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Date Printed: December 18, 2017: 03:08 PM

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This medical policy (medical coverage guideline) is Copyright 2017, Blue Cross and Blue Shield of Florida (BCBSF). All Rights Reserved. You may not copy or use this document or disclose its contents without the express written permission of BCBSF. The medical codes referenced in this document may be proprietary and owned by others. BCBSF makes no claim of ownership of such codes. Our use of such codes in this document is for explanation and guidance and should not be construed as a license for their use by you. Before utilizing the codes, please be sure that to the extent required, you have secured any appropriate licenses for such use. Current Procedural Terminology (CPT) is copyright 2017 American Medical Association. All Rights Reserved. No fee schedules, basic units, relative values, or related listings are included in CPT. The AMA assumes no liability for the data contained herein. Applicable FARS/DFARS restrictions apply to government use. CPT® is a trademark of the American Medical Association. The use of specific product names is illustrative only. It is not intended to be a recommendation of one product over another, and is not intended to represent a complete listing of all products available.

09-J2000-02

Original Effective Date: 09/15/13

Reviewed: 11/09/16

Revised: 12/15/16

Subject: Cysteamine Bitartrate (Procysbi™) Capsules

THIS MEDICAL COVERAGE GUIDELINE IS NOT AN AUTHORIZATION, CERTIFICATION, EXPLANATION OF BENEFITS, OR A GUARANTEE OF PAYMENT, NOR DOES IT SUBSTITUTE FOR OR CONSTITUTE MEDICAL ADVICE. ALL MEDICAL DECISIONS ARE SOLELY THE RESPONSIBILITY OF THE PATIENT AND PHYSICIAN. BENEFITS ARE DETERMINED BY THE GROUP CONTRACT, MEMBER BENEFIT BOOKLET, AND/OR INDIVIDUAL SUBSCRIBER CERTIFICATE IN EFFECT AT THE TIME SERVICES WERE RENDERED. THIS MEDICAL COVERAGE GUIDELINE APPLIES TO ALL LINES OF BUSINESS UNLESS OTHERWISE NOTED IN THE PROGRAM EXCEPTIONS SECTION.

           

Dosage/ Administration

Position Statement

Billing/Coding

Reimbursement

Program Exceptions

Definitions

   

       

Related Guidelines

Other

References

Updates

 

Previous Versions

       

   

DESCRIPTION:

Cystinosis is a rare genetic condition causing cystine accumulation in lysosomes throughout the body. A buildup of cystine in lysosomes leads to tissue destruction and can cause kidney failure, slow body growth and small stature, and ocular symptoms; the condition can be fatal if not treated in early childhood. Current treatment options for cystinosis include cysteamine bitartrate (Cystagon) immediate release tablets and cysteamine (Cystaran) ophthalmic solution. An estimated 500 patients in the United States are affected with cystinosis.

Cysteamine bitartrate (Procysbi) was approved by the U.S. Food and Drug Administration (FDA) on April 30, 2013 for the management of nephropathic cystinosis in children and adults. Prior to FDA approval, delayed release cysteamine received orphan drug status for the treatment of nephropathic cystinosis. Cysteamine decreases the amount of cystine in the lysosomes of individuals with cystinosis by converting cystine to cysteine and a cysteine-cysteamine complex – both of which are more easily transported out of the lysosome than cystine. Clinically, this slows the progression of renal failure.

The safety and efficacy of delayed release cysteamine bitartrate was evaluated in an noninferiority, randomized, crossover trial of subjects (mean age, 12 years; range, 6 to 26 years) with nephropathic cystinosis (n=43). Subjects were required to be on a stable every-6-hour dose of immediate release cysteamine prior to randomization to delayed release cysteamine bitartrate every 12 hours. The primary endpoint was depletion of WBC cystine levels. A comparison of WBC cystine levels (nmol ½ cystine/mg protein) in a per-protocol population (n=39) is seen below:

 

Immediate release cysteamine

Delayed release cysteamine

WBC cystine level (LS mean ± SE)

0.44 ± 0.06

0.52 ± 0.06

Treatment effect (LS mean ± SE)

0.08 ± 0.03 (95.8% CI: 0.01 to 0.15; p<0.0001)

Delayed release cysteamine was determined to be non-inferior to immediate release cysteamine in regard to the depletion of WBC cystine levels. The most commonly reported adverse reactions associated with delayed release cysteamine were vomiting, abdominal pain, diarrhea, breath odor, and skin odor.

POSITION STATEMENT:

Initiation and continuation of cysteamine bitartrate (Procysbi) delayed release capsules meets the definition of medical necessity for members meeting ALL of the following criteria:

  1. Diagnosis of nephropathic cystinosis
  2. Treatment failure or intolerance to cysteamine bitartrate (Cystagon) immediate release capsules – documentation must be submitted
  3. Age of 2 years or older
  4. Dosage will be achieved using the fewest number of tablets per day

Duration of approval: 1 year

DOSAGE/ADMINISTRATION:

THIS INFORMATION IS PROVIDED FOR INFORMATIONAL PURPOSES ONLY AND SHOULD NOT BE USED AS A SOURCE FOR MAKING PRESCRIBING OR OTHER MEDICAL DETERMINATIONS. PROVIDERS SHOULD REFER TO THE MANUFACTURER’S FULL PRESCRIBING INFORMATION FOR DOSAGE GUIDELINES AND OTHER INFORMATION RELATED TO THIS MEDICATION BEFORE MAKING ANY CLINICAL DECISIONS REGARDING ITS USAGE.

FDA-approved: indicated for the treatment of nephropathic cystinosis in adult and pediatric patients 2 years of age and older.

Switching from immediate release to delayed release cysteamine

Total daily dose of delayed release cysteamine equal to previous total daily dose of immediate release cysteamine

Cysteamine-naïve

Starting Dose: 0.2 to 0.3 grams/m2 per day divided into two doses given every 12 hours or weight based dosing based on a fraction of the maintenance dose. See the prescribing information to determine the starting dose as a fraction of the maintenance dose of delayed release cysteamine. Increase the dose gradually over 4 to 6 weeks until the maintenance dose is achieved.

Maintenance Dose: 1.3 gram/m2/day, in two divided doses every 12 hours

Dose Adjustments

May titrate dose based on WBC cystine or plasma cysteamine levels. If a dose adjustment is required, increase the dose by 10%. Do not exceed 1.95 grams/m2 per day due to an increased risk of adverse reactions.

Drug Availability

25 and 75 mg delayed-release capsules

PRECAUTIONS:

Boxed Warning

None

Contraindications

Hypersensitivity to penicillamine or cysteamine.

Precautions/Warnings

BILLING/CODING INFORMATION:

The following codes may be used to describe:

HCPCS Coding:

J8499

Prescription drug, oral, non-chemotherapeutic, Not otherwise specified

ICD-10 Diagnoses Codes That Support Medical Necessity: (Effective 10/01/15)

E72.04

Cystinosis

REIMBURSEMENT INFORMATION:

Refer to section entitled POSITION STATEMENT.

PROGRAM EXCEPTIONS:

Federal Employee Program (FEP): Follow FEP guidelines.

State Account Organization (SAO): Follow SAO guidelines.

Medicare Part D: BCBSF has delegated to Prime Therapeutics authority to make coverage determinations for the Medicare Part D services referenced in this guideline.

Medicare Advantage: No National Coverage Determination (NCD) and/or Local Coverage Determination (LCD) were found at the time of the last guideline revised date.

DEFINITIONS:

None

RELATED GUIDELINES:

None

OTHER:

None

REFERENCES:

  1. AHFS Drug Information. Bethesda (MD): American Society of Health-System Pharmacists, Inc; 2014 [cited 2014 Nov 12]. In: STAT!Ref Online Electronic Medical Library [Internet]. Available from: http://online.statref.com/.
  2. Clinical Pharmacology [Internet]. Tampa (FL): Gold Standard, Inc; 2016 [cited 2016 Oct 26]. Available from: http://www.clinicalpharmacology.com/.
  3. ClinicalTrials.gov [Internet]. Bethesda (MD): National Library of Medicine; 2000 Feb 29 - [cited 2014 Nov 12]. Available from: http://clinicaltrials.gov/.
  4. DRUGDEX® System [Internet]. Greenwood Village (CO): Thomson Micromedex; Updated periodically [cited 2016 Oct 26]. Available from: http://www.thomsonhc.com/.
  5. Orphan Drug Designations and Approval [Internet]. Silver Spring (MD): US Food and Drug Administration; 2016 [cited 2016 Oct 26]. Available from: http://www.accessdata.fda.gov/scripts/opdlisting/oopd/index.cfm/.
  6. Raptor Therapeutics, Inc. Procysbi Delayed Release (cysteamine bitartrate)capsule, delayed release pellets. 2016 [cited 2016 Oct 26]. In: DailyMed [Internet]. Bethesda (MD): National Library of Medicine. Available from: http://dailymed.nlm.nih.gov/dailymed/lookup.cfm?setid=b7b6e290-5168-46dc-9e7f-5995420ec1c1/.

COMMITTEE APPROVAL:

This Medical Coverage Guideline (MCG) was approved by the BCBSF Pharmacy Policy Committee on 11/09/16.

GUIDELINE UPDATE INFORMATION:

09/15/13

New Medical Coverage Guideline.

01/15/15

Review and revision to guideline; consisting of position statement, references.

12/15/16

Revision to guideline; consisting of updating position statement, precautions, coding, and references.

Date Printed: December 18, 2017: 03:08 PM