On Thursday, the European Commission granted marketing authorization under exceptional circumstances and maintained orphan designation for Biogen Inc's (NASDAQ:BIIB) Qalsody (tofersen) for amyotrophic lateral sclerosis (ALS) associated with a mutation in the superoxide dismutase 1 gene (SOD1-ALS).
Qalsody is the first treatment approved in the European Union to target a genetic cause of ALS, also known as motor neuron disease (MND).
SOD1-ALS is an ultra-rare genetic form of ALS estimated to affect less than 1,000 people in Europe.
Qalsody is Biogen's third rare disease therapy to be approved in the EU.
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In the Phase 3 VALOR study (n=108), patients were randomized 2:1 to receive treatment with either Qalsody 100 mg (n=72) or placebo (n=36) for 24 weeks.
The primary efficacy endpoint was the change from baseline to Week 28 in the ALS Functional Ratings Scale-Revised total score.
The results numerically favored tofersen but were not statistically significant.
At Week 28, mean plasma neurofilament light chain (NfL), a marker of axonal injury and neurodegeneration, was reduced by 55% in the tofersen-treated participants (ITT), compared to a 12% increase with placebo.
About 330 people with SOD1-ALS have received Qalsody across 18 EU countries through the Biogen early access program.
Last April, the FDA granted accelerated approval for Qalsody. In 2023, the drug generated sales of $5.9 million.
Biogen licensed Qalsody from Ionis Pharmaceuticals Inc (NASDAQ:IONS) under a collaborative development and license agreement. Ionis discovered Qalsody.
In addition to the ongoing open-label extension of the Phase 3 VALOR study, Qalsody is being studied in the Phase 3 ATLAS study to evaluate whether Qalsody can delay clinical onset when initiated in presymptomatic individuals with a SOD1 genetic mutation and biomarker evidence of disease activity.
Price Action: BIIB shares are up 1.90% at $224.26 at the last check on Friday.