WHA

Xuriden (uridine triacetate)

Indications for Prior Authorization

Xuriden (uridine triacetate)
  • For diagnosis of Hereditary orotic aciduria
    Indicated in adult and pediatric patients for the treatment of hereditary orotic aciduria.

Criteria

Xuriden

Prior Authorization (Initial Authorization)

Length of Approval: 12 Month(s)

  • Diagnosis of hereditary orotic aciduria [A]
    • AND
  • Prescribed by or in consultation with a medical geneticist or other specialist that treats inborn errors of metabolism [B]
Xuriden

Prior Authorization (Reauthorization)

Length of Approval: 12 Month(s)

  • Patient demonstrates positive clinical response to therapy [C]

  • Guideline ID
    GL-149278

  • Formulary
    premium

  • Effective date
    Sep 1, 2024

  • P&T approval date
    Nov 18, 2015

P & T Revisions

2024-07-01, 2023-10-04, 2023-06-16, 2022-06-14, 2021-08-03, 2020-07-14

  1. Xuriden Prescribing Information. Wellstat Therapeutics Corporation, December 2019.
  2. Balasubramaniam S, Duley JA, Christodoulou J. Inborn errors of pyrimidine metabolism: clinical update and therapy. J Inherit Metab Dis. 2014;37(5):687-98.
  3. Jurecka A. Inborn errors of purine and pyrimidine metabolism. J Inherit Metab Dis. 2009;32(2):247-263.
  4. Weinberg ME, Roman MC, Jacob P, et al. Enhanced uridine bioavailability following administration of a triacetyluridine-rich nutritional supplement. PloS one. 2011;6(2).
  1. Hereditary orotic aciduria (uridine monophosphate [UMP] synthase deficiency) or HOA is a rare congenital disorder of pyrimidine metabolism caused by a defect in UMP synthase, a bi-functional enzyme that catalyzes the final 2 steps of the de novo pyrimidine biosynthetic pathway in mammalian cells. Ten defects in pyrimidine metabolic pathways have been identified to date; all exhibit autosomal recessive inheritance, however, the ubiquitous presence of pyrimidine-derived compounds underlies the heterogeneity in clinical expression, even within families, thus often making recognition difficult. While the true prevalence of these rare disorders is unknown, inborn errors of pyrimidine metabolism are now increasingly being recognized in adults with partial deficiencies, and so may present from birth onwards. [1-2]
  2. Three subtypes of HOA have been described; alongside clinical presentation (notably macrocytic hypochromic megaloblastic anemia and crystalluria), the ratio of urinary orotidine to orotate provides a means of differentiating the 3 subtypes. The enzyme defect in HOA can be bypassed by the administration of oral uridine which is not Food and Drug Administration-approved, but is available over-the-counter in various dietary/food supplements or as a bulk powder from which doses may be compounded; oral uridine has been used as a treatment for patients with HOA for more than 4 decades. Because uridine is widely used for several non-HOA conditions, the addition of a specialist prescriber requirement was added to reserve the use of XURIDEN for those patients with rare HOA. [1-4]
  3. The recommended starting dosage of oral Xuriden is 60 mg/kg once daily. The dose can be increased to 120 mg/kg (not to exceed 8 grams) once daily for insufficient efficacy, such as the occurrence of one of the following: (1) levels of orotic acid in urine remain above normal or increase above the usual or expected range for the patient, (2) laboratory values (eg, red blood cell or white blood cell indices) affected by hereditary orotic aciduria show evidence of worsening, or (3) worsening of other signs or symptoms of the disease. Case reports have demonstrated that the effects of exogenous uridine were maintained over months and years, as long as treatment continued at sufficient doses (with appropriate dose increases based on body weight increases). Most hematologic abnormalities and orotic aciduria reappeared within days to up to 2 or 3 weeks when administration of uridine was stopped or the dose was reduced. If treatment was interrupted for longer periods, body weight growth receded. [1]
  • 2024-07-01: Annual Review - No criteria changes
  • 2023-10-04: Program update to standard reauthorization language. No changes to clinical intent
  • 2023-06-16: Annual Review - no criteria changes
  • 2022-06-14: Annual Review - no criteria changes
  • 2021-08-03: 2021 Annual Review, no changes to criteria.
  • 2020-07-14: 2020 Annual review. Removed reference to drug name in reauth criteria, no changes to clinical criteria.