News & Events
Temporary importation of CYSTADANE® (betaine anhydrous for oral solution) to address impending shortage
IMPORTANT PRESCRIBING INFORMATION
22 December 2017
Dear Health Care Provider,
There has been a manufacturing issue with the CYSTADANE® (betaine anhydrous for oral solution) approved for use and distribution in the United States (“U.S.-labeled CYSTADANE”), which has resulted in an interim shortage of this product in the U.S. market.
In order to alleviate the shortage of CYSTADANE in the U.S., Recordati Rare Diseases Inc. (Recordati) is coordinating with the U.S. Food and Drug Administration (FDA) to increase the availability of CYSTADANE. Recordati has initiated temporary importation of its CYSTADANE product approved for use in the United Kingdom into the U.S. market. This product is manufactured by Orphan Europe SARL in France. At this time, no other entity except Recordati, as the U.S. agent of Orphan Europe SARL, is authorized to import or distribute these products in the United States. FDA has not approved the product manufactured by Orphan Europe SARL’s manufacturing facility in France. The imported CYSTADANE is lot CYP1705. It will be dispensed by the U.S. specialty pharmacy, AnovoRx Group LLC.
There are some key differences between the FDA-approved CYSTADANE and the imported CYSTADANE.
- Measuring scoops: FDA-approved CYSTADANE is packaged with one scoop (white) that dispenses 1g of betaine anhydrous. Imported CYSTADANE is packaged with 3 measuring scoops (green, blue, and pink) that dispense 100mg, 150mg, or 1g of betaine anhydrous. The large 1g pink scoop in the imported CYSTADANE is equivalent to the white 1g scoop in the FDA-approved CYSTADANE. Patients should pay careful attention to use the appropriate scoop to take their prescribed dose. Please pass this information along to the patients. If the incorrect scoop is utilized, improper dosing may result, and the CYSTADANE treatment can be ineffective. This may result in an increased risk for cognitive disability and/or stroke due to the progression of the underlying disease.
- Dosing: The labeled dosing in the FDA-approved CYSTADANE is 6 grams/day in divided doses of 3 grams twice daily, and, for pediatric patients less than 3 years of age, dosage may be started at 100 mg/kg/day divided in twice daily doses and increased weekly by 50 mg/kg increments. The labeled dosing in the imported CYSTADANE is 100 mg/kg/day divided into 2 doses per day. Practitioners should be aware of these differences when prescribing and monitoring CYSTADANE.
A copy of the FDA-approved full prescribing information is being distributed with the imported CYSTADANE. Please refer to the FDA-approved package insert for full prescribing information. Storage conditions are the same as that of the FDA-approved product. Store at room temperature, 15º – 30ºC (59º – 86ºF). CYSTADANE is for oral use only. Protect from moisture. Keep this and all medications out of the reach of children.
U.S. FDA-approved Indication
CYSTADANE (betaine anhydrous for oral solution) 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) defect
For reporting of adverse events and more information
Any adverse effects or medication issues resulting from the use of this drug or quality issues should be reported to Recordati Rare Diseases Inc. at 1-888-575-8344.
Adverse events or quality problems experienced with the use of this product may also be reported to the FDA’s MedWatch Adverse Event Reporting program either online, by regular mail or by fax:
- Complete and submit the report Online: www.fda.gov/medwatch/report.htm
- Regular mail or Fax: Download form www.fda.gov/MedWatch/getforms.htm or call 1- 800-332-1088 to request a reporting form, then complete and return to the address on the pre-addressed form, or submit by fax to 1-800-FDA-0178.
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.
- 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.
We are committed to helping address patients’ unmet needs through our corporate mission. If you have questions or concerns regarding this product, please call AnovoRx Group, the specialty pharmacy that dispenses CYSTADANE, at 1-888-487-4703 or Recordati Rare Diseases Medical Information at 1-888-575-8344.
Steven R. Peltier
Vice President, Regulatory & Quality Compliance
Chief Compliance Officer
Recordati Rare Diseases Inc.