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Ravicti
At a glance
| Generic name | Ravicti |
|---|---|
| Also known as | glycerol phenylbutyrate, HPN-100, Glycerol Phenylbutyrate, GPB, Glycerol phenylbutyrate |
| Sponsor | University of Pittsburgh |
| Modality | Small molecule |
| Therapeutic area | Metabolic |
| Phase | FDA-approved |
Approved indications
- Disorder of the urea cycle metabolism
Common side effects
- Diarrhea
- Headache
- Flatulence
- Abdominal pain
- Vomiting
- Decreased appetite
- Fatigue
- Dyspepsia
- Nausea
- Dizziness
- Upper abdominal pain
- Rash
Serious adverse events
- Neurotoxicity
- Pancreatic insufficiency
- Intestinal malabsorption
- Gastroesophageal reflux
- Increased hepatic enzymes
- Anemia
- Dehydration
- Metabolic acidosis
- Thrombocytosis
- Thrombocytopenia
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:
- Ravicti CI brief — competitive landscape report
- Ravicti updates RSS · CI watch RSS
- University of Pittsburgh portfolio CI