Rare Disease Report

Insurance Company Loses Battle with SMA Patient, Must Pay for FDA Approved Drug

APRIL 26, 2017
James Radke
Spinraza (nusinersen) 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, has not stopped some insurance companies from trying to limit which SMA patients receive the expensive drug – and at $125,000 per dose, the drug is expensive.
Recently, Neighborhood Health Plan refused to pay for the drug to treat 13-year-old Tyler Hansen who has a less severe form of the condition – SMA type 3. The insurance company had argued that the FDA approval was based on patients with SMA type 1 taking the drug, not patients with SMA type 3.  On April 13, the Hansen family had a hearing before the Office of Medicaid Board of Hearings to appeal the insurance company’s decision.

Tyler Getting His Medicine

According to a report by the Boston Business Journal, the appeal has been successful. The Hansen family received a letter informing them that the appeal was successful, and that National Health Plan will likely have to cover the cost of the drug.

Does the Drug Work in SMA Type 3 Patients?

At this year’s AAN 2017 Annual Meeting, new data is emerging showing the drug to be effective in type 3, or later-onset SMA.
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.

 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 I SMA produce very little SMN protein and have a life expectancy of less than 2 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.
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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