Q. What is uridine triacetate?
A. It is an investigational, orally active prodrug of uridine currently under development as an antidote to overexposure of fluorouracil (5-FU). Although not yet approved by the U.S. Food and Drug Administration, it has been shown to be effective in treating patients in emergency 5-FU overdose settings43–45 and is available in the United States under the expanded access program.
Q. Who might benefit from use of uridine triacetate?
A. Patients who have experienced early-onset toxicity from high doses of 5-FU (within 2 to 3 days of infusion) or impaired elimination may be candidates for immediate treatment with uridine triacetate. Patients with known or suspected overexposure to 5-FU as a result of dihydropyrimidine dehydrogenase deficiency also may be candidates for treatment with uridine triacetate.
Q. When should uridine triacetate be given?
A. It seems that the earlier uridine triacetate is given after 5-FU overexposure the better. However, it appears to be of benefit in patients who experience early-onset toxicities (e.g., severe mucositis, fever, diarrhea, and rash) 2 or 3 days after their 5-FU infusion.
Q. What is the expanded access program?
A. The expanded access program is a means by which manufacturers make investigational new drugs available, under certain circumstances, to treat patients with a serious disease or condition for which there is no comparable or satisfactory alternative therapy outside a clinical trial. In such cases, the potential benefit justifies the potential risks of treatment.
Q. How is uridine triacetate obtained through the expanded access program?
A. A patient’s physician and the drug manufacturer make special arrangements to obtain the drug for the patient. In an emergency situation, the physician can request the drug via telephone or other rapid means of communication, and authorization to ship and use the drug may be given by the U.S. Food and Drug Administration official over the telephone. In addition, the physician must submit a brief clinical history of the patient, including the diagnosis, the disease status, prior therapy, response to prior therapy, and the rationale for requesting the proposed treatment. Signed informed consent by the patient is also necessary.
Q. How long does it take to get uridine triacetate once requested?
A. In emergency situations, the drug is delivered rapidly, generally the day requested or within the next 24 hours.
Q. Are there cost considerations involved with obtaining and using uridine triacetate?
A. Costs may include the manufacturer’s cost of preparing the drug and the institution’s cost of administering the drug. The cost associated with the use of uridine triacetate may be reimbursed by some health insurers but not all. As for physicians, the cost to provide access to this drug may not be fully compensated, there may be liability issues, and participation requires commitment (contacting the drug manufacturer and filing paperwork).