SucraidCongenital sucrase-isomaltase deficiency
Development and Regulatory status
Jan 17: No information on company website re plans for licensing in the EU .
Aug 13: Granted orphan designation (EU/3/13/1183) in the EU for treatment of congenital sucrase-isomaltase deficiency .
Jul 98: Launched in the US .
Trial or other data
Jan 17: Published papers include: Lücke T et al. Congenital and putatively acquired forms of sucrase-isomaltase deficiency in infancy: effects of sacrosidase therapy. J Pediatr Gastroenterol Nutr. 2009; 49(4): 485.; Puntis JW et al. Congenital sucrase-isomaltase deficiency: diagnostic challenges and response to enzyme replacement Arch Dis Child. 2015 Sep; 100(9): 869-71 .
May 16: A phase 4 study examining the efficacy and safety of a Sucraid (sacrosidase) oral solution in comparison to placebo in 150-200 subjects 16 years or older with chronic diarrhoea possibly attributable to sucrase deficiency begins. Response to Sucraid and placebo is based on improvement in daily stool consistency, as assessed by the Bristol Stool Form Scale (BSFS) over a 1-week treatment period in subjects with chronic diarrhea and sucrase deficiency using a sucrose hydrogen methane breath test (SHMBT). Collection of these data should complete Dec 17 .
Jul 15: A generic prevalence case-control study (NCT01914003), sponsored by QOL Medical, completes. It enrolled 53 children aged up to 18 years and assessed prevalence of CSID genetic variants in subjects with a primary symptom of chronic idiopathic diarrhoea or chronic abdominal pain without constipation .
CSID can be diagnosed by taking a small sample of tissue (biopsy) from the small intestine for a specific test known as a disaccharidase assay. Other tests may include a sucrose hydrogen breath test in which an abnormally high level of hydrogen will be detected in the breath of an affected individual after sucrose ingestion. Genetic testing may be indicated in some cases .