Biogen, Ionis win FDA nod for ALS therapy Qalsody
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Update 03:11 PM EST: Adds comments from Biogen (NASDAQ:BIIB)
The FDA announced Tuesday the approval of Qalsody (tofersen) developed by Biogen (BIIB) and Ionis Pharmaceuticals (NASDAQ:IONS) for certain patients with neurodegenerative disease amyotrophic lateral sclerosis (ALS).
Specifically, the FDA has cleared Qalsody for ALS linked to a mutation in the superoxide dismutase 1 (SOD1) gene, which accounts for about 2% of ALS cases.
Qalsody is an antisense oligonucleotide administered through a spinal injection by healthcare professionals with experience in lumbar punctures.
In the U.S., 16K – 32K patients have ALS, and the FDA estimates that those with the SOD1-ALS gene mutation number less than 500.
The agency has cleared Qalsody based on its accelerated approval pathway, under which the developers are required to prove the clinical benefit of an approved medicine through confirmatory studies.
Issuing a statement following the approval, Biogen (BIIB) said Tuesday that Qalsody shipments would begin in about a week for U.S. healthcare providers, and ATLAS, its ongoing confirmatory trial for the treatment, is scheduled for primary completion in 2026.
In March, an expert panel of the FDA issued mixed views on the companies’ marketing application for tofersen in ALS.