New myelodysplastic syndrome drug 'Reblozyl' lands Big 5 DC

Has passed drug committees (DC) of Seoul National University Hospital, St. Mary's Hospital, and Asan Medical Center

Still non-reimbursable after failing the DREC review in August last year

Product photo of Reblozyl.

The treatment for myelodysplastic syndrome (MDS), 'Reblozyl,' is available for prescription at tertiary general hospitals.

 

Sources said that Bristol Myers Squibb (BMS) Pharmaceutical Korea's Reblozyl (luspatercept) has passed all drug committees (DC) of the 'Big 5' medical centers, including Samsung Medical Center, Seoul National University Hospital, Seoul St.

 

Mary's Hospital, Seoul Asan Medical Center, and Sinchon Severance Hospital.

 

However, Reblozyl is still non-reimbursable.

 

The drug was not approved for reimbursement appropriateness at the Drug Reimbursement Evaluation Committee (DREC) of the Health Insurance Review and Assessment Service (HIRA), held August last year.

 

It has not been updated since then.

 

Reblozyl can be prescribed to treating ▲patients with very-low risk, low-to intermediate-risk MDS or myelodysplastic/myeloproliferative neoplasms with ring sideroblasts and thrombocytosis ▲anemia in adult patients with myeloproliferative neoplasms ▲patients with Beta Thalassemia who may need RBC transfusions.

 

Reblozyl is for adults who have not responded well to an Erythropoiesis-Stimulation Agent (ESA) or may need red blood cell (RBC) transfusions.

 

Reblozyl can be administered once every 3 weeks in patients with MDS or Beta Thalassemia at an initial dosage of 1.0 mg/kg.

 

Reblozyl's mechanism works by binding to TGF-β superfamily ligands, thereby diminishing the overactivation of the Smad 2/3 pathway.

 

The drug promotes erythroid maturation.

 

The efficacy of Reblozyl was demonstrated through the Phase 3 MEDALIST study.

 

During the 24-week follow-up period, the study results showed that 13% of the placebo group reached consecutive transfusion-free periods, whereas 38% reported in the Reblozyl group.

 

During the same period, the percentages of patients who reached a transfusion-free period over 12 weeks were 8% for the placebo group and 28% for the Reblozyl group.

 

Those for over 16 weeks were 4% for the placebo group and 19% for the Reblozyl group.

 

Extending the follow-up period to 48 weeks, the percentages of patients who reached a transfusion-free period over 16 weeks were 7% for the patient group, whereas 28% for the Reblozyl group.

 

Meanwhile, MDS is a type of malignant disease affecting blood stem cells in the bone marrow.

 

It is a disease of the elderly, with higher occurrence in elderly over 60 years of age.

 

MDS is characterized by immature blood cells and low counts of healthy white blood cells, red blood cells, or platelets in the peripheral blood.

 

The most common adverse reactions are fatigue from anemia, systemic weakness, and loss of motor ability.

 

Worsened anemia could cause palpitation, trouble breathing, chest pain, and the possibility of advancing to acute myeloid leukemia (AML).

 

The clinical outcomes and the progress were found to be categorically diverse.

 

There were cases of a stable life with slight anemia, but a case of death within a few months from complications associated with low counts of red blood cells or acute leukemia was reported.