Expanded Indication for Ravicti
April 28, 2017 – Ravicti® (glycerol phenylbutyrate) oral liquid, manufactured by Horizon Pharma, has received U.S. FDA approval for treatment of patients with a urea cycle disorder who are as young as 2 months of age. Urea cycle disorders are hereditary conditions caused by shortages of one or more enzymes that eliminate nitrogen (converted into ammonia) from the blood. These disorders are estimated to affect fewer than 150 new patients per year in the U.S. For patients with a urea cycle disorder, excess ammonia accumulates over time. This accumulation will eventually cause brain damage, which could lead to death.
The expanded indication increases the age range of patients eligible to receive Ravicti. Initially, Ravicti was FDA approved to treat urea cycle disorder in patients no younger than 2 years of age. Ravicti must be used along with a protein-restricted diet and, in some cases, dietary supplements. The recommended dose of Ravicti is 4.5mL/m2/day to 11.2mL/m2/day in three equally divided doses that are given, along with food or infant formula, by mouth, nasogastric tube or gastrostomy tube.
Adverse reactions or quality problems experienced with the use of a recalled product may be reported to the FDA’s MedWatch Adverse Event Reporting Program either online, by U.S. mail or by fax.
- Complete and submit the report Online: www.fda.gov/medwatch/report.htm
- U.S. Mail or Fax: Download form www.fda.gov/MedWatch/getforms.htm or call 1-800-332-1088 to request a reporting form, then complete and return to the address on the pre-addressed form, or submit by fax to 1800-FDA-0178
Actual drug patent expiration dates and availability of new medications are subject to change due to patent litigation, settlement agreements, additional patents, exclusivities, and final FDA approval. Distribution and availability of new medications at pharmacies may not occur immediately following FDA approval. Patients are advised to speak with their healthcare professional or pharmacist regarding appropriateness as well as actual availability.
*This is provided for information only. The reference to any medication above does not mean the medication is covered by your plan.