Tofersen for SOD-1-associated amyotrophic lateral sclerosis

Tofersen was one of the first authorisation approvals to be included in the EMA Raw Data Project, which was launched in 2022 to assess whether analysis of raw data from clinical trials by regulatory authorities improves the evaluation of marketing authorisation applications for new medicines. NfL co...

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Published inLancet neurology Vol. 23; no. 8; pp. 772 - 773
Main Authors Haberkamp, Marion, Aislaitner, Georgios, Martínez-Lapiscina, Elena H, Weise, Martina
Format Journal Article
LanguageEnglish
Published England Elsevier Ltd 01.08.2024
Elsevier Limited
Subjects
Amyotrophic lateral sclerosis
Amyotrophic Lateral Sclerosis - genetics
Biomarkers
Clinical trials
Committees
Consortia
Data analysis
Disease
Drug development
Humans
Marketing
Muscle strength
Mutation
Natural history
Proteins
Superoxide dismutase
Superoxide Dismutase-1 - genetics
Trends
Ventilation
United States > US
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Summary:Tofersen was one of the first authorisation approvals to be included in the EMA Raw Data Project, which was launched in 2022 to assess whether analysis of raw data from clinical trials by regulatory authorities improves the evaluation of marketing authorisation applications for new medicines. NfL concentrations can be measured in blood or CSF and are considered prognostic for disease progression. [...]NfL has been discussed as a reasonably likely surrogate endpoint in early established disease, and the EXPERTS-ALS programme in the UK announced that NfL levels will be utilised to help screen potential drugs for ALS and to select the best candidates to advance to phase 3 trials. [...]numerically larger effects of tofersen versus placebo were observed in patients with baseline NfL values above versus below median, suggesting larger effects in faster progressing patients. The Committee for Human Medicinal Products concluded that available data on safety and efficacy were not comprehensive enough to support a full marketing authorisation, and that such data cannot be generated under normal conditions of use, because SOD1-associated ALS is encountered so rarely (the reported prevalence is approximately 1000 cases in the EU). [...]based on the totality of evidence, marketing authorisation under exceptional circumstances was recommended.
Bibliography:ObjectType-Article-2
SourceType-Scholarly Journals-1
ObjectType-Correspondence-1
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ISSN:1474-4422
1474-4465
1474-4465
DOI:10.1016/S1474-4422(24)00259-X