Appeal type: Medical necessity
Decision: Overturned

Diagnosis: Spinal Muscular Atrophy (SMA)

Treatment: Spinraza injections
 
The insurer denied the Spinraza injections.

The denial was overturned.

This is a female patient who presented for a new patient visit with complaints of weakness.  The patient has a medical history of vitamin B12 deficiency.  The patient was born at 44 weeks after induction with a history of oligohydramnios. The patient sat at 6 months and walked at 1 year. The patient's mother stated that she noticed the patient's weakness after the patient started walking.  At a very early age the patient became very sick with high fevers and seemed to have developed weakness after this; she could not walk up the stairs, could not jump, nor get out of a chair.  At the time of the consultation, the patient was able to run, but did not run as fast as other kids; she had normal tone and muscle bulk.  The physical therapist noted there was proximal weakness with needing to push off the floor/legs for getting up off the floor and ascending the stairs.  There was neck flexor weakness with antigravity movement. She was able to maintain a half-kneeled position but required assistance to get to the position.  The patient was able to lift her head in the prone position and press up her upper body in this position.  The patient was unable to skip or hop on 1 foot. There was no family history of muscle weakness or hereditary neurodegenerative disease. The etiology was likely inherited/congenital myopathy.  The patient underwent a muscle biopsy that revealed 1 homozygous pathogenic variant identified as SM and 1.  SMN1 was associated with autosomal recessive spinal muscular atrophy (SMA). The patient was found to have spinal muscular atrophy, type III (SMA to copy numbers of 4 and clinical symptoms onset after 12 months).  The treatment plan included initiation of Spinraza.

Spinraza is a survival motor neuron-2 (SMN2)-directed antisense oligonucleotide with U.S. Food and Drug Administration (FDA)-approved indications for the treatment of spinal muscular atrophy (SMA) in pediatric and adult patients.  The literature indicates that treatment for spinal muscular atrophy, prior to approval of Spinraza, was mostly supportive.  It is the only medical treatment available for patients with this rare disease.  Studies with this medication have shown to improve and maintain motor functions and increase the patient's life span.

SPINRAZA is the first and only treatment approved by the FDA indicated for spinal muscular atrophy (SMA) in pediatric and adult patients.  SPINRAZA has been studied across multiple clinical trials in patients with varying types of SMA, including presymptomatic and symptomatic infantile-onset and later-onset SMA. The patients in these studies had or were likely to develop SMA Type 1, 2, or 3. The overall findings of the controlled trial support the effectiveness of SPINRAZA across the range of patients with SMA and appear to support the early initiation of treatment with SPINRAZA. These findings are supported by open-label, uncontrolled clinical trials in infantile-onset SMA. In addition to ENDEAR and CHERISH, the efficacy of SPINRAZA was also evaluated in open-label clinical trials conducted in patients with presymptomatic and symptomatic infantile SMA, as well as later-onset SMA.  The patients in these studies had or were likely to develop SMA Type 1, 2, or 3.

The results of the controlled trials in infantile-onset (ENDEAR) and later-onset (CHERISH) patients with SMA were supported by open-label, uncontrolled trials conducted in symptomatic patients with SMA (infantile onset and later onset).  

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

The carrier's denial of coverage for Spinraza injections is overturned.  The medical necessity is substantiated.