Romiplate receives reimbursement in Korea

Reimbursement covers combined use with ATG and CsA in treatment-naive patients with severe disease

Hematologic response rate reaches 76.5% at Week 27…also in progress of accumulating long-term treatment evidence

The expansion of National Health Insurance reimbursement for Romiplate to first-line treatment is expected to change initial treatment strategies for patients with severe aplastic anemia.

Previously, reimbursement was primarily available for patients who failed to respond to immunosuppressive therapy or were refractory to it. Under the revised criteria, treatment-naive patients can now also receive Romiplate (romiplostim) in combination with standard immunosuppressive therapy from the outset.

On Aug. 12, DKSH Korea’s Healthcare Business Unit held a press conference at the Park Hyatt Seoul in Samseong-dong to discuss the clinical significance of Romiplate’s first-line reimbursement for severe aplastic anemia and future treatment strategies.

Professor Jun Ho Jang, Department of Hemato-Oncology, Samsung Medical Center

Per a notice issued by the Ministry of Health and Welfare, Romiplate has been reimbursed since Aug. 1 in combination with antithymocyte globulin (ATG) and cyclosporine (CsA) for patients with severe aplastic anemia who are unable to undergo allogeneic hematopoietic stem cell transplantation or who do not have a suitable donor. Reimbursement is available for up to 6 months of treatment.

Previously, reimbursement mainly applied to adults with severe aplastic anemia who were refractory to immunosuppressive therapy or were unable to receive such treatment. The revised criteria therefore allow patients to receive Romiplate combination therapy from the initial stage of treatment rather than waiting until conventional therapy fails.

Romiplate is a thrombopoietin receptor agonist (TPO-RA) that promotes platelet production. It acts on hematopoietic stem and progenitor cells in the bone marrow to help restore hematopoietic function.

Originally used to treat chronic immune thrombocytopenia, Romiplate has gradually expanded its role into severe aplastic anemia. In July 2024, its indication was expanded to include first-line use in combination with immunosuppressive therapy in treatment-naive patients with severe aplastic anemia.

Accumulating evidence for first-line combination therapy in clinical studies

Aplastic anemia is a disorder in which impaired bone marrow function results in insufficient production of red blood cells, white blood cells, and platelets. In patients with severe disease, the risks of infection and bleeding are high, making rapid hematologic recovery a major treatment goal.

Treatment is selected based on factors such as patient age and the availability of a hematopoietic stem cell donor, with options including hematopoietic stem cell transplantation (HSCT) and immunosuppressive therapy. In patients who are not candidates for transplantation, combination immunosuppressive therapy with ATG and CsA has been a mainstay of treatment.

However, a persistent issue remained, as not all patients achieve an adequate hematologic response with immunosuppressive therapy alone. More recently, strategies incorporating a TPO-RA from the initial treatment stage have been used to improve hematologic response.

The first-line evidence for Romiplate is based on this approach. In the Phase 2/3 531-003 study conducted in Asian patients with aplastic anemia and reported last year, the combination of Romiplate + ATG + CsA demonstrated efficacy.

At Week 27, the overall hematologic response rate, combining complete and partial responses, was 76.5%. The company said this represented an improvement compared with the approximately 50% response rate previously reported with conventional rabbit ATG (rATG) + CsA immunosuppressive therapy.

Long-term follow-up data are also accumulating. In a long-term follow-up study presented at the 2025 American Society of Hematology (ASH) Annual Meeting, treatment-naive patients with aplastic anemia were followed for up to 5 years after receiving Romiplate combination therapy.

Among 15 enrolled participants from the 531-003 study who received rATG, CsA, and Romiplate, the hematologic response rate at two years was 93.3%. Patients showed sustained response thereafter, while the proportion of patients dependent on transfusions was lower at the final follow-up than at baseline.

In terms of safety, increased bone marrow reticulin was observed in some patients, but only at Grade 1. No new chromosomal abnormalities or progression to acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS) were observed in the study.

Jun Ho Jang, Professor of Hemato-Oncology at Samsung Medical Center, said, “Achieving a rapid and sufficient hematologic response during initial treatment is important in severe aplastic anemia. With Romiplate now available in combination with immunosuppressive therapy from the outset, physicians can consider a more proactive treatment strategy from the early stage rather than waiting until patients fail to respond to conventional therapy.”

The allowance of proactive reimbursement in Korea…”expected to drive a treatment paradigm shift”

The key significance of the reimbursement expansion is that Romiplate can now be used at an earlier stage, shifting from treatment after failure of existing therapies to initial treatment.

In aplastic anemia, an insufficient hematologic response after initial therapy can lead to repeated transfusions and continued risks of infection and bleeding. Increasing the likelihood of hematopoietic recovery from the time of diagnosis is therefore considered important in reducing the subsequent treatment burden.

In particular, Romiplate does not replace conventional immunosuppressive therapy, but is added to ATG and CsA, effectively providing an additional option within the existing treatment framework.

Professor Jang said, “This reimbursement expansion has broadened treatment options for severe aplastic anemia in Korea, including transplantation. As Korea has moved proactively to provide reimbursement in this space, we are looking forward to changes in the domestic treatment paradigm.”