Transfer from | Two applications for injection at Celgene, a subsidiary of BMS, have been included in the proposed priority review list for the treatment of adult β-Thalassemia patients who need regular infusions of red blood cells (RBCs), according to the latest announcement from the Drug Review Center (CDE) of the State Drug Administration of China.
public information, Reblozyl is a "first-in-class" red blood cell maturant that represents an innovative drug type that reduces the burden of blood transfusion by regulating the later hemoerythocyte maturation process.
Screenshot Source: According to public information, Reblozyl is a soluble fusion protein that is fused between the Fc domain of human immunoglobulin G1 (IgG1) and the extracellular domain of the active protein IIB (ActRIIB).
it can act as a ligand trap for TGF-β, preventing TGF-β from activating the Smad2/3 signal path, thereby promoting differentiation and maturation of late red blood cells.
has been granted orphan drug eligibility, fast-track eligibility, and priority review eligibility by the FDA.
November 2019, the FDA approved Reblozyl for the treatment of blood transfusion dependence β anemia symptoms in people with thalassemia.
In China, the proposed inclusion of adaptation applications for "adult β-thalassemia patients who need regular infusions of red blood cells" means that this innovative treatment is expected to accelerate the benefits to Chinese patients.
Based on research published when Reblozyl was approved in the United States, a Phase 3 clinical trial of 336 patients with blood transfusion dependence β thalassemia showed that 21 percent of patients treated with Reblozyl had a blood transfusion burden that was more than 33 percent lower than the baseline, significantly better than the control group that received a placebo treatment (4.5 percent, p.lt;0.0001).
noted that in April 2020, Reblozyl was also approved by the FDA to treat anemia in patients with very low- to moderate-risk bone marrow prolific abnormal syndrome.
these patients failed to receive red blood cell production stimulants (ESA) treatment and needed regular red blood cell infusions to relieve anemia symptoms.
, Reblozyl is the first new treatment option for such patients in more than a decade, according to a press release.
Public information shows that anaemia is a common symptom affecting nearly 25 per cent of the world's population, and that most patients have temporary symptoms of anemia, but some people with rare diseases (including β thalmphatic anemia, bone marrow growth syndrome, bone marrow fibrosis) develop chronic anemia over a long period of time.
with chronic anemia often rely on regular blood-red blood cells to relieve their symptoms.
often blood transfusions not only put a living and economic burden on patients, but can also lead to iron overdose, infection, immune response and other side effects.
References: China's State Drug Administration Drug Review Center (CDE). Retrieved Jan 22,2021, from FDA approves first therapy to treat patients with rare blood disorder. Retrieved November 8, 2019, from U.S. Food and Drug Administration (FDA) Approves Reblozyl® (luspatercept-aamt), the First and Only Erythroid Maturation Agent, to Treat Anemia in Adults with Lower-Risk Myelodysplastic Syndromes (MDS). Retrieved 2020-04-04, from