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Sacrosidase

Published
Topics: MedicineNew MedicinesNHS England commissioned new medicinesNutritional and metabolic disorders
Contents
  1. dm+d
  2. New Medicines
    1. Sucraid · Congenital sucrase-isomaltase deficiency

dm+d

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New Medicines

SucraidCongenital sucrase-isomaltase deficiency

Information

Sucraid
New molecular entity
Sucraid
QOL Medical

Development and Regulatory status

Unknown
Unknown
Launched
Yes

Jan 17: No information on company website re plans for licensing in the EU [6].

Aug 13: Granted orphan designation (EU/3/13/1183) in the EU for treatment of congenital sucrase-isomaltase deficiency [2].

Jul 98: Launched in the US [1].

Category

An enzyme replacement therapy for the missing enzyme responsible for CSID, a glycoside hydrolase.
CSID is an autosomal recessive disease characterised by a complete or almost complete lack of sucrase activity, a marked reduction in isomaltase activity and a moderate decrease in maltase activity. Children and infants with the disease have chronic diarrhoea and failure to thrive. The only current treatment is life-long adherence to a sucrose-free, low carbohydrate diet [1]. CSID affects approximately 2 in 10,000 people in the EU [2].
Congenital sucrase-isomaltase deficiency
Oral

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 [5].

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 [4].

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 [4].

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 [3].