Background

Zilbrysq is a subcutaneous complement C5 inhibitor (macrocyclic peptide). It’s indicated as an add-on therapy for adult patients with generalized myasthenia gravis (gMG) who are anti-acetylcholine receptor (AChR) antibody-positive. Myasthenia gravis (MG) is a relatively rare acquired autoimmune disorder caused by an antibody- mediated blockade of neuromuscular transmission resulting in skeletal muscle weakness.

Policy (Criteria)

Coverage of these drugs is provided when the criteria below is met and for non-preferred products there has been a trial and failure of preferred therapy (if applicable).

Zilucoplan may be considered MEDICALLY NECESSARY for the following condition(s):

Generalized Myasthenia Gravis (gMG)

1. Diagnosis of generalized myasthenia gravis (gMG)
2. Member is anti-acetylcholine receptor (AChR) antibody positive
3. One of the following:
   1. Trial and failure, contraindication, or intolerance to two immunosuppressive therapies (e.g., glucocorticoids, azathioprine, cyclosporine, mycophenolate mofetil, methotrexate, tacrolimus)
   2. Both of the following:
      1. Trial and failure, contraindication, or intolerance to one immunosuppressive therapy (e.g., glucocorticoids, azathioprine, cyclosporine, mycophenolate mofetil, methotrexate, tacrolimus) AND
      2. Trial and failure, contraindication, or intolerance to one of the following:
         1. Chronic plasmapheresis or plasma exchange (PE)
         2. Intravenous immunoglobulin (IVIG)
4. Prescribed by or in consultation with a neurologist

Continuation therapy is considered MEDICALLY NECESSARY when the following criteria are met:

Documentation of positive clinical response to therapy

Authorization duration: 12 months

References

1. U.S. Food and Drug Administration. (2024, [Month unspecified]). ZILBRYSQ® (zilucoplan) injection, for subcutaneous use: Highlights of prescribing information. Retrieved from https://www.accessdata.fda.gov/drugsatfda_docs/label/2024/216834s001lbl.pdf
2. Clinical Pharmacology [Internet]. Tampa (FL): Gold Standard, Inc. Accessed May 1, 2025 2.
3. DRUGDEX® System [Internet]. Greenwood Village (CO): Thomson Micromedex; Accessed May 1, 2025
4. National Organization of Rare Diseases. https://rarediseases.org/rare-diseases.
5. Orphan Drug Designations and Approval [Internet]. Silver Spring (MD): US Food and Drug Administration; 2025 [cited May 1, 2025]. Available from: http://www.accessdata.fda.gov/scripts/opdlisting/oopd/index.cfm/.
6. Zilbrysq (zilucoplan) injection. UCB, Inc. Smyrna, GA. February 2025.

UCB Press Release – FDA Approval Announcement
UCB. (2023, October 17). U.S. FDA approval of ZILBRYSQ® (zilucoplan) for the treatment of adults with generalized myasthenia gravis. Retrieved from https://www.ucb.com/newsroom/press-releases/article/ucb-announces-us-fda-approval-of-zilbrysqr-zilucoplan-for-the-treatment-of-adults-with-generalized-myasthenia-gravis

Coding Section

Procedure and diagnosis codes on Medical Policy documents are included only as a general reference tool for each Policy. They may not be all-inclusive.

This medical policy was developed through consideration of peer-reviewed medical literature generally recognized by the relevant medical community, U.S. FDA approval status, nationally accepted standards of medical practice and accepted standards of medical practice in this community, technology assessment program (TEC) and other non-affiliated technology evaluation centers, reference to federal regulations, other plan medical policies, and accredited national guidelines.

"Current Procedural Terminology © American Medical Association. All Rights Reserved" 
 

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