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SODIUM PHENYLBUTYRATE
At a glance
| Generic name | SODIUM PHENYLBUTYRATE |
|---|---|
| Modality | Small molecule |
| Phase | FDA-approved |
| First approval | 1996 |
Approved indications
Common side effects
- Amenorrhea/menstrual dysfunction
- Acidosis
- Hypoalbuminemia
- Anemia
- Alkalosis
- Hyperchloremia
- Increased alkaline phosphatase
- Hypophosphatemia
- Decreased appetite
- Increased liver transaminases
- Leukopenia
- Leukocytosis
Serious adverse events
- Aplastic anemia
- Pancreatitis
- Peptic ulcer disease
- Rectal bleeding
- Renal tubular acidosis
- Arrhythmia
- Neurotoxicity
- Syncope
- Hyperbilirubinemia
- Hypernatremia
Key clinical trials
- PROFIL Study to Investigate the Effect of GPB on NfL Levels in Patients With Corticobasal Syndrome (CBS) (PHASE2)
- Na-Phenylbutyrate VAscular Trial (EARLY_PHASE1)
- Phenylbutyrate for Monogenetic Developmental and Epileptic Encephalopathy (EARLY_PHASE1)
- A Clinical Study of Glycerol Phenylbutyrate in Chinese Patients With Urea Cycle Disorders
- Efficacy and Safety of the Treatment of Pyruvate Dehydrogenase Deficiency Patients With Glycerol Phenylbutyrate (RAVICTI) (PHASE2)
- Phenylbutyrate in Proteinuric Nephropathies (PHASE2, PHASE3)
- Study of Sodium Phenylbutyrate (ACER-001) for the Treatment of Patients With Medium Chain Acyl-CoA Dehydrogenase Deficiency (MCADD) (PHASE2)
- PBA Use for Treatment of ATF6-/- Patients (EARLY_PHASE1)
Primary sources
Every claim on this page is sourced from regulatory or scientific primary sources. See our editorial policy for full methodology.
| Source | Used for |
|---|---|
| FDA label | Mechanism, indications, dosing, boxed warnings, drug interactions |
| ClinicalTrials.gov | Trial enrolment, design, endpoints, results |
Competitive intelligence
For the full competitive landscape — auto-detected comparators, recent regulatory actions across the set, upcoming PDUFA, patent timeline, sponsor landscape:
- SODIUM PHENYLBUTYRATE CI brief — competitive landscape report
- SODIUM PHENYLBUTYRATE updates RSS · CI watch RSS