CYSTADANE is a prescription medicine 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 high levels of homocysteine?

When someone has a genetic disorder that causes 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 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 genetic disorders that cause homocystinuria:

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

What are the possible side effects?

In patients with CBS deficiency (classical homocystinuria), CYSTADANE may worsen high methionine blood levels, and accumulation of excess fluid in the brain has been reported. 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.

The most common side effects of CYSTADANE therapy are nausea and gastrointestinal (GI) distress, based on a survey of doctors.

For safety information, click here.

How is CYSTADANE taken?

CYSTADANE should only be taken as directed by your doctor.

A hand shaking a bottle of CYSTADANE® (betaine anhydrous for oral solution) 1

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

The measuring scoop provided with your CYSTADANE prescription 2

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 3a

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 3b

Immediately eat or drink the CYSTADANE mixture.

Hands replacing the cap on a bottle of prescription CYSTADANE 4

Replace the bottle cap tightly to protect CYSTADANE from moisture.

A CYSTADANE prescription bottle on a shelf 5

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 (classical homocystinuria), MTHFR deficiency, or cbl defect – and other factors. Treatment may include:

  • Taking vitamin B6 (pyridoxine) and additional B vitamins (for people with classical homocystinuria)
  • Eating a low-protein diet (for people with classical homocystinuria)
  • Taking a methionine-free amino acid formula (for people with classical homocystinuria)
  • Getting hydroxocobalamin injections (for people with cbl defect)

Always check with your doctor about what medicines 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 in children and adults 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) defect

IMPORTANT SAFETY INFORMATION

  • Hypermethioninemia in Patients with CBS Deficiency: CYSTADANE may worsen high methionine blood levels and accumulation of excess fluid in the brain has been reported. 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.
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IMPORTANT SAFETY INFORMATION

  • Hypermethioninemia in Patients with CBS Deficiency: CYSTADANE may worsen high methionine blood levels and accumulation of excess fluid in the brain has been reported. 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.