Recordati Rare Diseases Canada Inc., a biopharmaceutical company providing orphan therapies for patients with rare diseases, today announced the approval of CYSTADROPS, the first cysteamine solution approved by Health Canada for the treatment of corneal cystine crystal deposits in patients with cystinosis, from two years of age.
Cystinosis is an ultra-rare genetic metabolic disease that causes cystine “ an amino acid “ to accumulate in the bodys organs. It affects the kidneys, eyes, liver, muscles, pancreas, brain, and white blood cells, causing serious complications, including muscle wasting, growth delay, diabetes, hypothyroidism, and eventual kidney failure. The disease has an estimated incidence of 1 to 2 per 150,000 births, with up to 100 Canadians thought to be affected today.
Recordati is excited to launch this newly-developed viscous eyedrop for cystinosis patients, says Paul McCabe, Vice President and General Manager, Recordati Rare Diseases Canada. Until now, in Canada, there was no approved cysteamine eye drops solution for the management of ophthalmic symptoms in cystinosis patients.
Ocular manifestations of cystinosis can cause significant long-term morbidity and develop into more serious ocular complications, including blindness. Left untreated, cystine crystal deposits form in the cornea, causing light sensitivity, eye pain, involuntary closing of the eyelids, and degeneration, resulting in partial or complete loss of vision.
Unfortunately, current forms of cysteamine eyedrops require hourly application throughout the waking day, making sustained treatment difficult, says Dr. Paul Goodyer, Professor of Pediatrics and Human Genetics, McGill University Health Centre, and a leading expert on therapeutic strategies for hereditary renal disease. The recent arrival of CYSTADROPS in Canada may represent a step forward. The drug is delivered in a viscous vehicle, thought to prolong exposure at the eye surface, and is administered only four times per day. Further studies are needed to understand how to optimize CYSTADROPS use, but its stability at room temperature for one week and reduced dosing frequency could well improve eye health among Canadian cystinosis patients.
CYSTADROPS (cysteamine ophthalmic solution, DIN 02485605) is a cysteamine solution approved in Canada for the treatment of corneal cystine crystal deposits in patients with cystinosis. For more information, visit www.recordatirarediseases.ca.
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. is part of Recordati, an international pharmaceutical group committed to the research and development of new specialties with a focus on treatments for rare disease.
Recordati Rare Diseases Canadas mission is to reduce the impact of ultra-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 companys Canadian corporate headquarters are located in Toronto, with global headquarters located in Milan, Italy.
Recordati Rare Diseases Canada Inc.