NEW YORK – The European Commission has granted Biogen marketing authorization under exceptional circumstances for Qalsody (tofersen), making it the first drug approved in the EU to treat a genetic form of amyotrophic lateral sclerosis (ALS), the company said Thursday.
The EC grants marketing authorization under exceptional circumstances when it deems the benefit-risk assessment of a treatment to be positive, but due to the rarity of the disease, it's unlikely that comprehensive data can be obtained. In this case, Qalsody is designed as a treatment for a rare form of ALS that is associated with a mutation in the SOD1 gene and is estimated to affect less than 1,000 people in Europe.
The European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP) in February issued a positive opinion on Qalsody, an antisense oligonucleotide that binds to and helps degrade SOD1 mRNA to reduce the production of the SOD1 protein.
Qalsody's approval is based on a review of the totality of evidence, according to Biogen, including the targeted mechanism of action, biomarker, and clinical data.
In the randomized-controlled Phase III VALOR study, patients on Qalsody experienced a 55 percent decrease in plasma neurofilament light (NfL), a blood-based biomarker associated with axonal injury and neurodegeneration, after 28 weeks as compared to baseline, while a 12 percent increase in NfL was observed in the placebo group. There were also trends suggesting that Qalsody improved patients' physical abilities based on the ALS Functional Rating Scale-Revised, although the outcomes were not statistically significant.
Common adverse events that may affect more than 1 in 10 people treated with Qalsody were fever, feeling tired, an increase in protein or white blood cell count occurring in the fluid that surrounds the brain and spinal cord, and pain in the back, arms, legs, muscles, or joints.
Qalsody received accelerated approval from the US Food and Drug Administration last year, and Cambridge, Massachusetts-based Biogen said it is continuing to engage with regulatory authorities to bring Qalsody to other regions.