Klinische Studiendaten

Produkt: Qalsody™, Injektionslösung 100 mg
Wirkstoff: Tofersen
Zulassungsnummer: 68577 
Datum der Zulassung: 19.12.2025

Die Zulassungsstudien finden Sie hier:
VALOR (Part C): An Efficacy, Safety, Tolerability, Pharmacokinetics and Pharmacodynamics Study of BIIB067 (Tofersen) in Adults With Inherited Amyotrophic Lateral Sclerosis (ALS) (233AS101)

• Clinical Trials register - 2015-004098-33
• ClinicalTrials.gov - NCT02623699
• Publikationen:

  • Miller TM, Cudkowicz ME, Genge A, et al. Trial of Antisense Oligonucleotide Tofersen for SOD1 ALS. N Engl J Med. 2022;387(12):1099-1110.

  • Miller T, Cudkowicz M, Shaw PJ, et al. Phase 1-2 trial of antisense oligonucleotide tofersen for SOD1 ALS. N Engl J Med. 2020;383(2):109-119.

Long-Term Evaluation of BIIB067 (Tofersen) (233AS102)

• Clinical Trials register - 2016-003225-41
• ClinicalTrials.gov - NCT03070119
• Publikation: Miller TM, Cudkowicz ME, Genge A, et al. Trial of Antisense Oligonucleotide Tofersen for SOD1 ALS. N Engl J Med. 2022;387(12):1099-1110.

Produkt: Skyclarys™, Hartkapseln 50 mg
Wirkstoff: Omaveloxolone
Zulassungsnummer: 69610
Datum der Zulassung: 24.09.2024

Die Zulassungsstudien finden Sie hier:
MOXIe: A Phase 2 Study of the Safety, Efficacy, and Pharmacodynamics of RTA 408 in the Treatment of Friedreich’s Ataxia (1402)

• Clinical Trials register - 2015-002762-23
• Clinical Trial.gov - NCT02255435
• Publikation: Lynch DR, Chin MP, Delatycki MB, et al. Safety and Efficacy of Omaveloxolone in Friedreich Ataxia (MOXIe Study). Ann Neurol. 2021 Feb;89(2):212-225.