Rare Disease Report

Insurance Companies Limit Coverage for Approved SMA Drug

FEBRUARY 08, 2017
James Radke
Insurance companies Humana and Anthem have announced they will cover Biogen’s recently-approved orphan drug Spinraza (nusinersen), but only for patients with the most severe type of the disorder,  spinal muscular atrophy (SMA) type 1. In December the FDA approved Spinraza for SMA types 1 – 4.
On the plus side, UnitedHealth Group said it will cover Spinraza to treat SMA type 1, 2 or 3 in patients with at least two copies of survival of motor neuron 2 centromeric (SMN2) and certain mutations or deletions including survival of motor neuron 1 telomeric (SMN1).

What is Spinal Muscular Atrophy?

SMA is a genetic condition that leads to a deficiency in the spinal motor neuron (SMN) protein as a result of mutations of the survival motor neuron 1 (SMN1) gene. The severity of SMA correlates with the amount of SMN protein. Generally, the muscles most affected are those near the shoulders, hips, thighs and upper back. Muscles used for breathing and swallowing may also be affected. Infants with Type 1 SMA produce very little SMN protein and have a life expectancy of less than two years. Children with Type 2 have greater amounts of SMN protein but still have a shortened lifespan and are never able to stand independently. Children with Type 3 have a normal lifespan but accumulate life-long physical disabilities as they grow.

    Age of Onset   Highest function acheived
 Type 1   0 - 6 months   Never sit
 Type 2    7 - 18 months   Sit, never stand
 Type 3   > 18 monhts   Stand and walk during adulthood
 Type 4   2nd to 3rd decade   Walk unaided
Spinraza (nusinersen) is an antisense oligonucleotide (ASO) designed to alter the splicing of pre-mRNA from the SMN2 gene in order to increase production of fully functional SMN protein. Spinraza is approved for Type 1, 2, and 3 SMA populations.

Costly Drug

The drug costs $125,000 per injection, which amounts to $750,000 for the first year of use and $375,000 each subsequent year for life.
Humira also stated that it will continue to cover the drug for a patient but only if the infant responds to treatment.

Is the Drug Effective?

The efficacy of Spinraza was demonstrated in a clinical trial in 121 patients with SMA type 1 randomized to receive an injection of Spinraza, into the fluid surrounding the spinal cord, or undergo a mock procedure without drug injection (a skin prick). The trial assessed the percentage of patients with improvement in motor milestones, such as head control, sitting, ability to kick in supine position, rolling, crawling, standing and walking.
The FDA asked the sponsor to conduct an interim analysis as a way to evaluate the study results as early as possible; 82 of 121 patients were eligible for this analysis and they found that 40% of patients treated with Spinraza achieved improvement in motor milestones while none of the control patients did.
The most common adverse events with Spinraza were upper respiratory infection, lower respiratory infection, and constipation. The FDA approved the drug with warnings and precautions for low blood platelet count and renal toxicity. 

In the study, 23% of SMA patients taking Spinraza died in the study compared to 43% in the control group.  
Spinraza is an antisense oligonucleotide that modulates splicing of SMN2 mRNA. The drug is being marketed by Biogen which obtained the drug from Ionis Pharmaceuticals.

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