Reblozyl can be prescribed in general hospitals in KOR

New drug for myelodysplastic syndrome

Passes drug committees of medical institutions including Samsung Medical Center

Reblozyl, a treatment for myelodysplastic syndrome, can now be prescribed in general hospitals in Korea.

 

According to industry sources, BMS Korea’s Reblozyl (luspatercept-aamt) passed the drug committees (DCs) of several medical institutions in Korea including Samsung Medical Center and Sinchon Severance Hospital.

 

Reblozyl is indicated to treat anemia in patients who showed inadequate response to or are intolerant to treatment with an erythropoiesis-stimulation agent (ESA) who have ▲ very low- to intermediate-risk myelodysplastic syndrome with ring sideroblasts (MDS-RS), or ▲ very low- to intermediate-risk myelodysplasia/myeloproliferative tumor with ring sideroblastic cells and thrombocytosis, or ▲myeloproliferative neoplasm with ring sideroblasts and thrombocytosis, or ▲ anemia in adult patients with beta-thalassemia who require regular red blood cell (RBC) transfusions.

 

The starting dose is 1.0 mg/kg subcutaneously once every 3 weeks for myelodysplastic syndrome and beta-thalassemia.

 

Reblozyl has a mechanism of action that induces red blood cell maturation by inhibiting hyperactivation of the Smad 2/3 pathway caused by TGF-β superfamily ligands.

 

Reblozyl demonstrated efficacy in the Phase III MEDALIST trial.

 

Study results showed that the proportion of patients in the placebo arm who achieved continuous blood transfusion (transfusion independence) for at least 8 consecutive weeks during the 24-week observation period was only 13% while 38% in the Reblozyl arm reached 38%.

 

During the same period, the rates of achieving transfusion independence were 8% and 28% respectively for 12 weeks or longer, and 4% and 19% respectively, for 16 weeks or longer in the placebo and Reblozyl arm, When extending the period to 48 weeks, only 7% of patients in the placebo arm achieved transfusion independence for longer than 16 weeks, whereas in the Reblozyl arm, the rate was 28%.

 

Meanwhile, myelodysplastic syndrome is a malignant disease that occurs in hematopoietic cells in the bone marrow.

 

It is a geriatric disease whose incidence increases rapidly in people over 60 years of age.

 

It is characterized by ineffective and dysplastic hematopoiesis in the bone marrow and a decrease in normal white blood cells, red blood cells, and platelets in the peripheral blood.

 

The most common symptoms include fatigue, general weakness, and decreased exercise capacity due to anemia.

 

In severe anemia, symptoms such as palpitations, shortness of breath, and chest pain may also appear and develop into acute myeloid leukemia.

 

Due to different clinical manifestations and course of the disease in each subcategory, some people live stably for decades with only mild anemia, but others develop complications due to a decrease in red blood cells or develop acute leukemia and die within a few months.