CYSTADANE is a prescription medication that only contains the active ingredient betaine anhydrous (powder).

Betaine is produced naturally by the body. Some foods contain tiny amounts of betaine. CYSTADANE is manufactured according to the high quality and consistency standards required for prescription drugs.

How does CYSTADANE work to treat homocystinuria?

When someone has homocystinuria, their body cannot properly process methionine or homocysteine—two amino acids that the body needs. This can cause homocysteine in the blood to build up to dangerous levels.

CYSTADANE provides the body with an alternate biochemical pathway, or a different way to help convert homocysteine back to methionine. This lowers the level of homocysteine in the blood. CYSTADANE has been shown to be effective in treating the 3 types of homocystinuria.

What are the possible side effects?

CYSTADANE may worsen high methionine levels in people who have CBS deficiency, possibly causing fluid to build up in the brain. Therefore, doctors will monitor blood methionine levels of people with CBS deficiency while they are taking CYSTADANE. This will help ensure that methionine levels stay in the appropriate range.

The most common side effects of CYSTADANE therapy are nausea and gastrointestinal (GI) upset.

For safety information, click here.

How is CYSTADANE taken?

Always take CYSTADANE as prescribed by your doctor.

A hand shaking a bottle of CYSTADANE® (betaine anhydrous for oral solution)

Shake the bottle of CYSTADANE lightly, then remove the cap.

The measuring scoop provided with your CYSTADANE prescription

Use the scoop provided to measure the number of scoops as prescribed by your doctor.* One level scoop equals 1 gram of powdered betaine.

CYSTADANE powder being mixed with water

Mix the powder with 4 to 6 ounces (120 to 180 mL) of water, juice, milk, or formula until completely dissolved, or mix the powder with food.

Person drinking a mixture of CYSTADANE powder and water

Eat or drink the CYSTADANE mixture right away.

Hands replacing the cap on a bottle of prescription CYSTADANE

Replace the bottle cap tightly to protect CYSTADANE from moisture.

A CYSTADANE prescription bottle on a shelf

Store the bottle at room temperature.

*Over time the doctor may adjust the amount of CYSTADANE taken based on tests of blood homocysteine levels and other factors.

Can CYSTADANE be taken with other therapies?

CYSTADANE is usually prescribed along with other therapies. Individual treatment will be based on the cause of homocystinuria—CBS deficiency, MTHFR deficiency, or cbl defects—and other factors. Treatment may include:

  • Taking B vitamins, such as vitamin B6 (pyridoxine), vitamin B9 (folic acid), and/or vitamin B12 (cobalamin) (for people with CBS deficiency)
  • Eating a diet low in protein and/or methionine (for people with CBS deficiency)
  • Getting B12 (hydroxocobalamin) shots (for people with cbl defects)
  • Taking methionine (an amino acid) (for people with MTHFR deficiency or cbl defects)

Always check with your doctor about what medications and diet are right for you.

Adding CYSTADANE to other therapies may further reduce homocysteine in the blood toward target levels.

INDICATIONS AND USAGE

CYSTADANE (betaine anhydrous for oral solution) is indicated for the treatment of homocystinuria to decrease high homocysteine blood levels. Homocystinuria is a rare genetic disorder in which there is an abnormal accumulation of the amino acid homocysteine in the blood and urine. The following are considered to be homocystinuria disorders:

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

IMPORTANT SAFETY INFORMATION

  • Hypermethioninemia: CYSTADANE may worsen high methionine blood levels in patients with CBS deficiency. Accumulation of excess fluid in the brain has been reported.
  • Monitoring: If you have been told you have CBS deficiency, your doctor will be monitoring your methionine blood levels to see if changes in your diet and dosage are necessary.
  • Most common side effects were nausea and gastrointestinal distress, based on a survey of doctors.
  • 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: The effects of CYSTADANE on pregnancy and an unborn baby are not known.
  • Nursing women: Breast-feeding by a patient taking CYSTADANE is not recommended.
  • Pediatrics: 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 slowly adjusting the dose until the desired medical result is achieved.
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IMPORTANT SAFETY INFORMATION

  • Hypermethioninemia: CYSTADANE may worsen high methionine blood levels in patients with CBS deficiency. Accumulation of excess fluid in the brain has been reported.
  • Monitoring: If you have been told you have CBS deficiency, your doctor will be monitoring your methionine blood levels to see if changes in your diet and dosage are necessary.