Background

Veopoz is a complement inhibitor, indicated for the treatment of adult and pediatric patients 1 year of age and older with CD55-deficient protein-losing enteropathy (PLE), also known as CHAPLE disease. As the first FDA approved therapy for CHAPLE disease, Veopoz is a monoclonal antibody that targets complement factor C5, a protein involved in complement system activation. Because life threatening and fatal meningococcal infections have occurred in patients treated with complement inhibitors, patients must receive or update vaccination against meningococcal infection according to the most current Advisory Committee on Immunization Practices (ACIP) recommendations at least 2 weeks prior to the first dose of Veopoz. If vaccination cannot be given at least two weeks prior to the start of therapy, the package insert recommends that patients should receive prophylactic antibiotics. Veopoz requires a weight-based intravenous loading dose, followed by a weight-based subcutaneous dose on day 8, and then weekly subcutaneous maintenance doses thereafter. The maintenance dosage may be increased once weekly if there is inadequate clinical response after at least 3 weekly doses. The maximum maintenance dosage is 800 mg subcutaneously once a week.

CHAPLE Disease (CD55-deficient protein-losing enteropathy (PLE)): Complement hyperactivation, angiopathic thrombosis, and protein-losing enteropathy (CHAPLE) disease is a rare but life-threatening immune condition caused by a biallelic loss-of-function mutation in the CD55 gene. CD55 inhibits early complement activation by accelerating the degradation of C3 convertase, a key regulator of the complement cascade, and blocking cleavage of C5 into C5a and C5b, thereby preventing the formation of the membrane-attack complex (C5b-C9, a structure mediating cell lysis). The mutation at CD55 allows overactivation of the complement system causing damage to blood and lymph vessels along the upper digestive tract and leading to a loss of circulating proteins. Patients may experience abdominal pain, diarrhea, vomiting, malabsorption, edema, delayed growth, intestinal lymphangiectasia, infections, and potentially life threatening thrombotic vascular occlusions. Onset occurs in infancy or childhood with approximately 10 patients in the US and 100 patients worldwide being affected by the disease.

There are no other FDA-approved therapies for CHAPLE disease. Soliris has been used off-label. Supportive therapies include albumin infusions, IgG replacement therapy, corticosteroids, bowel resection surgery, and vitamin and micronutrient supplements.

Policy

Veopoz is considered MEDICALLY NECESSARY for when the following criteria has been met:

1. 1. Diagnosis of active CD55-deficient protein-losing enteropathy (PLE), also known as CHAPLE disease
   2. Patient has a confirmed genotype of biallelic CD55 loss-of-function mutation
   3. Patient is 1 year of age or older
   4. Patient has hypoalbuminemia (serum albumin concentration of ≤3.2 g/dL)
   5. Patient has at least one of the following signs or symptoms within the last six months:
      • abdominal pain
      • diarrhea
      • peripheral edema
      • facial edema
   6. Individual has completed or updated meningococcal vaccination at least 2 weeks prior to administration of the first dose of Veopoz (pozelimab-bbfg), unless the risks of delaying Veopoz (pozelimab-bbfg) outweigh the risk of meningococcal infection.
   7. Prescribed by or in consultation with one of the following:
      • Immunologist
      • Geneticist
      • Hematologist

Continuation

Continuation of Veopoz is considered medically necessary when there is documentation of positive clinical response to therapy (e.g., decrease in albumin transfusions and hospitalizations, normalization of serum IgG concentrations, etc.).

References

1. Dho SH, et al. Beyond the Role of CD55 as a Complement Component. Immune Netw. 2018; 20;18(1):e11.
2. Veopoz Drug Evaluation. Express Scripts. Updated September 2023.
3. Veopoz [package insert]. Regeneron Pharmaceuticals, Inc. Tarrytown, NY. October 2023
4. Veopoz New Drug Review. IPD Analytics. Updated September 2023.
5. Ozen A, Chongsrisawat V, Sefer AP, et al. A Phase 2/3 Study Evaluating the Efficacy and Safety of Pozelimab in Patients with CD55 Deficiency with Hyperactivation of Complement, Angiopathic Thrombosis, and Protein-Losing Enteropathy (CHAPLE Disease). The Lancet PrePrint article, available at SSRN: https://ssrn.com/abstract=4485593 or http://dx.doi.org/10.2139/ssrn.4485593
6. Ozen A. CHAPLE syndrome uncovers the primary role of complement in a familial form of Waldmann's disease. Immunol Rev. 2019 Jan;287(1):20-32. doi: 10.1111/imr.12715. PMID: 30565236.
7. Ozen A, Comrie WA, Ardy RC, et al. CD55 Deficiency, Early-Onset Protein-Losing Enteropathy, and Thrombosis. N Engl J Med. 2017 Jul 6;377(1):52-61. doi: 10.1056/NEJMoa1615887. Epub 2017 Jun 28. PMID: 28657829; PMCID: PMC6690356.

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, Blue Cross Blue Shield Association technology assessment program (TEC) and other nonaffiliated technology evaluation centers, reference to federal regulations, other plan medical policies, and accredited national guidelines.

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