Appeal type: Medical necessity
Decision: Overturned

Diagnosis: Spinal Muscle Atrophy (SMA)

Treatment: Medication Zolgensma

The insurer denied coverage for medication Zolgensma

The denial is overturned

This case involves an infant with a diagnosis of spinal muscle atrophy (SMA). The documentation indicated she was symptomatic when she was treated with Spinraza. She was originally treated with this medication due to a high troponin level precluded her from the planned Zolgensma treatment. The documentation indicated the physicians wanted to treat the patient given that the natural history may cause the patient to lose the ability to breathe, swallow, move independently as well as sit unassisted, crawl or walk. The plan was to always treat the patient with the requested medication; however, given the circumstance above it was not initiated. The documentation does indicate the current liver function testing as well as platelet count and troponin are all within normal limits. She also does not currently have an acute viral infection.

The patient does meet the criteria:

 Diagnosis of SMA confirmed by either bi-allelic deletion or dysfunctional point mutation of the survival of motor neuron 1 (SMN1) gene; AND 

Must have SMA phenotype 1 confirmed by: 

1 or 2 copies of the  survival of motor neuron 2 (SMN2) gene; OR
3 copies of the SMN2 gene WITHOUT the c.859G>C single base substitution modification in exon 7; AND 
NOT have advanced SMA (e.g., permanent ventilation support; complete limb paralysis); AND 
NOT have pre-existing hepatic insufficiency; AND 
Baseline anti- adeno associated virus type 9 (AAV9) antibody titer of <=1:50 (as measured by ELISA [enzyme linked immunosorbent assay]); AND 
Must be used with systemic corticosteroids (e.g., 1 milligram (mg)/kilogram (kg)/day oral prednisone or equivalent) as directed; AND 
NOT to be used in combination with nusinersen; AND 
Therapy to be administered prior to recipient's 6 month birthday without symptoms

A new gene therapy, Zolgensma (Novartis/AveXis), was developed to treat patients with SMA. Zolgensma, formerly known as AVXS [AveXis]-101, uses the adeno- associated virus serotype 9 vector to deliver a copy of SMN to supplement the defective SMN1. Zolgensma was studied as a one-time, intravenous administration. The Food and Drug Administration (FDA) granted Zolgensma a Breakthrough Therapy Designation and Fast Track Designation.

Results were reported in study of Zolgensma showed clinical meaningful improvement as noted by a 4 point change in response.

The currently available data for Zolgensma (SMA Type I) show prolonged survival and improved motor function compared with historical controls or sham injections. Considering that SMA is a rare disease, the existing evidence base contains many of the common limitations pervasive in rare disease areas, including a small patient population, clinical trial design challenges, and lack of long- term safety and efficacy data. Overall, where data were available Zolgensma demonstrated improvements in motor function, survival, and reduced need for permanent ventilatory support. 

This treatment is deemed medically necessary and previous determination should be overturned.

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

The insurer's denial of coverage for Zolgensma is reversed. Medical necessity is substantiated.