Biogen Gets FDA Priority Review of Tofersen in Rare Form of ALS
July 26 2022 - 7:32AM
Dow Jones News
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 colin.kellaher@wsj.com
(END) Dow Jones Newswires
July 26, 2022 08:17 ET (12:17 GMT)
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