Rare Disease Report

Insurance Company Agrees to Pay for Expensive SMA Drug

MAY 04, 2017
James Radke
Insurance company Neighborhood Health Plan has confirmed that they will provide Spinraza (nusinersen) for all patients with spinal muscular atrophy (SMA). 
Last week, the family of a 13-year-old SMA patient who had been refused the drug successfully appealed that decision before the Office of Medicaid Board of Hearings.
The appeal was fairly self explanatory. Spinraza is indicated for “the treatment of spinal muscular atrophy (SMA) in pediatric and adult patients.”  That wording in the prescribing information for Spinraza  is very straightforward – if a patient has SMA, they can receive the drug.
That wording, however, does not quite match the data that led to the drug’s approval. That data was based on patients with early-onset SMA taking the drug, not patients with late-onset SMA which is a less severe form of the disease.
And given the cost of the drug — $750,000 for the first year of treatment and $375,000 after that — it is not surprising that some health insurance companies were reluctant to provide it for some patients without the data to back up its efficacy in those patietns.
However, new data recently presented at the AAN 2017 Annual Meeting showed the drug is also effective in late-onset SMA and based on that data (and the decision of the Office of Medicaid Board of Hearings), Neighborhood Health Plan has stated they will pay for the drug moving forward.

The CHERISH study is a phase 3, global, multicenter, randomized, double-blind, sham-procedure controlled study in which patients with late-onset SMA (aged 2-12) were given Spinraza (nusinersen) [ 4 intrathecal injections (totaling 12mg non-scaled)] and a sham procedure. Patients were monitored over 15 months, with a primary endpoint of change in Hammersmith Functional Motor Scale–Expanded (HFMSE) score at month 15.
Eugenio Mercuri, MD, of Catholic University and Centro Clinico Nemo in Rome presented findings from the CHERISH trial during the Emerging Science session at the AAN meeting.
The study has shown that the efficacy trends of Spinraza in the later-onset SMA children are similar to that observed in early-onset SMA children—namely that the drug is effective. In the data, available, Dr. Mercuri noted that in more than 57% of the treated patients, HFMSE scores improved by 3 or more points at month 15, compared to about 20% of the controls. The trial reported a “favorable safety profile” with no withdrawals due to adverse events.
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 early-onset SMA produce very little SMN protein and have a life expectancy of less than 2 years. Children with late-onset have greater amounts of SMN protein but may still have a shortened lifespan and accumulate life-long physical disabilities.
Spinraza (nusinersen) is an antisense oligonucleotide that is designed to alter the splicing of pre-mRNA from the SMN2 gene in order to increase production of fully functional SMN protein.  Its approval last December was based on data from patients with the more severe form of SMA but the FDA did approve the drug for all SMA patients

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