CYSTARAN, Cysteamine HCl Form

Cysteamine (Cystaran®, Cystadrops®) ophthalmic solution is a cystine-depleting agent.
____ *For Health Insurance Marketplace (HIM) and Commercial, if request is through pharmacy benefit, Cystadrops is non-formulary and should not be approved using these criteria; refer to the formulary exception policies, HIM.PA.103 for HIM and CP.CPA.190 for Commercial.
FDA Approved Indication(s) Cystaran and Cystadrops are indicated for the treatment of corneal cystine crystal accumulation in patients with cystinosis. Policy/Criteria Provider must submit documentation (such as office chart notes, lab results or other clinical information) supporting that member has met all approval criteria.
It is the policy of health plans affiliated with Centene Corporation® that Cystaran and Cystadrops are medically necessary when the following criteria are met:
I. Initial Approval Criteria
A. Corneal Cystine Crystal Accumulation (must meet all):

1. Diagnosis of cystinosis;

   2. Prescribed by or in consultation with an ophthalmologist;
   3. Presence of corneal cystine accumulation;
   4. Dose does not exceed one of the following (a or b): a. For Cystaran (i and ii):
      i. 1 drop in each eye every hour while awake; ii. 1 bottle per week;
      b. For Cystadrops (i and ii):
      i. 1 drop in each eye 4 times a day while awake;
      ii. 1 bottle per week.
      Approval duration:
      Commercial/HIM – 6 months for Cystaran (for Cystadrops, refer to HIM.PA.103 for HIM and CP.CPA.190 for Commercial) Medicaid – 6 months Page 1 of 5

   CLINICAL POLICY Cysteamine Ophthalmic B. Other diagnoses/indications (must meet 1 or 2):

2. If this drug has recently (within the last 6 months) undergone a label change (e.g., newly approved indication, age expansion, new dosing regimen) that is not yet reflected in this policy, refer to one of the following policies (a or b): a. For drugs on the formulary (commercial, health insurance marketplace) or PDL (Medicaid), the no coverage criteria policy for the relevant line of business: CP.CPA.190 for commercial, HIM.PA.33 for health insurance marketplace, and CP.PMN.255 for Medicaid; or b. For drugs NOT on the formulary (commercial, health insurance marketplace) or PDL (Medicaid), the non-formulary policy for the relevant line of business: CP.CPA.190 for commercial, HIM.PA.103 for health insurance marketplace, and CP.PMN.16 for Medicaid; or
3. If the requested use (e.g., diagnosis, age, dosing regimen) is NOT specifically listed under section III (Diagnoses/Indications for which coverage is NOT authorized) AND criterion 1 above does not apply, refer to the off-label use policy for the relevant line of business: CP.CPA.09 for commercial, HIM.PA.154 for health insurance marketplace, and CP.PMN.53 for Medicaid.
   II. Continued Therapy A. Corneal Cystine Crystal Accumulation (must meet all):
4. Member meets one of the following (a or b): a. Currently receiving medication via Centene benefit or member has previously met initial approval criteria; b. Member is currently receiving medication and is enrolled in a state and product with continuity of care regulations (refer to state specific addendums for CC.PHARM.03A and CC.PHARM.03B);
   2. Member is responding positively to therapy;
   3. If request is for a dose increase, new dose does not exceed one of the following (a or b): a. For Cystaran (i and ii):
      i. 1 drop in each eye every hour while awake; ii. 1 bottle per week; b. For Cystadrops (i and ii):
      i. 1 drop in each eye 4 times a day while awake;
      ii. 1 bottle per week.
      Approval duration: Commercial/HIM – 12 months for Cystaran (for requests for Cystadrops, refer to HIM.PA.103 for HIM and CP.CPA.190 for Commercial) Medicaid – 12 months B. Other diagnoses/indications (must meet 1 or 2):

5. If this drug has recently (within the last 6 months) undergone a label change (e.g., newly approved indication, age expansion, new dosing regimen) that is not yet reflected in this policy, refer to one of the following policies (a or b): a. For drugs on the formulary (commercial, health insurance marketplace) or PDL (Medicaid), the no coverage criteria policy for the relevant line of business: Page 2 of 5

   CLINICAL POLICY Cysteamine Ophthalmic CP.CPA.190 for commercial, HIM.PA.33 for health insurance marketplace, and CP.PMN.255 for Medicaid; or b. For drugs NOT on the formulary (commercial, health insurance marketplace) or PDL (Medicaid), the non-formulary policy for the relevant line of business: CP.CPA.190 for commercial, HIM.PA.103 for health insurance marketplace, and CP.PMN.16 for Medicaid; or

6. If the requested use (e.g., diagnosis, age, dosing regimen) is NOT specifically listed under section III (Diagnoses/Indications for which coverage is NOT authorized) AND criterion 1 above does not apply, refer to the off-label use policy for the relevant line of business: CP.CPA.09 for commercial, HIM.PA.154 for health insurance marketplace, and CP.PMN.53 for Medicaid.
   III. Diagnoses/Indications for which coverage is NOT authorized:
   A. Non-FDA approved indications, which are not addressed in this policy, unless there is sufficient documentation of efficacy and safety according to the off label use policies – CP.CPA.09 for commercial, HIM.PA.154 for health insurance marketplace, and CP.PMN.53 for Medicaid, or evidence of coverage documents.
   IV. Appendices/General Information Appendix A: Abbreviation/Acronym Key FDA: Food and Drug Administration Appendix B: Therapeutic Alternatives
   Not applicable
   Appendix C: Contraindications/Boxed Warnings None reported
   V. Dosage and Administration
   Drug Name Dosing Regimen Cystaran (cysteamine) Cystadrops (cysteamine) 1 drop in each eye every waking hour 1 drop in each eye, 4 times a day during waking hours Dose Limit/ Maximum Dose 1 drop/eye/hour during waking hours See dosing regimen VI. Product Availability
   Drug Name Cystaran (cysteamine) Cystadrops (cysteamine) Availability Ophthalmic solution: 6.5 mg/mL of cysteamine hydrochloride equivalent to 4.4 mg/mL of cysteamine (0.44%) Ophthalmic solution containing 3.8 mg/mL of cysteamine (0.37%) in 5 mL bottle VII.