BMS Reblozyl (ingredient: luspatercept) anemia treatment hit the Korean market only two and a half years after the company completed development of the drug in the United States
The Ministry of Food and Drug Safety has authorized Reblozyl to treat adult patients with anemia who have failed to respond to an erythropoiesis-stimulating agent (ESA) and required red blood cell transfusions due to myelodysplastic syndromes (MDS) or myeloproliferative neoplasms (MPN). Also, the drug is indicated for adult patients with beta-thalassemia who require regular transfusions of red blood cells.
Patients with MDS must be at very low to intermediate risk with ring sideroblasts (MDS-RS) to receive treatment. People with MDS/MPN should accompany ring sideroblasts and thrombocytosis (MDS/MPN-RS-T).
Reblozyl is the first-in-class erythroid maturation agent that reduces Smad2/3 signaling and enhances red blood cell production by selectively binding to TGF-β ligands.
The treatment gained US FDA approval to treat beta-thalassemia for the first time in November 2019 and expanded indications to treat anemic patients with MDS-RS or MDS/MPN-RS-T in April 2020 .
Reblozyl got the Korean license only two and a half years after the first approval in the United States
In April last year, the MFDS designated Reblozyl as an orphan drug.
The Korean permit was based on the results of two Phase 3 studies – BELIEVE and MEDALIST.
In the BELIEVE trial of 336 adult patients with beta-thalassemia who required red blood cell transfusions, 21.4% of the Reblozyl group (224 patients) achieved reduction in the burden of red blood cell transfusions. In contrast, only 4.5% of the placebo group did.
In MEDALIST out of 229 patients who did not respond to ESA and who had very low, low and moderate risk MDS-RS or MDS/MPN-RS-T, 37.9% of the Reblozyl group (153) remained transfusion-free for eight weeks or more, compared with 13.2 percent of the placebo group.
As Reblozyl dramatically reduced the burden of transfusions in blood cancer patients through a new mechanism, its sales jumped immediately after market launch.
According to a BMS annual report in 2021, Reblozyl sold $551 million worldwide last year, a jump of more than 100% from $274 million the previous year.
Industry watchers say the expansion of indications for Reblozyl has just begun.
The drug expanded the scope of treatment for MDS/MPN and planned to do so for beta-thalassemia patients without red blood cell transfusion dependency, supported by promising data.
Late last year, BMS asked the FDA to expand indications for Reblozyl to treat non-transfusion-dependent beta-thalassemia (NTD) based on results from the Phase 2 BEYOND trial. . The FDA is expected to rule by June.
Reblozyl is also being tested in the global Phase 3 INDEPENDENCE trials in patients at 150 institutions in Europe, North America, Asia and Australia, based on strong data from the Phase 2 patient study with anemia associated with myelofibrosis.
BMS said Reblozyl could become the leader in blood cancer treatment.