FDA review period.

Write to Colin Kellaher at [e-mail]

(END) Dow Jones Newswires

July 26, 2022 08:17 ET (12:17 GMT)

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