Evrysdi reimb expansion will improve SMA treatment landscape

The newly reimbursed tablet form offers longer prescription periods…bidirectional switching with injectables now allowed

Strengthened long-term management through oral therapy…real-world data confirm effectiveness and durability

The expansion of reimbursement criteria for the spinal muscular atrophy (SMA) treatment Evrysdi is expected to significantly improve treatment convenience for SMA patients in Korea.

Experts note that the introduction of the tablet formulation, the extension of the prescription period, and the allowance for switching between therapies have reduced the burden of long-term therapy and increased the sustainability of treatment in daily life.

On the 23rd, Roche Korea held a press conference at its headquarters in Gangnam-gu, Seoul, to commemorate the launch of the tablet formulation of its SMA treatment Evrysdi (risdiplam) and the expansion of the drug’s reimbursement criteria.

Evrysdi is the only oral treatment among SMA therapies and is regarded as a drug that has brought about a shift in the treatment landscape, which was previously dominated by injectable formulations. This contrasts with Biogen’s Spinraza (nusinersen) and Novartis’s Zolgensma (onasemnogene abeparvovec), both of which are injectable therapies.

Since its first approval in Korea in 2020 as a dry syrup formulation and subsequent reimbursement in 2023, a tablet formulation was later approved. Starting this March, the tablet formulation became eligible for reimbursement, and the existing reimbursement criteria were expanded.

The key elements of the revised reimbursement criteria include: ▲ allowing one-time bidirectional substitution with injectable therapies ▲ extending the maximum prescription period to approximately 2 months ▲ and refining assessment tools to better reflect the patient’s condition.

Previously, prescriptions for the dry syrup were limited to 3 bottles (a 36-day supply), effectively allowing only monthly prescriptions; however, following the revision, the reimbursement criteria have been expanded to a maximum of 5 bottles (a 64-day supply). Prescriptions for tablet formulations can now also be issued for up to 2 packs (56 days’ supply), helping to reduce the treatment burden on patients and their caregivers.

Jong-Hee Chae, Professor of Pediatrics at Seoul National University Hospital

In addition, whereas previously only a one-time switch from Spinraza to Evrysdi was allowed, the revision now permits bidirectional switching, greatly expanding flexibility in treatment strategy.

Jong-Hee Chae, Professor of Pediatrics at Seoul National University Hospital (Chair, Korean Child Neurology Society), said, “In the past, once a treatment was selected, it was difficult to change, but now we can apply more flexible strategies based on the patient’s age, environment, and treatment response.”

She added, “With the assessment tools now more finely subdivided, it has also become possible to evaluate treatment effects more precisely in a way that reflects each patient’s actual condition.”

SMA is a rare genetic disorder characterized by the gradual loss of motor neurons due to a deficiency of the survival motor neuron (SMN) protein, affecting respiration, swallowing, and overall motor function. It is classified into Types 1 through 4 based on the age of onset and functional level, with Type 1 being a particularly severe form with high mortality if untreated.

Professor Chae said, “Because SMA is a disease that requires long-term treatment, it is crucial to ensure the sustainability of treatment in daily life. The introduction of tablets that can be stored at room temperature and the extension of the prescription period represent meaningful changes in improving the quality of life for patients and their families.”

Long-term effects confirmed through real-world data

Evrysdi has also shown sustained effectiveness in real-world data (RWD).

This drug is a small molecule that modulates SMN2 gene splicing, and it works by crossing the blood-brain barrier and increasing production of the SMN protein throughout the body, including the central nervous system.

Hyungjun Park, Professor of Neurology at Gangnam Severance Hospital

Consistent efficacy has been confirmed across 4 global clinical trials, ranging from infants before symptom onset to patients with prior treatment experience. Notably, in Types 1 and 2/3 patients, long-term data spanning over 5 years demonstrated the maintenance of motor function.

Similar results were also confirmed in real-world data (RWD) recently published in Europe. In a study of patients in the Czech Republic and Slovakia, early improvements in motor function were observed even in severely affected patients, along with the maintenance of respiratory and motor function for up to 3 years.

In another Croatian study, patients who switched from an existing injectable therapy to Evrysdi showed motor function improvement over 12 months comparable to prior treatment, confirming non-inferiority.

Hyungjun Park, Professor of Neurology at Gangnam Severance Hospital, said, “Recent real-world data from Europe show that Evrysdi exhibits a pattern of significant functional improvement within the first 6 months of treatment, followed by stable maintenance over the long term. In particular, improvements and maintenance of motor function were confirmed in patients with SMA Types 2 and 3, and results showing stable maintenance without functional decline were also confirmed in Type 1 patients.”

He further emphasized, “Given the natural course of SMA, which is characterized by continued functional deterioration, this represents a meaningful change. Because consistent efficacy and long-term durability have been confirmed across a wide range of patient groups, the drug has strong practical value in real-world clinical practice.”