New medicine for rare diseases! Fda approved Ravicti (benzoate glyceride) for urea circulation disorders under 2 months:

Horizon Pharma is a pharmaceutical company focused on research, development and commercialization of innovative drugs for the treatment of rare and rheumeumatological diseases. Recently, the company announced that the U.S. Food and Drug Administration (FDA) has approved the expansion of oral fluids from Gavicti (glycerol phenylbutyrate) to include infants under 2 months of age with urea circulation disorder (UCD).
is an waste product produced by protein metabolism, and urea circulation removes it from the blood and converts it into urea, which is excreted through urine. Nitrogen accumulates in UCD patients and remains in the body in the form of ammonia.
Ravicti oral solution has been approved by the FDA as a nitrogen binding agent for long-term management of UCD adult and child patients who cannot be treated with dietary protein restrictions and/or supplements alone. Ravicti must be used with dietary protein restrictions and, in some cases, with dietary supplements such as essential amino acids, arginine, guarine, protein-free calorie supplements.
"FDA approval of Ravicti for UCD infants under 2 months of age provides them with a new treatment that is easy to take and give to infants," said Nicola Longo, a clinical geneticist at the University of Utah Hospital and lead researcher on a neonatal study at Ravicti. UCD is a very serious and potentially life-threatening disease, and we hope to improve the prognosis of patients in combination with early diagnosis (neonatal screening or measurement of ammonia levels) and new treatments. The
FDA's approval of Ravicti's expansion of the applicable population is based on the safety, efficacy, and pharmacodynamics of pediatric studies conducted in 2-month-old UCD infants. In the study, 10 patients transitioned from sodium benzoate to Ravicti, 3 patients transitioned from intravenous sodium benzoate and sodium benzoate to Ravicti, and 3 patients were first-time patients. The average time for these patients to receive Ravicti treatment was 10.7 months. The results showed that in infants under 2 months of age with UCD, ravicti patients maintained stable levels of ammonia compared to pre-study patients, confirming the safety and effectiveness of Ravicti. In addition, the average ammonia level during Ravicti treatment is lower than the baseline value.
UCD is a rare genetic disorder affecting about 1/35,000 live births in the United States. It is caused by a lack of an enzyme in the urea cycle, a process that converts excess ammonia from the bloodstream and eventually removes it from the body. As a result, people with UCD experience high levels of ammonia, or elevated levels of ammonia in the blood, which then reach the brain, causing irreversible brain damage, coma, or death. UCD symptoms may first appear at any age, depending on the severity of the disease, and more severe defects occur early in life. (Bio Valley)