雅虎香港財經 Europe Approves Biogen's Tofersen For Adult Patients With Rare Type Of Neurodegenerative Disorder
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.
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.
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