Drug updated on 11/5/2024
Dosage Form | suspension (oral; sodium phenylbutyrate 3 g, taurursodiol 1 g) |
Drug Class | Unknown |
Ongoing and Completed Studies | ClinicalTrials.gov |
Indication
- Indicated for the treatment of amyotrophic lateral sclerosis (ALS) in adults.
Latest News
Summary
- This summary is based on the review of two randomized controlled trial(s). [1-2]
- Functional Decline (ALSFRS-R Score): Sodium phenylbutyrate-taurursodiol (PB-TURSO) slowed the decline in the ALS Functional Rating Scale-Revised (ALSFRS-R) score over 24 weeks compared to placebo, with a mean rate of change of -1.24 points per month for the active drug versus -1.66 points per month for placebo. The difference was 0.42 points per month (95% Confidence Interval (CI), 0.03 to 0.81; P = 0.03).
- Survival: Median overall survival was 25.0 months for participants randomized to PB-TURSO, compared to 18.5 months for placebo, reflecting a 6.5-month longer survival for the PB-TURSO group (hazard ratio, 0.56; 95% CI, 0.34-0.92; P = 0.023).
- Secondary Outcomes: There were no significant differences between PB-TURSO and placebo in secondary outcomes, including isometric muscle strength, plasma phosphorylated neurofilament levels, slow vital capacity, and time to death or hospitalization.
- Adverse Events: Adverse events associated with PB-TURSO were predominantly gastrointestinal in nature.
- There is no information on significant safety concerns or adverse effects in specific population types or subgroups in the reviewed documents.
- There is no population types or subgroups information available in the reviewed documents.
Product Monograph / Prescribing Information
Document Title | Year | Source |
---|---|---|
Relyvrio (sodium phenylbutyrate and taurursodiol) Prescribing Information. | 2022 | Amylyx Pharmaceuticals, Inc., Cambridge, MA |
Randomized Controlled Trials
Document Title | Sex Distribution | Year | Source |
---|---|---|---|
Long-term survival of participants in the CENTAUR trial of sodium phenylbutyrate-taurursodiol in amyotrophic lateral sclerosis | 135Subjects F: 31% M: 69% | 2021 | Muscle & Nerve |
Trial of Sodium Phenylbutyrate-Taurursodiol for Amyotrophic Lateral Sclerosis | 135Subjects F: 31% M: 69% | 2020 | The New England Journal of Medicine |
Document Title
Sex Distribution:
F:31%
M:69%
135Subjects
Year:
2021
Source:Muscle & Nerve
Sex Distribution:
F:31%
M:69%
135Subjects
Year:
2020
Source:The New England Journal of Medicine