Biogen Inc. — Qalsody FDA Review (NDA215887, ORIG 1, 2023)
Biogen Inc. · 2023 · FDA Review (openFDA) · company profile →
Report summary
Biogen's Qalsody (tofersen) received accelerated FDA approval for SOD1-ALS based on significant reduction in plasma neurofilament light chain (NfL), a surrogate endpoint. While the primary clinical endpoint (ALSFRS-R) in Study 101C was not statistically significant, favorable trends were observed in open-label extension studies and post hoc analyses. Qalsody demonstrated an acceptable safety profile, though serious neurological events like myelitis, aseptic meningitis, and radiculitis were noted. Immunogenicity was observed, but without clear impact on efficacy or safety. Postmarketing requirements include a Phase 3 study (ATLAS) to verify clinical benefit in presymptomatic patients and carcinogenicity studies.
Key findings — cited to the page
| Priority or standard review | PRIORITY | Qalsody (tofersen) · p.2 |
| Received date | 5/25/2022 | Qalsody (tofersen) · p.2 |
| Application type | NDA | Qalsody (tofersen) · p.2 |
| Application number | 215887 | Qalsody (tofersen) · p.2 |
| Submit date | 5/25/2022 | Qalsody (tofersen) · p.2 |
| NDA number | 215887 | Qalsody (tofersen) · p.21 |
| Approval type | accelerated approval | Qalsody (tofersen) · p.21 |
| Indication | treatment of adults with amyotrophic lateral sclerosis (ALS) who have a mutation in the SOD1 gene (SOD1-ALS) | Qalsody (tofersen) · p.21 |
| NfL reduction after switching to active treatment | 44 % | · p.80 |
| NDA submission date | May 25, 2022 | Qalsody (tofersen) · p.179 |
| Regulatory action | Accelerated approval | Qalsody (tofersen) · p.2 |
| PDUFA goal date | 4/25/2023 | Qalsody (tofersen) · p.2 |
Source: Biogen Inc. ↗ · License: US Government public domain. Summary and findings are extracted from the published document; every figure cites its page. Informational only — not investment advice. Explore the Neurology library →