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ph-1144

Sucraid (sacrosidase) Prior Authorization with Quantity Limit Program Summary

Policy Number: PH-1144

This program applies to Blue Partner, Commercial, GenPlus, NetResults A series, SourceRx and Health Insurance Marketplace formularies.

POLICY REVIEW CYCLE

Effective Date	Date of Origin
01-01-2025

FDA LABELED INDICATIONS AND DOSAGE

Agent(s)	FDA Indication(s)	Notes	Ref#
Sucraid® (sacrosidase) Oral solution	Oral replacement therapy for treatment of genetically determined sucrase deficiency, which is part of congenital sucrase-isomaltase deficiency (CSID)		1

Agent(s)

FDA Indication(s)

Notes

Ref#

Sucraid®

(sacrosidase)

Oral solution

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

See package insert for FDA prescribing information: https://dailymed.nlm.nih.gov/dailymed/index.cfm

CLINICAL RATIONALE

CSID

Congenital sucrase-isomaltase deficiency (CSID) is a rare, chronic, autosomal recessive disorder characterized by the absence or deficiency of the enzymes sucrase and isomaltase.(3) Patients with CSID have two defective copies of the sucrase-isomaltase (SI) gene. The SI enzyme complex is naturally produced in the brush border lining of the small intestine and assists in the breakdown of certain sucrose and products of starch digestion (dextrins). When sucrase-isomaltase is absent or deficient, non-absorbed carbohydrates enter the distal small intestine and colon where they are fermented, leading to the excessive production of short-chain fatty acids and gases such as hydrogen, methane, and hydrogen sulfide. This in turn can lead to abdominal distension, cramping, pain, excessive flatulence, nausea/vomiting, and osmotic diarrhea. If left untreated, significant sucrase-isomaltase deficiency (SID) can result in inadequate growth and failure to thrive in children as well as weight loss in adults.(4)

The gold standard for the diagnosis of CSID remains small intestinal biopsy specimens assayed for lactase, sucrase, isomaltase, and maltase activity. Criteria to make the diagnosis of CSID include normal small bowel morphology in the presence of markedly reduced or absent sucrase activity, isomaltase activity varying from zero to full activity, and reduced maltase activity. Lactase activity can be normal or reduced in children with a sucrase:lactase ratio of less than 1.0. Genetic sequencing of the SI gene can identify homozygous and compound heterozygous mutations responsible for CSID. 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 including false-positive results, false-negative results, and lack of validation data.(2)

Previously, treatment of CSID has required lifelong adherence to a sucrose-free diet.(2-4) Data suggest that even after diagnosis and dietary treatment, major gastrointestinal symptoms persists, and there is a high frequency of decreased weight for height and age in these patients.(5) Treatment has improved considerably with the availability of enzyme replacement therapy (sacrosidase) which has allowed consumption of a more normal diet and decreased the high incidence of chronic gastrointestinal problems.(2-5) Access to a physician or dietician who is knowledgeable about CSID is essential for guiding patients and their families.(4)

Safety

Sucraid is contraindicated in patients known to be hypersensitive to yeast, yeast products, glycerin (glycerol), or papain.(1)

REFERENCES

Number	Reference
1	Sucraid prescribing information. QOL Medical, LLC. December 2023.
2	Treem WR. Clinical Aspects and Treatment of Congenital Sucrase‐Isomaltase Deficiency. Journal of Pediatric Gastroenterology and Nutrition. 2012;55(S2). doi:10.1097/01.mpg.0000421401.57633.90
3	Congenital Sucrase-Isomaltase Deficiency - Symptoms, Causes, treatment \| NORD. National Organization for Rare Disorders. https://rarediseases.org/rare-diseases/disaccharide-intolerance-i/
4	Congenital Sucrase-Isomaltase Deficiency: What, when, and how? – Gastroenterology & Hepatology. https://www.gastroenterologyandhepatology.net/supplements/congenital-sucrase-isomaltase-deficiency-what-when-and-how/
5	Treem, W. R., McAdams, L., Stanford, L., Kastoff, G., Justinich, C., & Hyams, J. (1999). Sacrosidase therapy for Congenital Sucrase-Isomaltase Deficiency. Journal of Pediatric Gastroenterology and Nutrition, 28(2), 137–142. https://doi.org/10.1097/00005176-199902000-00008

POLICY AGENT SUMMARY PRIOR AUTHORIZATION

Target Brand Agent(s)	Target Generic Agent(s)	Strength	Targeted MSC	Available MSC	Final Age Limit	Preferred Status

Sucraid	sacrosidase soln	8500 UNIT/ML	M ; N ; O ; Y	N

POLICY AGENT SUMMARY QUANTITY LIMIT

Target Brand Agent Name(s)	Target Generic Agent Name(s)	Strength	QL Amount	Dose Form	Day Supply	Duration	Addtl QL Info	Allowed Exceptions	Targeted NDCs When Exclusions Exist

Sucraid	sacrosidase soln	8500 UNIT/ML	300	mLs	30	DAYS

CLIENT SUMMARY – PRIOR AUTHORIZATION

Target Brand Agent Name(s)	Target Generic Agent Name(s)	Strength	Client Formulary
Sucraid	sacrosidase soln	8500 UNIT/ML	Blue Partner ; Commercial ; GenPlus ; Health Insurance Marketplace ; NetResults A Series ; SourceRx

CLIENT SUMMARY – QUANTITY LIMITS

Target Brand Agent Name(s)	Target Generic Agent Name(s)	Strength	Client Formulary
Sucraid	sacrosidase soln	8500 UNIT/ML	Blue Partner ; Commercial ; GenPlus ; Health Insurance Marketplace ; NetResults A Series ; SourceRx

PRIOR AUTHORIZATION CLINICAL CRITERIA FOR APPROVAL

Module	Clinical Criteria for Approval
PA	Initial Evaluation Target Agent(s) will be approved when ALL of the following are met: The patient has a diagnosis of congenital sucrase-isomaltase deficiency (CSID) confirmed by ONE of the following: Genetic testing of the sucrase-isomaltase (SI) gene indicates a pathogenic mutation OR 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 The prescriber is a specialist in the area of the patient’s diagnosis (e.g., gastroenterologist, geneticist, endocrinologist), or the prescriber has consulted with a specialist in the area of the patient’s diagnosis AND 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(s) will be approved when ALL of the following are met: The patient has been previously approved for the requested agent through the plan’s Prior Authorization process [Note: patients not previously approved for the requested agent will require initial evaluation review] AND The patient has had clinical benefit with the requested agent AND The prescriber is a specialist in the area of the patient’s diagnosis (e.g., gastroenterologist, geneticist, endocrinologist), or the prescriber has consulted with a specialist in the area of the patient’s diagnosis AND 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.

Module

Clinical Criteria for Approval

Initial Evaluation

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

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
The prescriber is a specialist in the area of the patient’s diagnosis (e.g., gastroenterologist, geneticist, endocrinologist), or the prescriber has consulted with a specialist in the area of the patient’s diagnosis AND
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(s) will be approved when ALL of the following are met:

The patient has been previously approved for the requested agent through the plan’s Prior Authorization process [Note: patients not previously approved for the requested agent will require initial evaluation review] AND
The patient has had clinical benefit with the requested agent AND
The prescriber is a specialist in the area of the patient’s diagnosis (e.g., gastroenterologist, geneticist, endocrinologist), or the prescriber has consulted with a specialist in the area of the patient’s diagnosis AND
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.

QUANTITY LIMIT CLINICAL CRITERIA FOR APPROVAL

Module	Clinical Criteria for Approval
QL with PA	Quantity Limit for the Target Agent(s) will be approved when ONE of the following is met: The requested quantity (dose) does NOT exceed the program quantity limit OR The requested quantity (dose) exceeds the program quantity limit AND ONE of the following: BOTH of the following: The requested agent does NOT have a maximum FDA labeled dose for the requested indication AND There is support for therapy with a higher dose for the requested indication OR BOTH of the following: The requested quantity (dose) does NOT exceed the maximum FDA labeled dose for the requested indication AND There is support for why the requested quantity (dose) cannot be achieved with a lower quantity of a higher strength that does not exceed the program quantity limit OR BOTH of the following: The requested quantity (dose) exceeds the maximum FDA labeled dose for the requested indication AND There is support for therapy with a higher dose for the requested indication Length of Approval: up to 12 months

Module

Clinical Criteria for Approval

QL with PA

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

The requested quantity (dose) does NOT exceed the program quantity limit OR
The requested quantity (dose) exceeds the program quantity limit AND ONE of the following:
1. BOTH of the following:
  1. The requested agent does NOT have a maximum FDA labeled dose for the requested indication AND
  2. There is support for therapy with a higher dose for the requested indication OR
2. BOTH of the following:
  1. The requested quantity (dose) does NOT exceed the maximum FDA labeled dose for the requested indication AND
  2. There is support for why 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. BOTH of the following:
  1. The requested quantity (dose) exceeds the maximum FDA labeled dose for the requested indication AND
  2. There is support for therapy with a higher dose for the requested indication

Length of Approval: up to 12 months

This pharmacy policy is not an authorization, certification, explanation of benefits or a contract. Eligibility and benefits are determined on a case-by-case basis according to the terms of the member’s plan in effect as of the date services are rendered. All pharmacy policies are based on (i) information in FDA approved package inserts (and black box warning, alerts, or other information disseminated by the FDA as applicable); (ii) research of current medical and pharmacy literature; and/or (iii) review of common medical practices in the treatment and diagnosis of disease as of the date hereof. Physicians and other providers are solely responsible for all aspects of medical care and treatment, including the type, quality, and levels of care and treatment.

The purpose of Blue Cross and Blue Shield of Alabama’s pharmacy policies are to provide a guide to coverage. Pharmacy policies are not intended to dictate to physicians how to practice medicine. Physicians should exercise their medical judgment in providing the care they feel is most appropriate for their patients.

Neither this policy, nor the successful adjudication of a pharmacy claim, is guarantee of payment.

Commercial _ PS _ Sucraid_sacrosidase__PAQL _ProgSum_ 01-01-2025