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Ravicti (GLYCEROL PHENYLBUTYRATE)
PAA, derived from glycerol phenylbutyrate, conjugates with glutamine to form PAGN, providing an alternate pathway for nitrogen excretion.
Ravicti (Glycerol Phenylbutyrate) is a small molecule drug developed by Hyperion Therap Inc and currently owned by Horizon Therap US. It is used to treat disorders of the urea cycle metabolism, a condition where the body is unable to properly remove ammonia. Ravicti works by breaking down ammonia into a less toxic form that can be excreted by the body. It is available as a branded product, with a generic manufacturer available. Key safety considerations include the potential for gastrointestinal side effects and the need for regular monitoring of liver function.
At a glance
| Generic name | GLYCEROL PHENYLBUTYRATE |
|---|---|
| Sponsor | Horizon Therap Us |
| Drug class | glycerol phenylbutyrate |
| Modality | Small molecule |
| Therapeutic area | Metabolic |
| Phase | FDA-approved |
| First approval | 2013 |
Mechanism of action
Glycerol phenylbutyrate is metabolized to PAA, which then binds to glutamine in the liver and kidneys to form PAGN. This compound is excreted by the kidneys, allowing for the removal of excess nitrogen in patients with UCDs.
Approved indications
- Disorder of the urea cycle metabolism
Common side effects
- diarrhea
- headache
- flatulence
- abdominal pain
- vomiting
- decreased appetite
- fatigue
- dyspepsia
- nausea
- dizziness
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)
Patents
| Patent | Expiry | Type |
|---|---|---|
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 |
| FDA Orange Book | Patents + exclusivity |
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
- Horizon Therap Us portfolio CI