Cystadane
Generic: BETAINE
Basic Information
Manufacturer
Recordati Rare Diseases
Product Type
HUMAN PRESCRIPTION DRUG
Route of Administration
ORAL
FDA Set ID
31a684d0-cd98-4e46-b43a-ccbd17c41559
Indications & Usage
1 INDICATIONS AND USAGE CYSTADANE ® is indicated for the treatment of homocystinuria to decrease elevated homocysteine blood concentrations in pediatric and adult patients.
Included within the category of homocystinuria are: Cystathionine beta-synthase (CBS) deficiency 5,10-methylenetetrahydrofolate reductase (MTHFR) deficiency Cobalamin cofactor metabolism (cbl) defect 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 ( 1 ): Cystathionine beta-synthase (CBS) deficiency 5,10-methylenetetrahydrofolate reductase (MTHFR) deficiency Cobalamin cofactor metabolism (cbl) defect
Included within the category of homocystinuria are: Cystathionine beta-synthase (CBS) deficiency 5,10-methylenetetrahydrofolate reductase (MTHFR) deficiency Cobalamin cofactor metabolism (cbl) defect 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 ( 1 ): Cystathionine beta-synthase (CBS) deficiency 5,10-methylenetetrahydrofolate reductase (MTHFR) deficiency Cobalamin cofactor metabolism (cbl) defect
Adverse Reactions
6 ADVERSE REACTIONS The following serious adverse reactions are described elsewhere in labeling: Hypermethioninemia and cerebral edema in patients with CBS deficiency [ see Warnings and Precautions ( 5.1 ) ].
Most common adverse reactions (> 2%) are: nausea and gastrointestinal distress, based on physician survey.
( 6.1 ) 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.
6.1 Clinical Trials Experience Because clinical trials are conducted under widely varying conditions, adverse reaction rates observed in clinical trials of a drug cannot be directly compared to rates in the clinical trials of another drug and may not reflect the rates observed in practice.
The assessment of clinical adverse reactions is based on a survey study of 41 physicians, who treated a total of 111 homocystinuria patients with CYSTADANE.
Adverse reactions were retrospectively recalled and were not collected systematically in this open-label, uncontrolled, physician survey.
Thus, this list may not encompass all types of potential adverse reactions, reliably estimate their frequency, or establish a causal relationship to drug exposure.
The following adverse reactions were reported (Table 1): Table 1: Number of Patients with Adverse Reactions to CYSTADANE by Physician Survey Adverse Reactions Number of Patients Nausea 2 Gastrointestinal distress 2 Diarrhea 1 "Bad Taste" 1 "Caused Odor" 1 Questionable psychological changes 1 "Aspirated the powder" 1 6.2 Postmarketing Experience The following adverse reactions have been identified during post approval use of CYSTADANE.
Because these reactions are reported voluntarily from a population of uncertain size, it is not always possible to reliably estimate their frequency or establish a causal relationship to drug exposure.
Severe cerebral edema and hypermethioninemia have been reported within 2 weeks to 6 months of starting CYSTADANE therapy, with complete recovery after discontinuation of CYSTADANE.
All patients who developed cerebral edema had homocystinuria due to CBS deficiency and had severe elevation in plasma methionine concentrations (range 1,000 to 3,000 microM).
As cerebral edema has also been reported in patients with hypermethioninemia, secondary hypermethioninemia due to betaine therapy has been postulated as a possible mechanism of action [ see Warnings and Precautions ( 5.1 ) ].
Other adverse reactions include: anorexia, agitation, depression, irritability, personality disorder, sleep disturbed, dental disorders, diarrhea, glossitis, nausea, stomach discomfort, vomiting, hair loss, hives, skin odor abnormalities, and urinary incontinence.
Most common adverse reactions (> 2%) are: nausea and gastrointestinal distress, based on physician survey.
( 6.1 ) 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.
6.1 Clinical Trials Experience Because clinical trials are conducted under widely varying conditions, adverse reaction rates observed in clinical trials of a drug cannot be directly compared to rates in the clinical trials of another drug and may not reflect the rates observed in practice.
The assessment of clinical adverse reactions is based on a survey study of 41 physicians, who treated a total of 111 homocystinuria patients with CYSTADANE.
Adverse reactions were retrospectively recalled and were not collected systematically in this open-label, uncontrolled, physician survey.
Thus, this list may not encompass all types of potential adverse reactions, reliably estimate their frequency, or establish a causal relationship to drug exposure.
The following adverse reactions were reported (Table 1): Table 1: Number of Patients with Adverse Reactions to CYSTADANE by Physician Survey Adverse Reactions Number of Patients Nausea 2 Gastrointestinal distress 2 Diarrhea 1 "Bad Taste" 1 "Caused Odor" 1 Questionable psychological changes 1 "Aspirated the powder" 1 6.2 Postmarketing Experience The following adverse reactions have been identified during post approval use of CYSTADANE.
Because these reactions are reported voluntarily from a population of uncertain size, it is not always possible to reliably estimate their frequency or establish a causal relationship to drug exposure.
Severe cerebral edema and hypermethioninemia have been reported within 2 weeks to 6 months of starting CYSTADANE therapy, with complete recovery after discontinuation of CYSTADANE.
All patients who developed cerebral edema had homocystinuria due to CBS deficiency and had severe elevation in plasma methionine concentrations (range 1,000 to 3,000 microM).
As cerebral edema has also been reported in patients with hypermethioninemia, secondary hypermethioninemia due to betaine therapy has been postulated as a possible mechanism of action [ see Warnings and Precautions ( 5.1 ) ].
Other adverse reactions include: anorexia, agitation, depression, irritability, personality disorder, sleep disturbed, dental disorders, diarrhea, glossitis, nausea, stomach discomfort, vomiting, hair loss, hives, skin odor abnormalities, and urinary incontinence.