Subject: Wilate SGM 1952-A P2025

Drug

WILATE  (von Willebrand factor/coagulation factor VIII complex [human])

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

• Wilate is indicated in children and adults with von Willebrand Disease (VWD) for:
  • On-demand treatment and control of bleeding episodes
  • Perioperative management of bleeding
  • Routine prophylaxis to reduce the frequency of bleeding episodes
• Wilate is indicated in adolescents and adults with hemophilia A for:
  • Routine prophylaxis to reduce the frequency of bleeding episodes
  • On-demand treatment and control of bleeding episodes

Compendial Uses^2,3,5

Acquired von Willebrand Syndrome

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

Prescriber Specialties

Must be prescribed by or in consultation with a hematologist.

Coverage Criteria

Von Willebrand Disease^1,2,6

Authorization of 12 months may be granted for members with VWD when either of the following criteria is met:

• Member has type 1, 2A, 2M, or 2N VWD and has had an insufficient response to desmopressin or a documented clinical reason for not using desmopressin (see Appendix B).
• Member has type 2B or type 3 VWD.

Acquired Von Willebrand Syndrome^2,3,5

Authorization of 12 months may be granted for treatment of acquired von Willebrand syndrome.

Hemophilia A^1,9

Authorization of 12 months may be granted for hemophilia A when the requested medication will be used for either of the following:

• Member has mild disease (see Appendix A) and has had an insufficient response to desmopressin or a documented clinical reason for not using desmopressin (see Appendix B).
• Member has moderate or severe disease (see Appendix A).

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 when the member is experiencing benefit from therapy (e.g., reduced frequency or severity of bleeds).

Appendix

Appendix A: Classification of Hemophilia by Clotting Factor Level (% Activity) and Bleeding Episodes⁴

Factor assay levels are required to determine the diagnosis and are of value in monitoring treatment response.

Severity	Clotting Factory Level % activity	Bleeding Episodes
Severe	<1%	Spontaneous bleeding episodes, predominantly into joints and muscles   Severe bleeding with trauma, injury or surgery
Moderate	1% to 5%	Occasional spontaneous bleeding episodes   Severe bleeding with trauma, injury or surgery
Mild	6% to 40%	Severe bleeding with serious injury, trauma or surgery

Appendix B: Clinical Reasons For Not Utilizing Desmopressin in Patients with Hemophilia A and Type 1, 2A, 2M and 2N VWD^6-9

• Age < 2 years
• Pregnancy
• Fluid/electrolyte imbalance
• High risk for cardiovascular or cerebrovascular disease (especially the elderly)
• Predisposition to thrombus formation
• Trauma requiring surgery
• Life-threatening bleed
• Contraindication or intolerance to desmopressin
• Severe type 1 von Willebrand disease
• Stimate Nasal Spray is unavailable due to backorder/shortage issues (where applicable)

Place of Service:

Outpatient

The above policy is based on the following references:

1. Wilate [package insert]. Paramus, NJ: Octapharma USA Inc.; December 2023.
2. National Institutes of Health. The diagnosis, evaluation, and management of von Willebrand disease. Bethesda, MD: US Dept of Health and Human Services, National Institutes of Health; 2007. NIH publication No. 08-5832.
3. Tiede A, Rand J, Budde U, et al. How I treat the acquired von Willebrand syndrome. Blood. 2011;117(25):6777-85.
4. Srivastava A, Santagostino E, Dougall A, et al. WFH Guidelines for the Management of Hemophilia, 3rd edition. Haemophilia. 2020 Aug;26 Suppl 6:1-158.
5. Federici A, Budde U, Castaman G, Rand J, Tiede A. Current diagnostic and therapeutic approaches to patients with acquired von Willebrand syndrome: a 2013 update. Semin Thromb Hemost. 2013;39(2):191-201.
6. National Hemophilia Foundation. MASAC Recommendations Concerning Products Licensed for the Treatment of Hemophilia and Selected Disorders of the Coagulation System. Revised April 2024. MASAC Document #284. https://www.bleeding.org/sites/default/files/document/files/MASAC-Products-Licensed.pdf. Accessed October 16, 2024.
7. National Hemophilia Foundation. MASAC recommendations regarding the treatment of von Willebrand disease. Revised February 2021. MASAC Document #266. https://www.hemophilia.org/sites/default/files/document/files/266.pdf. Accessed October 16, 2024.
8. Stimate [package insert]. King of Prussia, PA: CSL Behring LLC; June 2021.
9. Leissinger C, Carcao M, Gill JC, et al. Desmopressin (DDAVP) in the management of patients with congenital bleeding disorders. Haemophilia. 2014;20:158-167.

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.

April 20, 2025