Last reviewed · How we verify
HPN-100
At a glance
| Generic name | HPN-100 |
|---|---|
| Also known as | GT4P, Glyceryl tri-(4-phenylbutyrate), GT4P, GT4P, Glyceryl tri-(4-phenylbutyrate), RAVICTI, GT4P, glyceryl tri-(4-phenylbutyrate), RAVICTI |
| Sponsor | Amgen |
| Modality | Small molecule |
| Phase | FDA-approved |
Approved indications
Common side effects
Key clinical trials
- PROFIL Study to Investigate the Effect of GPB on NfL Levels in Patients With Corticobasal Syndrome (CBS) (PHASE2)
- 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)
- PBA Use for Treatment of ATF6-/- Patients (EARLY_PHASE1)
- To Evaluate the Safety of Long-term Use of HPN-100 in the Management of Urea Cycle Disorders (UCDs) (PHASE4)
- Study of the Safety, Pharmacokinetics and Efficacy of HPN-100, in Pediatric Subjects With Urea Cycle Disorders (UCDs) (PHASE3)
- Study of the Safety and Tolerability of HPN-100 Compared to Sodium Phenylbutyrate in Children With Urea Cycle Disorders (PHASE2)
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 |
|---|---|
| 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:
- HPN-100 CI brief — competitive landscape report
- HPN-100 updates RSS · CI watch RSS
- Amgen portfolio CI