Ravicti (glycerol phenylbutyrate) for Treatment of Urea Cycle Disorders (UCD)

Global ammonia capacity is expected to experience moderate growth over the next five years from 239.40 mtpa in 2022 to 293.52 mtpa by 2027. Around 95 planned and announced ammonia projects are expected to come online predominantly in North America, followed by the Middle East over the upco


Urea Cycle Disorders (UCD) can be treated with Ravicti (glycerol phenylbutyrate) in both adults and children over the age of two. Ammonia Market and Ucyclyd Pharma co-developed the medication.

The Food and Drug Administration (FDA) granted Hyperion approval in February 2013 to treat UCD with Ravicti in patients over the age of two.

Ucyclyd Pharma sold Hyperion the worldwide rights to Ravicti in March 2012. In the United States, Ravicti is expected to go on sale in April 2013.

Buphenyl, a drug developed by Ucyclyd Pharma for the same purpose, competes with Ravicti.

Urea cycle disorder "In February 2013, Hyperion received FDA approval for Ravicti as a treatment for UCD in patients aged two and older," Hyperion said.
A genetic condition known as urea cycle disorder (UCD) results from a deficiency in one of the six enzymes that remove ammonia from the blood. The nitrogen in UCD patients builds up as ammonia, which can cause brain damage, coma, or death.

The mechanism of action of Ravicti Ravicti contains a nitrogen-binding agent. The drug works by removing the harmful ammonia that has built up in the brain and blood of UCD patients. Additionally, the drug maintains safe levels of ammonia in the body throughout the day and night. The medication is available as a liquid that can be taken orally.

Ravicti (glycol phenylbutyrate) clinical trials The FDA approved Ravicti for adults and children based on a number of clinical studies.

"A genetic defect known as urine cycle disorder (UCD) is caused by a deficiency in one of the six enzymes that are responsible for the removal of ammonia from the blood stream."
A randomised, double-blind, active-controlled, non-inferiority clinical study was the foundation for the FDA's adult approval. Over 45 UCD patients who had been treated with sodium phenylbutyrate prior to enrollment were included in the study. The purpose of the study was to compare sodium phenylbutyrate and Ravicti. Establishing non-inferiority in the 24-hour AUC (a measure of ammonia exposure over 24 hours) was the primary endpoint of the study.

Ravicti and sodium phenylbutyrate were given to each patient at random for two weeks. Similar amounts of the drugs were given three times a day with meals. At the conclusion of the study, a total of 44 patients were examined for analysis.

To know about the assumptions considered for the study, Request for Free Sample Report

According to the findings of the study, the 24-hour AUC for ammonia in patients treated with Ravicti was not different from that in patients treated with sodium phenylbutyrate. The Ravicti group's mean 24-hour AUC was 866 mol/L an hour, while the sodium phenylbutyrate group's was 977 mol/L an hour, according to the findings.

Ravicti was the subject of yet another open-label clinical trial for the treatment of adult UCD patients. Monthly ammonia control and hyperammonemic crisis were evaluated in this uncontrolled 12-month study. Over 51 adult patients participated in the study. Patients' venous ammonia levels were monitored on a monthly basis.

Long-term mean fasting venous ammonia values for patients treated with Ravicti were found to be within normal limits, according to the study's findings.

Two open label studies provided the foundation for Ravicti's FDA approval in pediatrics. Patients with UCD between the ages of 2 and 17 participated in the studies. There were a total of 26 patients in the studies. Ravicti and sodium phenylbutyrate were given to the patients at the same dose. Patients were observed for venous ammonia measurements, blood and urine PK assessments, and treatment completion.

The study's 24-hour AUC for ammonia was 604 mol-h/L in the Ravicti group, compared to 815 mol-h/L in the sodium phenylbutyrate group. "The drug works by removing the toxic ammonia accumulated in the blood and the brain of the UCD-affected patients."
Ravicti was the subject of two additional 12-month clinical studies. They were uncontrolled, open-label studies. Monthly ammonia control and hyperammonemic crisis were the subjects of the studies.

The two studies revealed that Ravicti-administered patients' mean fasting venous ammonia values were within normal limits.