- Another major U.S. insurer has limited coverage for Biogen's newly approved Spinraza (nusinersen), adding to the likelihood that uptake for the pricey spinal muscular atrophy (SMA) drug will be gradual at first.
- Kentucky-based Humana announced this week it would judge the treatment to be medically necessary only in patients with the most severe form of the neuromuscular disease.
- Spinraza is the first drug approve to treat SMA, securing a broad label from the Food and Drug Administration for all adult and pediatric patients late last year. But a $750,000 price tag for the first year of treatment has led some payers to balk at waving the drug through without some checks on coverage.
Biogen and its partner Ionis Pharma won an early Christmas present from the FDA with Spinraza's ahead-of-schedule approval. Forecasts peg the drug to eventually reach blockbuster status over the longer term. But initial payer reaction probably signals a more moderate ramp-up in sales initially.
Anthem, another large U.S. insurer, has also restricted coverage to only those with the most severe form of the disease, known as type 1 SMA. And both Anthem and Humana will require proof patients are responding to therapy in order to cover continuation of treatment after the first 6 months.
United Health, on the other hand, will cover the drug more broadly — including all patients with types 1, 2 and 3 — but also placed some limitations around initial therapy and continuation of treatment.
Biogen and Ionis hope that new data from the Phase 3 CHERISH and ENDEAR studies will help improve the drug's uptake once incorporated into the label. Topline results from CHERISH, in particular, showed Spinraza's efficacy in later-onset SMA. Full results will be presented at a future medical meeting.
Analysts at Jefferies predict that insurer coverage is more likely to mirror the policies set by Humana and Anthem rather than that of United Health, but see room for expansion as more data comes out.
"We see good long-term global sales potential for Spinraza," the analysts add, with the proviso that this will depend on how coverage and physician engagement evolves.
Apart from Spinraza, the only other options for SMA patients are for disease management and supportive care. Chicago-based AveXis is developing a gene therapy known as AVXS-101, but it is still only in early clinical development with a pivotal trial planned for sometime in 2017.
Spinal muscular atrophy affects around one in 6,000-10,000 live births. Patients with SMA suffer from progressive muscular atrophy and weakness resulting from the loss of motor neurons in the spinal cord and lower brain stem. The most severe form of SMA — type 1 — represents about 60% of SMA diagnoses, according to Anthem, and can result in paralysis and difficulty breathing or swallowing.