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An evaluation of the combination of sodium phenylbutyrate and taurursodiol for the treatment of amyotrophic lateral sclerosis.

Publication ,  Journal Article
Sun, Y; Li, X; Bedlack, R
Published in: Expert Rev Neurother
January 2023

INTRODUCTION: Amyotrophic lateral sclerosis (ALS) is a rapidly progressive, fatal neurodegenerative motor neuron disease. Despite the overwhelming need for effective therapeutics for ALS, riluzole and edaravone were the only two FDA-approved disease-modifying therapies prior to 2022. The randomized, double-blind, multicenter, placebo-controlled CENTAUR trial demonstrated the safety and efficacy of sodium phenylbutyrate-taurursodiol (PB-TURSO) in persons with ALS (PALS), leading to its conditional approval in Canada in June 2022 and full approval in the USA in September 2022. AREAS COVERED: Herein, the authors provide a review of the pharmacology and clinical trials evaluating sodium phenylbutyrate and/or taurursodiol in PALS. EXPERT OPINION: The safety and tolerability of both PB and TURSO were previously demonstrated in small PALS trials. The phase 2 CENTAUR study and its open-label extension demonstrated the safety and efficacy of AMX0035 (a sachet containing a fixed co-formulation of 3 g of PB and 1 g of TURSO given twice daily) in PALS. A phase 3 PHOENIX trial (NCT05021536) will offer more insight into safety and efficacy of AMX0035. AMX0035 currently costs $ 158,000 annually in the US, which may become a financial barrier for PALS to receive the medication.

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Published In

Expert Rev Neurother

DOI

EISSN

1744-8360

Publication Date

January 2023

Volume

23

Issue

1

Start / End Page

1 / 7

Location

England

Related Subject Headings

  • Randomized Controlled Trials as Topic
  • Neurology & Neurosurgery
  • Multicenter Studies as Topic
  • Humans
  • Edaravone
  • Amyotrophic Lateral Sclerosis
  • 3214 Pharmacology and pharmaceutical sciences
  • 3209 Neurosciences
  • 3202 Clinical sciences
  • 1115 Pharmacology and Pharmaceutical Sciences
 

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Sun, Y., Li, X., & Bedlack, R. (2023). An evaluation of the combination of sodium phenylbutyrate and taurursodiol for the treatment of amyotrophic lateral sclerosis. Expert Rev Neurother, 23(1), 1–7. https://doi.org/10.1080/14737175.2023.2174018
Sun, Yuyao, Xiaoyan Li, and Richard Bedlack. “An evaluation of the combination of sodium phenylbutyrate and taurursodiol for the treatment of amyotrophic lateral sclerosis.Expert Rev Neurother 23, no. 1 (January 2023): 1–7. https://doi.org/10.1080/14737175.2023.2174018.
Sun, Yuyao, et al. “An evaluation of the combination of sodium phenylbutyrate and taurursodiol for the treatment of amyotrophic lateral sclerosis.Expert Rev Neurother, vol. 23, no. 1, Jan. 2023, pp. 1–7. Pubmed, doi:10.1080/14737175.2023.2174018.
Journal cover image

Published In

Expert Rev Neurother

DOI

EISSN

1744-8360

Publication Date

January 2023

Volume

23

Issue

1

Start / End Page

1 / 7

Location

England

Related Subject Headings

  • Randomized Controlled Trials as Topic
  • Neurology & Neurosurgery
  • Multicenter Studies as Topic
  • Humans
  • Edaravone
  • Amyotrophic Lateral Sclerosis
  • 3214 Pharmacology and pharmaceutical sciences
  • 3209 Neurosciences
  • 3202 Clinical sciences
  • 1115 Pharmacology and Pharmaceutical Sciences