The VALOR trial (Study 1 Part C) lasted 28 weeks, and the results were analyzed at the end of the study. At the end of VALOR, all remaining participants were given the opportunity to continue in the open-label extension (OLE) study. All participants enrolled in the OLE study were treated with QALSODY.
There was an initial analysis of the OLE study when all participants had the opportunity to complete 52 weeks of treatment. This allowed the researchers to compare results of participants who started QALSODY at the start of the VALOR study to those who only received QALSODY later during the OLE study.
VALOR studied the effectiveness and safety of QALSODY compared to placebo in people with weakness due to amyotrophic lateral sclerosis (ALS) who have a mutation, or change, in the superoxide dismutase 1 (SOD1) gene.
A total of 108 people with SOD1-ALS participated in VALOR
Participants received treatment for 24 weeks (3 loading doses followed by 5 maintenance doses).
Participants were allowed to also take riluzole and/or edaravone during the study.
The faster progressing group was the Primary analysis population that included 60 participants who met the following conditions:
The slower progressing group included 48 participants who met the following criteria:
Baseline disease characteristics
The primary outcome, or main question VALOR was designed to answer, was:
In participants who met the criteria for faster progressing disease, how much did their ALSFRS-R scores change at 28 weeks compared to the start of the study?
Neurofilament light chain, or NfL, is a blood-based biomarker of nerve damage and deterioration.
QALSODY® (tofersen) is a prescription medicine used to treat adults with amyotrophic lateral sclerosis (ALS) who have a mutation, or change, in the superoxide dismutase 1 (SOD1) gene. QALSODY is approved under accelerated approval based on reduction in NfL in the blood observed in patients treated with QALSODY. Continued approval for QALSODY may depend on results of additional studies to confirm that there is a clinical benefit.
After 28 weeks, NfL levels in the blood decreased an average of 55% (Geometric mean ratio to baseline) from the start of the study in the QALSODY-treated participants and increased by 12% (Geometric mean ratio to baseline) from the start of the study in the placebo group. The Difference in geometric mean ratios between the QALSODY-treated participants and the placebo group was 60%.
NfL levels in the blood declined for approximately 113 days, then the reductions leveled off.
Difference in geometric mean ratios: 60% (95% CI): (0.33, 0.49)
Continued approval for QALSODY may depend on results of additional studies to confirm that there is a clinical benefit.
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