Sodium Phenylbutyrate
Regulatory milestones approvals, filings & regulatory actions · 4 recorded
| Milestone | Jurisdiction | Brand | Indication | Date | Sentence it was read from |
|---|---|---|---|---|---|
| Label expansion | US (FDA) | OLPRUVA | — | 2025-10-10 | fda.gov ↗ |
| Refused (EMA) | EU (EMA) | Albrioza | — | 2024-01-05 | europa.eu ↗ |
| Approved | EU (EMA) | Ammonaps | — | 1999-12-07 | europa.eu ↗ |
| Approved | US (FDA) | BUPHENYL | — | 1996-04-30 | fda.gov ↗ |
Trials 37 · a red edge is where a trial was stopped
Also used as a comparator or background therapy in 13 trials
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 11 trial | ||||||
| Phase 1 | NCT00977600 Comparator | Mar → Jul 2005 | — | Amgen | Completed | No outcome recorded |
| Phase 1/24 trials | ||||||
| Phase 1/2 | NCT00528268 Comparator | Jul 2007 → Dec 2013 | spinal muscular atrophy | University of Utah | Completed | No outcome recorded |
| Phase 1/2 | NCT00590538 Comparator | Feb 2003 → Dec 2008 | cystic fibrosis | Children's Hospital of Philadelphia | Terminated | No outcome recorded |
| Phase 1/2 | NCT00016744 Comparator | Sep 2001 → Oct 2005 | cystic fibrosis | Children's Hospital of Philadelphia | Completed | No outcome recorded |
| Phase 1/2 | NCT00002796 Background | May 1997 → Dec 2004 | colon mucinous adenocarcinoma, colon signet ring cell adenocarcinoma, rectal signet ring cell adenocarcinoma, rectum mucinous adenocarcinoma | National Cancer Institute (NCI) | Terminated | No outcome recorded |
| Phase 25 trials | ||||||
| Phase 2 | NCT03533257 Comparator | Sep 2018 → Oct 2020 | Alzheimer disease | Amylyx Pharmaceuticals Inc. | Completed | No outcome recorded |
| Phase 2 | NCT01702974 Comparator | Sep 2012 → Aug 2015 | HIV infectious disease | Karolinska Institutet | Completed | No outcome recorded |
| Phase 2 | NCT01698476 Comparator | Sep 2012 → Aug 2015 | pulmonary tuberculosis | Karolinska Institutet | Completed | No outcome recorded |
| Phase 2 | NCT01580007 Comparator | Dec 2010 → Dec 2014 | pulmonary tuberculosis | International Centre for Diarrhoeal Disease Research, Bangladesh | Completed | No outcome recorded |
| Phase 2 | NCT00551200 Comparator | Oct 2007 → Jul 2008 | urea cycle disorder | Amgen | Completed | No outcome recorded |
| Phase 2/31 trial | ||||||
| Phase 2/3 | NCT01529060 Comparator | Feb 2013 → Sep 2017 | maple syrup urine disease | Brendan Lee | Completed | No outcome recorded |
| Phase 41 trial | ||||||
| Phase 4 | NCT03335488 Comparator | Feb 2018 → Jul 2022 | urea cycle disorder | Amgen | Completed | No outcome recorded |
| Phase not applicable1 trial | ||||||
| — | NCT02111200 Comparator | Sep 2014 → Oct 2015 | urea cycle disorder | Baylor College of Medicine | Completed | No outcome recorded |
Evidence & citations 15 cited values
Every value below carries the sentence it was read from. 51 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | sodium phenylbutyrate | “The purpose of this study is to evaluate the safety, tolerability and clinical impact of 15-grams daily of sodium phenylbutyrate (phenylbutyrate) in Huntington's disease and to...” NCT00212316 ↗30“We, recently, showed that stimulating BCAA oxidation for 2 weeks with sodium-phenylbutyrate (NaPB) treatment -a drug known to accelerate BCAA oxidation- decreased BCAA plasma...” NCT05836350 ↗ “Our study seeks to investigate the potential small molecule inhibition of the kinase that regulates BCKDC by applying a novel activity of sodium phenylbutyrate (NaPBA), in MSUD.” NCT01529060 ↗ “Dose of interventions: 5,000 IU of vitamin D (cholecalciferol tablets) once daily and 500 mg PBA (sodium phenylbutyrate tablets) twice daily for 16 weeks.” NCT01698476 ↗ “Dose of interventions: 5,000 IU of vitamin D (cholecalciferol tablets) once daily and 500 mg PBA (sodium phenylbutyrate tablets) twice daily for 16 weeks.” NCT01702974 ↗ “The purpose of this study is to determine whether sodium phenylbutyrate can reduce Lcn2 urinary expression in proteinuric patients.” NCT02343094 ↗ “Oral sodium phenylbutyrate (Buphenyl®) equivalent to 3 g/m2 of 4-phenylbutyric acid (PBA) per dose” NCT00977600 ↗ “AMX0035 twice daily--a combination of Sodium Phenylbutyrate (3g) and Taurursodiol (1g)” NCT03533257 ↗ “Tauroursodeoxycholic Acid and Sodium Phenylbutyrate orally given twice a day” NCT04516096 ↗ “oral AMX0035 (sodium phenylbutyrate + taurursodiol) versus placebo” NCT07699380 ↗ “Fixed dose combination of Sodium Phenylbutyrate and taurursodiol.” NCT04987671 ↗ “Sodium Phenylbutyrate (3g) and Taurursodiol (1g)” NCT03488524 ↗ “oral sodium phenylbutyrate” NCT00002909 ↗ |
| Known as | 4-phenylbutyrate | “This is a single patient compassionate use protocol to determine whether Buphenyl (4-phenylbutyrate) will improve the poor bile flow associated with Byler Disease.” NCT01784718 ↗ |
| Known as | 4-phenylbutyric acid, sodium salt | “500 mg sodium phenylbutyrate (4-phenylbutyric acid, sodium salt) in tablet form twice daily and 5000 IU of cholecalciferol once daily will be given orally for 2 months” NCT01580007 ↗ |
| Known as | 4PBA | “Sodium 4-Phenylbutyrate (4PBA)” NCT00016744 ↗ |
| Known as | ACER-001 | “The medication is sodium phenylbutyrate (ACER-001), which is currently FDA approved for the treatment of Urea Cyle Disorders.” NCT06069375 ↗ |
| Known as | Buphenyl | “Buphenyl is currently approved for the treatment of rare inherited disorders of the urea cycle.” NCT00533559 ↗ |
| Known as | Buphenyl-TM | “Sodium phenylbutyrate (Buphenyl-TM) is a drug that has been used to treat other types of urea cycle disorders.” NCT00345605 ↗ |
| Known as | NaPB | “The purpose of the study is to evaluate the safety of sodium phenylbutyrate (NaPB) treatment in subjects with amyotrophic lateral sclerosis (ALS) and the ability to take this...” NCT00107770 ↗2“This study will investigate if the drug sodium phenylbutyrate (NaPB) impacts blood pressure and vascular function in healthy volunteers and in patients with diabetes.” NCT06645769 ↗ “This has led us to try an alternative method, which is to lower BCAA levels using Sodium Phenylbutyrate (NaPB).” NCT05028803 ↗ |
| Known as | NaPBA | “Our study seeks to investigate the potential small molecule inhibition of the kinase that regulates BCKDC by applying a novel activity of sodium phenylbutyrate (NaPBA), in MSUD.” NCT01529060 ↗3“Buphenyl (NaPBA) will be the comparator drug to HPN-100 in this study.” NCT00992459 ↗ |
| Known as | PBA | “Dose of interventions: 5,000 IU of vitamin D (cholecalciferol tablets) once daily and 500 mg PBA (sodium phenylbutyrate tablets) twice daily for 16 weeks.” NCT01702974 ↗1“Immunotherapy using vitamin D (vitD3 ) and phenylbutyrate (PBA) may support standard drug regimens used to treat infectious diseases.” PMID 29696707 ↗ May 2018 |
| Known as | phenylbutyrate | “500 mg sodium phenylbutyrate (4-phenylbutyric acid, sodium salt) in tablet form twice daily and 5000 IU of cholecalciferol once daily will be given orally for 2 months” NCT01580007 ↗16“Combination of 3 g phenylbutyrate and 1 g taurursodiol” NCT05619783 ↗ |
| Known as | Sodium 4-Phenylbutyrate | ClinicalTrials.gov intervention name — accepted as the source's own label NCT00016744 ↗ |
| Action | Restore | “Both drugs may be able to restore normal chloride movements in body organs and glands.” NCT00016744 ↗ |
| Modality | Small molecule | “Phenylbutyrate is an aromatic fatty acid” NCT00006450 ↗ |
| Route | Intravenous | “phenylbutyrate administered as a continuous intravenous infusion for 28 days” NCT00001565 ↗ |