TORONTO, April 20, 2017 /CNW/ - Recordati Rare Diseases Canada Inc., a biopharmaceutical company committed to providing orphan therapies to underserved rare disease communities in Canada, today announced the acquisition of the Canadian marketing rights to CYSTADANE® (betaine anhydrous for oral solution). CYSTADANE®, the only betaine anhydrous prescription medication approved for use by Health Canada, is indicated for the treatment of homocystinuria to decrease elevated homocysteine blood levels.
Homocystinuria is the term used for several rare genetic disorders (diseases passed down through families) that cause levels of homocysteine to build up in blood and urine. This build-up occurs when the body cannot properly process methionine. Methionine and homocysteine are two very important amino acids, and represent two important building blocks that the body uses to make proteins. There are multiple forms of homocystinuria, which are distinguished by their signs and symptoms and genetic cause. High levels of homocysteine and abnormal levels of methionine can cause many different symptoms in the body and lead to chronic, serious, and potentially life-threatening health issues.
CYSTADANE® is a prescription pharmaceutical product that helps to remove homocysteine from the blood by converting excess homocysteine to methionine. CYSTADANE® has been evaluated in observational studies without concurrent controls in patients with homocystinuria and in a double-blind, placebo-controlled study, however, no formal statistical analyses were performed. Plasma levels of homocysteine decreased by 83-88% regardless of pre-treatment level, and plasma homocysteine decreased by 71-83%, regardless of the pre-treatment level.
"We are committed to meeting the needs of the Canadian homocystinuria community by continuing to supply CYSTADANE®" said Paul McCabe, General Manager, Recordati Rare Diseases Canada Inc. "As a company, our top priority is to ensure that there will be no interruptions or changes to patients' access. Our distribution model will be continuous to ensure that patients can receive continued access to this important treatment and help to manage their diseases."
For more information about CYSTADANE® or Recordati Rare Diseases Canada Inc., visit www.recordatirarediseases.ca.
Important information about CYSTADANE® (betaine anhydrous for oral solution)
Indications and Usage
CYSTADANE® (betaine anhydrous for oral solution) is 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
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.
- To report SUSPECTED ADVERSE REACTIONS, contact Canada Vigilance at 1-866-234-2345, or online at www.healthcanada.gc.ca/medeffect.
- 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.
About Recordati Rare Diseases Canada Inc.
Recordati Rare Diseases Canada Inc. is a biopharmaceutical company committed to providing often overlooked orphan therapies to underserved rare disease communities in Canada.
Recordati Rare Diseases Canada Inc. and our sister company, Orphan Europe, are part of the rare disease business within the Recordati Group, a public global pharmaceutical company committed to the research and development of new specialties with a focus on treatments for rare disease.
Recordati Rare Diseases Canada's mission is to reduce the impact of extremely rare and devastating diseases by providing urgently needed therapies. We work side-by-side with rare disease communities to increase awareness, improve diagnosis and expand availability of treatments for people with rare diseases.
The company's Canadian corporate headquarters are located in Toronto, with global headquarters located in Milan, Italy.
SOURCE Recordati Rare Diseases Canada Inc.
For further information: Media contact: Paul McCabe, Vice President and General Manager, Recordati Rare Diseases Canada Inc., 1-647-255-8830