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ph-1113

Procysbi (cysteamine bitartrate) Prior Authorization Program Summary

Policy Number: PH-1113

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#
Procysbi® (cysteamine bitartrate delayed release) Oral capsule Oral granules	Treatment of nephropathic cystinosis in adults and pediatric patients 1 year of age and older		1

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

CLINICAL RATIONALE

Cystinosis	Cystinosis is a rare autosomal recessive lysosomal storage disorder in which cystine accumulates in lysosomes of cells. It is a systemic disease wherein cystine crystals accumulate in all body cells and tissues. Cystinosis has three major clinical presentations depending on the severity of mutations affecting the CTNS gene: the infantile nephropathic form, the juvenile nephropathic form, and the adult (non-nephropathic ocular) form. The infantile nephropathic form is the most common and severe form, occurring in over 95% of patients, with consecutively progressive loss of glomerular function leading to end-stage renal disease (ESRD). ESRD usually develops by the end of the first or second decade of life. Nearly all nephropathic cystinosis patients will develop major extra-renal symptoms including retinal, endocrine, and neuromuscular complications by 30 years of age if cystine depletion therapy is not initiated early.(2) Cysteamine, in combination with symptomatic care, is the standard of care for patients with cystinosis. Cysteamine can deplete the intralysosomal cystine through the reduction of cystine, and the formation of cysteine and a cysteamine-cysteine mixed disulfide which exits the lysosome via the cationic amino acid transporter PQLC2, thus bypassing the original genetic and biochemical defects of the disease. Treatment with cysteamine improves overall prognosis by delaying progression to ESRD, preventing hypothyroidism and extra-renal complications, and improves growth in affected children. Therefore, cysteamine treatment should be started as soon as possible and needs to be continued lifelong.(2)
Efficacy	A multicenter, open-label, randomized clinical trial was completed comparing Procysbi to immediate-release cysteamine bitartrate (Cystagon). All patients were required to be on a stable dose of immediate-release cysteamine bitartrate prior to randomization. The study demonstrated that Procysbi administered every 12 hours was non-inferior to immediate-release cysteamine bitartrate dosed every 6 hours.(1)
Safety	Procysbi is contraindicated in patients with a serious hypersensitivity reaction, including anaphylaxis, to penicillamine or cysteamine.(1)

REFERENCES

Number	Reference
1	Procysbi prescribing information. Horizon Therapeutics USA, Inc. February 2022.
2	Elmonem MA, Veys KR, Soliman NA, Van Dyck M, Van Den Heuvel LP, Levtchenko E. Cystinosis: a review. Orphanet Journal of Rare Diseases. 2016;11(1). doi:10.1186/s13023-016-0426-y

POLICY AGENT SUMMARY PRIOR AUTHORIZATION

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

Procysbi	cysteamine bitartrate cap delayed release	25 MG ; 75 MG	M ; N ; O ; Y	N
Procysbi	cysteamine bitartrate delayed release granules packet	300 MG ; 75 MG	M ; N ; O ; Y	N

CLIENT SUMMARY – PRIOR AUTHORIZATION

Target Brand Agent Name(s)	Target Generic Agent Name(s)	Strength	Client Formulary
Procysbi	cysteamine bitartrate cap delayed release	25 MG ; 75 MG	Blue Partner ; Commercial ; GenPlus ; Health Insurance Marketplace ; NetResults A Series ; SourceRx
Procysbi	cysteamine bitartrate delayed release granules packet	300 MG ; 75 MG	Blue Partner ; Commercial ; GenPlus ; Health Insurance Marketplace ; NetResults A Series ; SourceRx

PRIOR AUTHORIZATION CLINICAL CRITERIA FOR APPROVAL

Module	Clinical Criteria for Approval
PA	Target Agent(s) will be approved when ALL of the following are met: ONE of the following: The patient has a diagnosis of nephropathic cystinosis OR The patient has another FDA labeled indication for the requested agent and route of administration OR The patient has an indication that is supported in compendia for the requested agent and route of administration AND If the patient has an FDA labeled indication, then ONE of the following: The patient’s age is within FDA labeling for the requested indication OR There is support for using the requested agent for the patient’s age for the requested indication AND ONE of the following: The patient has tried and had an inadequate response to Cystagon (immediate release cysteamine) OR The patient has an intolerance or hypersensitivity to Cystagon that is not expected to occur with the requested agent OR The patient has an FDA labeled contraindication to Cystagon that is not expected to occur with the requested agent AND The prescriber is a specialist in the area of the patient’s diagnosis (e.g., nephrologist), 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 Compendia Allowed: AHFS or DrugDex 1 or 2a level of evidence Length of Approval: 12 months *Step therapy requirement may not apply if a prior health plan paid for the medication - documentation of a paid claim may be required.

Module

Clinical Criteria for Approval

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

ONE of the following:
1. The patient has a diagnosis of nephropathic cystinosis OR
2. The patient has another FDA labeled indication for the requested agent and route of administration OR
3. The patient has an indication that is supported in compendia for the requested agent and route of administration AND
If the patient has an FDA labeled indication, then ONE of the following:
1. The patient’s age is within FDA labeling for the requested indication OR
2. There is support for using the requested agent for the patient’s age for the requested indication AND
ONE of the following:
1. The patient has tried and had an inadequate response to Cystagon (immediate release cysteamine) OR
2. The patient has an intolerance or hypersensitivity to Cystagon that is not expected to occur with the requested agent OR
3. The patient has an FDA labeled contraindication to Cystagon that is not expected to occur with the requested agent AND
The prescriber is a specialist in the area of the patient’s diagnosis (e.g., nephrologist), 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

Compendia Allowed: AHFS or DrugDex 1 or 2a level of evidence

Length of Approval: 12 months

*Step therapy requirement may not apply if a prior health plan paid for the medication - documentation of a paid claim may be required.

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.