Biogen seeks to expand reimb for high-dose Spinraza

Clinical studies demonstrate functional improvement and recovery in SMA

Reimbursement adequacy assessment underway...DREC review expected within the year

The spinal muscular atrophy therapy Spinraza is seeking expanded reimbursement with its high-dose regimen.

Dailypharm has found that Biogen Korea filed an application in July to expand National Health Insurance coverage for a high-dose regimen of its spinal muscular atrophy drug Spinraza (nusinersen), which consists of a 50 mg loading dose and 28 mg maintenance doses. The regimen has now entered a full reimbursement review by the Health Insurance Review & Assessment Service (HIRA).

Korea’s SMA treatment landscape has diversified as gene therapies and oral treatments have gained reimbursement alongside Spinraza. However, reimbursement criteria for existing treatments remain restrictive, prompting continued calls to address gaps in care, including concerns over treatment discontinuation and improved patient access.

More recently, SMA treatment goals have moved beyond simply prolonging survival or preserving function. Treatment is increasingly aimed at achieving meaningful improvements in quality of life and helping patients reach normal developmental milestones, creating demand for a more proactive approach that seeks to maximize recovery of neuromuscular function rather than merely slow disease progression. The high-dose Spinraza is designed to address these unmet clinical needs.

High-dose Spinraza has demonstrated its clinical value in the global Phase II/III DEVOTE trial and the ONWARD long-term extension study.

In DEVOTE, high-dose Spinraza produced a mean 15.1-point improvement from baseline on the Children’s Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP-INTEND) in patients with Type 1 SMA whose symptoms emerged before six months of age, compared with an 11.1-point decline in untreated controls, demonstrating a significant improvement in motor function. It also reduced plasma neurofilament light chain (NfL), a marker of neuronal injury, by as much as 93%, indicating a reduction in neuronal damage.

Positive findings were also reported in ONWARD, a multicenter long-term extension study evaluating the effects of switching patients from the conventional 12 mg dose to the high-dose regimen. After 302 days of follow-up, Hammersmith Functional Motor Scale–Expanded (HFMSE) scores improved in both ambulatory patients (mean 2.5 points from baseline) and non-ambulatory patients (mean 1.1 points). Revised Upper Limb Module (RULM) scores also showed clear overall improvement.

Clinical experts view high-dose Spinraza not simply as a higher-dose formulation, but as a treatment option that could more strongly suppress neuronal damage and help optimize treatment for patients with SMA.

As a result, some expect discussions on setting reimbursement criteria to be completed as early as this month, with the regimen potentially reaching the Drug Reimbursement Evaluation Committee within the year. Therefore, attention is now rising on whether the clinical benefits demonstrated with high-dose Spinraza will translate into timely reimbursement and broader patient access.