Biogen’s Qalsody wins European approval for rare form of ALS

2024-05-30

临床3期上市批准临床结果寡核苷酸

Biogen announced Thursday that the European Commission has approved Qalsody (tofersen) to treat amyotrophic lateral sclerosis (ALS) associated with mutations in the SOD1 gene, which is estimated to represent about 2% of patients.

The clearance, which was expected after the European Medicines Agency voiced its support for the SOD1-targeting antisense oligonucleotide in February, marks the second ALS treatment authorised in the EU along with Rilutek (riluzole) from Sanofi – and the first the first treatment targeting a genetic cause of ALS, according to the European Organisation for Professionals and People living with ALS.

The approval was based on data from the Phase III VALOR trial, which showed that Qalsody reduced mean plasma neurofilament light chain (NfL), a marker of axonal injury and neurodegeneration, by 55% at week 28, compared with a 12% increase in patients who received placebo.

The 108-patient study, however, missed the primary endpoint of improvement in the ALS Functional Ratings Scale-Revised total score. While Qalsody achieved a numerically higher mean change versus placebo, the results were not statistically significant

The European clearance comes a year after the FDA granted Qaslody accelerated approval in the same patient population. At the time, the US regulator said that the reductions in NfL were "reasonably likely to predict a clinical benefit in patients."

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