Appeal type: Medical necessity
Decision: Overturned

Diagnosis: Pemphigus vulgaris.
Treatment: Kesimpta for the treatment of pemphigus vulgaris.

The insurer denied coverage for Kesimpta for the treatment of pemphigus vulgaris. The denial is overturned.

This is a female patient with a history of pemphigus vulgaris. The patient has severe disease, with involvement of her trunk, extremities, gastrointestinal (GI) tract, face, and mouth. The patient's disease is associated with significant morbidity requiring multiple hospitalizations. The patient has previously attempted prednisone, intravenous immunoglobulin (IVIG), mycophenolate, methotrexate, and rituximab. Rituximab resulted in a severe allergic reaction. The requested medication is Kesimpta (ofatumumab). 

According to Rapp et al. (2018), "Rituximab is a chimeric anti-CD20 monoclonal antibody that is very effective in treating patients with pemphigus vulgaris. Though infrequent, the development of human anti-chimeric antibodies in patients receiving rituximab results in loss of efficacy. Ofatumumab is a second-generation fully-human anti-CD20 monoclonal antibody currently used to treat chronic lymphocytic leukemia. We report a case of a patient with pemphigus vulgaris successfully treated with ofatumumab after developing human anti-chimeric antibodies to rituximab." Klufas et al. (2020)'s case report, further supports that there is "further evidence for ofatumumab as an effective alternative treatment for pemphigus vulgaris in patients who do not respond to or do not tolerate rituximab."

In this case, the patient has severe pemphigus vulgaris, an autoimmune blistering disease with high morbidity that if inadequately treated can lead to death. The patient has tried and failed standard treatment options including oral corticosteroids, systemic steroid sparing agents such as methotrexate and mycophenolate. The patient has also developed an anaphylactic reaction to rituximab, the only Food and Drug (FDA) approved agent for severe, refractory pemphigus. Ofatumumab is a fully human, second-generation, high-affinity anti-CD20 monoclonal antibody approved for treatment of chronic lymphocytic leukemia. Because of the rarity of pemphigus, higher quality evidence is not available. Ofatumumab, with a similar mechanism of action to rituximab has been show in case reports to also be effective for the treatment of refractory pemphigus. The authors of these studies postulated that due to the fully human quality of ofatumumab, it may have less immunogenicity than rituximab, possibly explaining why it has been tolerated by patients who experienced adverse reactions to rituximab (Klufas et al., 2020; Rapp et al., 2018). 

Given the severity of this patient's disease and the failure of all other options for her pemphigus condition, Kesimpta (ofatumumab) is considered medically necessary for this patient.     

The health plan did not act reasonably with sound medical judgment, and in the best interest of the patient.                                               

Based on the above, the medical necessity for Kesimpta for the treatment of pemphigus vulgaris is substantiated. The insurer's denial should be overturned.