glycerol phenylbutyrate (ravicti) Report issue

Small molecule Orphan Drug FDA Approved FDA Fast Track FDA
AACT ChEMBL DrugBank Drug Central Drugs@FDA KEGG MeSH PubChem repoDB

Active Ingredient History

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Glycerol phenylbutyrate (USAN), trade name Ravicti, is a medication used in the treatment of certain inborn urea cycle disorders. The medication works by preventing the harmful buildup of ammonia in the body. It is an FDA-approved prescription drug in the US. It is approved for anyone over 2 months of age. It was developed by Hyperion Therapeutics based on the existing drug Buphenyl, and received approval on February 1, 2013. Hyperion has been criticized for setting a high price for the drug. The price was set at US$250,000–290,000. In 2014, the drug generated $30.8 million in net sales, far behind the older and less expensive Buphenyl. In March 2015, Horizon Pharma acquired Hyperion Therapeutics and thus Raviciti.   Wikipedia

Chemistry

  • SMILES: O=C(CCCc1ccccc1)OCC(COC(=O)CCCc2ccccc2)OC(=O)CCCc3ccccc3
  • InChIKey: ZSDBFLMJVAGKOU-UHFFFAOYSA-N
  • Mol. Mass: 530.66
  • ALogP: 6.05
  • ChEMBL Molecule:
    glycerol phenylbutyrate  
More Chemistry

Pharmacology

  • Mechanism of Action:
  • Multi-specific: Missing data
  • Black Box: No
  • Availability: Prescription Only
  • Delivery Methods: Oral
  • Pro Drug: Yes

Drug Pricing (per unit)

United States

$138.7932 - $191.5364
More Pricing Detail

Note: This drug pricing data is preliminary, incomplete, and may contain errors.

Alternative names

glycerol phenylbutyrate | glyceryl tri-4-phenylbutyrate | gpb | gt4p | hpn100 | hpn 100 | hpn-100 | ravicti

Organizations (16)

Research & Development (15)

Organization Org Type FDA approvals Clinical Trials involvement Org ID Force Sort

Marketing (3)

Organization Org Type FDA approvals Clinical Trials involvement Org ID Force Sort

Indications (11)

Approved Indications (1)


 

Urea Cycle Disorders, Inborn

Experimental Indications - Clinical Trial Phases 1-4 (11)


 

Color Vision Defects (Phase 1)

Cystic Fibrosis (Phase 1/Phase 2)

Deficiency Diseases (Phase 1)

Drug-Related Side Effects and Adverse Reactions (Phase 1)

Epilepsy (Phase 1)

Fibrosis (Phase 2)

Healthy Volunteers (Phase 1)

Hepatic Encephalopathy (Phase 2)

Parkinson Disease (Phase 1)

Sports Medicine (Phase 3)

Urea Cycle Disorders, Inborn (Phase 4)

Overview

  • Highest Phase: Phase 4
  • # of Trials: 20
  • # of Trial Organizations: 15

Trials by Phase

Trial Phase Start Date Organizations Indications

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