Subject: Cystaran-Cystadrops SGM 2090-A P2025

Drug

CYSTARAN  (cysteamine ophthalmic solution)

CYATADROPS  (cysteamine ophthalmic solution)

Policy:

Indications

The indications below including FDA-approved indications and compendial uses are considered a covered benefit provided that all the approval criteria are met and the member has no exclusions to the prescribed therapy.

FDA-approved Indications¹

Cystaran

Cystaran is indicated for the treatment of corneal cystine crystal accumulation in patients with cystinosis.

Cystadrops

Cystadrops is indicated for the treatment of corneal cystine crystal deposits in adults and children with cystinosis.

All other indications are considered experimental/investigational and not medically necessary.

Documentation

Submission of the following information is necessary to initiate the prior authorization review: assay detecting increased cystine concentration in leukocytes or genetic testing results supporting the diagnosis.

Prescriber Specialties

This medication must be prescribed by or in consultation with an ophthalmologist or a physician who specializes in the treatment of metabolic disease and/or lysosomal storage disorders.

Coverage Criteria

Cystinosis^1,2

Authorization of 12 months may be granted for treatment of corneal cystine crystal accumulation when all of the following criteria are met:

• Diagnosis of cystinosis was confirmed by the presence of increased cystine concentration in leukocytes or by genetic testing; and
• Member has corneal cystine crystal accumulation.

Continuation of Therapy

Authorization of 12 months may be granted for continued treatment in members requesting reauthorization for an indication listed in the Coverage Criteria section who are responding to therapy met by either of the following criteria:

• Member has experienced a decrease in corneal cystine crystal accumulation; or
• Member did not experience an increase in corneal cystine crystal accumulation.

Place of Service:

Outpatient

The above policy is based on the following references:

1. Cystaran [package insert]. Gaithersburg, MD: Leadiant Biosciences, Inc.; February 2022.
2. Cystadrops [package insert]. Lebanon, NJ: Recordati Rare Diseases Inc.; August 2020.
3. Ivanova E, De Leo MG, De Matteis MA, Levtchenko E. Cystinosis: clinical presentation, pathogenesis, and treatment. Pediatr Endocrinol Rev. 2014;12(1):176-184.

Copyright Aetna Inc. All rights reserved. Pharmacy Clinical Policy Bulletins are developed by Aetna to assist in administering plan benefits and constitute neither offers of coverage nor medical advice. This Clinical Policy Bulletin contains only a partial, general description of plan or program benefits and does not constitute a contract. Aetna does not provide health care services and, therefore, cannot guarantee any results or outcomes. Participating providers are independent contractors in private practice and are neither employees nor agents of Aetna or its affiliates. Treating providers are solely responsible for medical advice and treatment of members. This Clinical Policy Bulletin may be updated and therefore is subject to change.

July 22, 2025