A) FORMS
1) HEREDITARY OROTIC ACIDURIA
a) Orange-flavored granules are available in single-use packet containing 2 grams of uridine triacetate in cartons of 30 packets (Prod Info XURIDEN(TM) oral granules, 2015).
2) ANTIDOTE
a) Orange-flavored granules are available in single-dose packets containing 10 grams of uridine triacetate for the emergency treatment of adult and pediatric patients following a fluorouracil or capecitabine overdose (Prod Info VISTOGARD(R) oral granules, 2015).
B) USES
1) HEREDITARY OROTIC ACIDURIA
a) Uridine triacetate, a pyrimidine analog, is used for uridine replacement for the treatment of hereditary orotic aciduria (Prod Info XURIDEN(TM) oral granules, 2015).
2) ANTIDOTE
a) As of December 2015, the FDA has approved uridine triacetate as an emergency treatment of adult and pediatric patients following a fluorouracil or capecitabine overdose regardless of the presence of symptoms or in those patients that exhibit early-onset of severe or life-threatening toxicity affecting the cardiac or central nervous system, and/or early onset of unusually severe adverse effects (eg., gastrointestinal toxicity and/or neutropenia) within 96 hours following the end of fluorouracil or capecitabine administration (Prod Info VISTOGARD(R) oral granules, 2015).
b) Formerly, uridine triacetate had orphan drug status as an antidote in 5-fluorouracil (5-FU) overdose (Bamat, Tremmel, and O'Neil, 2010; Von Borstel et al, 2009).
3) EFFICACY
a) Based on limited experience, uridine triacetate appears to be an effective and lifesaving antidote. All patients treated to date (more than 35 patients) with uridine triacetate following 5-FU overdose have recovered completely. Patients did not develop the anticipated gastrointestinal and hematologic toxicities following 5-FU overdose. Uridine triacetate was found to be effective after exposure to a range of doses of 5-FU; the highest dose for which a patient recovered was 10,000 mg of 5-FU infused over 3 hours. Two other patients recovered fully after receiving uridine triacetate following 5-FU doses of 8960 mg over 3 hours and 5000 mg over 17 minutes, respectively (Bamat, Tremmel, and O'Neil, 2010).
b) Seventeen patients treated with uridine triacetate (formerly known as vistonuridine) following a 5-FU overdose were compared to data from 13 patients with similar 5-FU overdoses who received supportive care only. All patients receiving uridine triacetate began treatment within 8 to 96 hours after overdose. A full recovery was seen in all 17 patients treated with uridine triacetate, even though a fatal outcome was predicted for 13 of these patients based on 5-FU dose and rate of administration. Eleven of the 13 patients who received symptomatic care had a fatal outcome predicted by 5-FU dose and rate of administration, and death occurred in all 11 despite supportive efforts (Von Borstel et al, 2009).