Treating Homocystinuria

Creating an alternate remethylation pathway with CYSTADANE

Image of arrows and chemical compositions representing the creation of an alternate remethylation pathway with CYSTADANE

INDICATIONS AND USAGE

Cystadane is a methylating agent indicated for the treatment of homocystinuria to decrease elevated homocysteine blood levels. Included within the category of homocystinuria are:

  • Cystathionine beta-synthase (CBS) deficiency
  • 5,10-methylenetetrahydrofolate reductase (MTHFR) deficiency
  • Cobalamin cofactor metabolism (cbl) defects

IMPORTANT SAFETY INFORMATION

  • Hypermethioninemia: Cystadane may worsen elevated plasma methionine concentrations in patients with CBS deficiency. Cerebral edema has been reported in patients receiving Cystadane.
  • Monitoring: Monitor plasma methionine concentrations in patients with CBS deficiency. Keep plasma methionine concentrations below 1,000 μmol/L through dietary modification and, if necessary, a reduction of Cystadane dose.
  • Most common adverse reactions (incidence > 2%) were nausea and gastrointestinal distress, based on physician survey.
  • To report SUSPECTED SIDE EFFECTS, contact Recordati Rare Diseases Inc. at 1-888-575-8344, or FDA at 1‑800-FDA-1088 or www.fda.gov/medwatch.
  • Pregnancy: Animal reproduction studies have not been conducted with Cystadane. Use only if clearly needed.
  • Nursing women: It is not known whether Cystadane is excreted in human milk. Use only if clearly needed.
  • Pediatrics: Pediatric patients ranging in age from 24 days to 17 years have been treated with Cystadane. Children younger than 3 years of age may benefit from dose titration.
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IMPORTANT SAFETY INFORMATION

  • Hypermethioninemia: Cystadane may worsen elevated plasma methionine concentrations in patients with CBS deficiency. Cerebral edema has been reported in patients receiving Cystadane.
  • Monitoring: Monitor plasma methionine concentrations in patients with CBS deficiency. Keep plasma methionine concentrations below 1,000 μmol/L through dietary modification and, if necessary, a reduction of Cystadane dose.