Biogen B V — Qalsody EMA EPAR public assessment report (EMEA/H/C/005493, 2024)
Biogen B V · 2024 · EMA EPAR public assessment report · company profile →
Report summary
The European Medicines Agency (EMA) granted Qalsody (tofersen) marketing authorization under exceptional circumstances for adults with SOD1-mutated Amyotrophic Lateral Sclerosis (ALS). Despite the primary endpoint of the pivotal 28-week study not reaching statistical significance, the CHMP issued a positive opinion based on consistent trends favoring tofersen in long-term data, particularly when adjusting for baseline neurofilament light chain (NfL) levels, and significant reductions in CSF SOD1 protein and plasma NfL. These biomarker changes support the drug's mechanism of action. Safety concerns include neuroinflammatory events like myelitis and radiculitis, along with common adverse reactions such as pain and fatigue. The overall benefit-risk balance was considered favorable given the rarity and severity of the disease, with ongoing studies planned to gather further long-term efficacy and safety data.
Key findings — cited to the page
| Tofersen manufacturing steps | 7 | · p.15 |
| QALSODY concentration | 6.7 mg/mL | QALSODY · p.18 |
| Revised CHMP positive opinion date | 19 April 2024 | Qalsody · p.11 |
| Number of diastereoisomers of tofersen | 32,768 | · p.15 |
| Tofersen active substance retest period | 24 months | · p.18 |
| QALSODY single dose | 100 mg | QALSODY · p.18 |
| Tofersen ED50 for SOD1 mRNA reduction in cervical cord of SOD1-G93A mice | 44 µg | · p.23 |
| CHMP positive opinion date | 22 February 2024 | Qalsody · p.11 |
| Marketing authorization application submission date | 31 October 2022 | Qalsody · p.8 |
| Tofersen recommended dose | 100 mg | · p.13 |
| Orphan medicinal product designation date | 29 August 2016 | Qalsody · p.8 |
| QALSODY pH | 7.2 | QALSODY · p.18 |
Source: Biogen B V ↗ · License: EMA - reuse permitted with acknowledgement. Summary and findings are extracted from the published document; every figure cites its page. Informational only — not investment advice. Explore the Neurology library →