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 in pediatric and adult patients for the treatment of homocystinuria to decrease elevated homocysteine blood concentrations. Included within the category of homocystinuria are:

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

IMPORTANT SAFETY INFORMATION

  • Hypermethioninemia in Patients with CBS Deficiency: CYSTADANE may worsen elevated plasma methionine concentrations and cerebral edema has been reported. Monitor plasma methionine concentrations in patients with CBS deficiency. Keep plasma methionine concentrations below 1,000 micromol/L through dietary modification and, if necessary, a reduction of CYSTADANE dosage.
  • Most common adverse reactions (> 2%) are: nausea and gastrointestinal distress, based on physician survey.
  • To report SUSPECTED ADVERSE REACTIONS, contact Recordati Rare Diseases Inc. at 1-888-575-8344, or FDA at 1‑800-FDA-1088 or www.fda.gov/medwatch.
See More

IMPORTANT SAFETY INFORMATION

  • Hypermethioninemia in Patients with CBS Deficiency: CYSTADANE may worsen elevated plasma methionine concentrations and cerebral edema has been reported. Monitor plasma methionine concentrations in patients with CBS deficiency. Keep plasma methionine concentrations below 1,000 micromol/L through dietary modification and, if necessary, a reduction of CYSTADANE dosage.
  • Most common adverse reactions (> 2%) are: nausea and gastrointestinal distress, based on physician survey.