Effective Date

Date of Origin 

1/1/2023

Sucraid® 

(sacrosidase)

Oral solution

Oral replacement therapy for treatment of genetically determined sucrase deficiency, which is part of congenital sucrase-isomaltase deficiency (CSID)

1

Overview

Congenital sucrase-isomaltase deficiency (CSID) is a rare, chronic, autosomal recessive disorder characterized by the absence or deficiency of endogenous sucrase and isomaltase activity. Patients with CSID have two defective copies of the sucrase-isomaltase (SI) gene. The SI enzyme complex is naturally produced in the brush border of the small intestine, primarily the distal duodenum and jejunum. Unhydrolyzed sucrose and starch are not absorbed from the intestine and their presence in the intestinal lumen may lead to osmotic retention of water. This may result in diarrhea. Unabsorbed sucrose in the colon is fermented by bacterial flora to produce increased amounts of hydrogen, methane, and water. As a consequence, excessive gas, bloating, abdominal cramps, nausea, and vomiting may occur. Chronic malabsorption of disaccharides may result in malnutrition. Undiagnosed/untreated CSID patients often fail to thrive and fall behind in their expected growth and development curves.(2-4)

A number of diagnostic tests are available to support the diagnosis of CSID, with assay of duodenal biopsy specimens considered the gold standard.(2,4) Endoscopic biopsy of the small bowel should demonstrate normal small bowel morphology in the presence of decreased (or absent) sucrase activity, isomaltase activity varying from decreased to normal activity, and decreased maltase activity.(4) Genetic sequencing of the SI gene can identify homozygous and compound heterozygous mutations responsible for CSID.(2,4) A number of noninvasive diagnostic tests can also help establish the diagnosis, including the sucrose challenge test, lactose breath test, and hydrogen-methane breath test. However, many of these tests have limitations which include false-positive or false-negative results, and lack of validation data.(4)

Previously, the treatment of CSID has required the continual use of a strict sucrose-free diet and starch-restricted diets. Treatment has improved considerably with the availability of enzyme replacement therapy (sacrosidase), which is derived from baker’s yeast.(2-4) Access to a physician or dietician who is knowledgeable about CSID is essential for guiding patients and their families.(4)

Efficacy(1, 5)

The efficacy of Sucraid was studied in a two-phase (dose response preceded by a breath hydrogen phase) double blind, multi-site, crossover trial conducted in 28 patients with confirmed CSID. Patients showed a marked decrease in breath hydrogen output when they received sacrosidase in comparison to placebo in the breath hydrogen phase. This first phase consisted of three sucrose breath hydrogen tests with three single-dose treatments (placebo, sacrosidase, and sacrosidase plus milk), each test undergone after 1 week of a sucrose-free, low starch diet and a 12-hour fasting interval. At the conclusion of the breath tests, each patient underwent another week of a sucrose-free, low-starch diet before beginning the second phase, dose response. During the dose response phase, the patients were challenged with an ordinary sucrose-containing diet while receiving each of four doses of sacrosidase: full strength (9000 IU/mL) and three dilutions (1:10, 1:100, and 1:1000) in random order for a period of 10 days. The primary efficacy endpoints were fewer total stools and lower total symptom scores. Symptom scores were based on severity of symptoms (gas, bloating, nausea, vomiting, and abdominal cramps) recorded daily by each patient. The symptoms were assigned values of 0 = none, 1 = mild, 2 = moderate, and 3 = severe.  A dose response relationship was shown between the two higher and the two lower doses, with the two higher doses meeting the primary efficacy endpoints.

Safety(1)

Sucraid is contraindicated in patients known to be hypersensitive to yeast, yeast products, glycerin (glycerol), or papain.

1

Sucraid prescribing information. QOL Medical, LLC. May 2022.

2

Treem WR. Clinical Aspects and Treatment of Congenital Sucrase-Isomaltase Deficiency. J Pediatr Gastroenterol Nutr. 2012;55:S7-S13.

3

National Organization for Rare Disorders (NORD). Congenital Sucrase-Isomaltase Deficiency. Rare Disease Database; 2005. Available at: https://rarediseases.org/rare-diseases/disaccharide-intolerance-i/. 

4

Chey WD, Cash B, Lembo A, et al. Congenital Sucrase-Isomaltase Deficiency: What, When, and How? Gastroenterol Hepatol. 2020;16(10):1-11.

5

Treem WR, McAdams L, Stanford L, et al. Sacrosidase Therapy for Congenital Sucrase-Isomaltase Deficiency, J Pediatr Gastroenterol Nutr. 1999;28(2):137-142

SUCRAID*sacrosidase soln

8500 UNIT/ML

M ; N ; O ; Y

N

512000600020

SUCRAID*sacrosidase soln

8500 UNIT/ML

300.0

MLS

30

Days

SUCRAID*sacrosidase soln

8500 UNIT/ML

Blue Partner ; Commercial ; GenPlus ; Health Insurance Marketplace ; NetResults A Series ; SourceRx

SUCRAID*sacrosidase soln

8500 UNIT/ML

Blue Partner ; Commercial ; GenPlus ; Health Insurance Marketplace ; NetResults A Series ; SourceRx

Initial Evaluation

Target Agent will be approved when ALL of the following are met:

1. The patient has a diagnosis of congenital sucrase-isomaltase deficiency (CSID) confirmed by ONE of the following:
   1. Genetic testing of the sucrase-isomaltase (SI) gene indicates a pathogenic mutation OR
   2. Endoscopic biopsy of the small bowel indicates normal small bowel morphology in the presence of decreased (or absent) sucrase activity, isomaltase activity varying from decreased to normal activity, and decreased maltase activity AND
2. The prescriber is a specialist in the area of the patient’s diagnosis (e.g., gastroenterologist, endocrinologist) or the prescriber has consulted with a specialist in the area of the patient’s diagnosis AND
3. The patient does NOT have any FDA labeled contraindications to the requested agent

Length of Approval:  3 months

NOTE: If Quantity Limit applies, please refer to Quantity Limit Criteria.

Renewal Evaluation

Target Agent will be approved when ALL of the following are met:

1. The patient has been previously approved for the requested agent through the plan’s Prior Authorization process AND
2. The patient has had clinical benefit with the requested agent AND
3. The prescriber is a specialist in the area of the patient’s diagnosis (e.g., gastroenterologist, endocrinologist) or the prescriber has consulted with a specialist in the area of the patient’s diagnosis AND
4. The patient does NOT have any FDA labeled contraindications to the requested agent AND

Length of Approval:  12 months

NOTE: If Quantity Limit applies, please refer to Quantity Limit Criteria.

QL with PA

Quantity Limit for the Target Agent(s) will be approved when ONE of the following is met:

1. The requested quantity (dose) does NOT exceed the program quantity limit OR
2. ALL of the following:
   1. The requested quantity (dose) is greater than the program quantity limit AND
   2. The requested quantity (dose) does NOT exceed the maximum FDA labeled dose for the requested indication AND
   3. The requested quantity (dose) cannot be achieved with a lower quantity of a higher strength that does not exceed the program quantity limit OR
3. ALL of the following:
   1. The requested quantity (dose) is greater than the program quantity limit AND
   2. The requested quantity (dose) is greater than the maximum FDA labeled dose for the requested indication AND
   3. The prescriber has provided information in support of therapy with a higher dose for the requested indication

Length of Approval:  Initial: 3 months; Renewal: 12 months