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sodium phenylbutyrate/tauroursodeoxycholic acid; sodium phenylbutyrate/taurursodiol (Relyvrio, AMX0035)

Indications: - investigational treatmment for amyotrophic lateral sclerosis (ALS) * failed ALS drug, pulled from U.S. & Canadian market Clinical significance: - median time to first hospitalization > 64 weeks (median time for placebo) - median time to death or need for tracheostomy/permanent assisted ventilation 104 weeks vs 80 weeks for placebo [2]

General

pharmacologic combination neurologic agent

Database Correlations

PUBCHEM cid=129316491

References

  1. Paganoni S Trial of Sodium Phenylbutyrate - Taurursodiol for Amyotrophic Lateral Sclerosis. N Engl J Med 2020; 383:919-930. Sept 3 PMID: 32877582 https://www.nejm.org/doi/full/10.1056/NEJMoa1916945 - George J Early Data for ALS Drug Show Survival Benefit - Mortality cut 44% over follow-up period MedPage Today October 16, 2020 https://www.medpagetoday.com/neurology/generalneurology/89172
  2. Susman E Novel ALS Drug Continues to Show Survival Benefit. Median time to first hospitalization not yet reached with AMX0035 in CENTAUR trial. MedPage Today. April 19, 2021 https://www.medpagetoday.com/meetingcoverage/aan/92166 - Paganoni S et al Long-term survival of participants in the CENTAUR trial of AMX0035 for ALS. American Academy of Neurology (AAN) 2021
  3. Bagha S Amylyx pulls ALS drug Relyvrio from market in US, Canada. Healio. April 4, 2024 https://www.healio.com/news/neurology/20240404/amylyx-pulls-als-drug-relyvrio-from-market-in-us-canada

Components

phenylbutyrate (Buphenyl, Olpruva) tauroursodeoxycholic acid; tauroursodeoxycholate; taurursodiol; taurolite