Rottecept for injection is approved for marketing, bringing a new treatment option for thalassemia patients

Recently, an erythrocyte maturation agent, rotercept for injection (trade name: Liblose®), was approved by the China National Medical Products Administration (NMPA) for the treatment of patients who require regular red blood cell transfusions.
Note ≤15 units/24 weeks in adult patients with beta-thalassemia
.

Public information shows that the approval of rotacept for injection will bring a new treatment option for adult patients with β-thalassemia, which is expected to reduce the burden of blood transfusion in patients with transfusion-dependent β-thalassemia and reduce the iron overload caused by blood transfusion.
(This is a disorder of iron metabolism, which can affect bone marrow hematopoietic function, etc.
) risk, while relieving the status quo of blood source tension
.

Screenshot source: NMPA official website

About beta-thalassemia

When it comes to anemia, many people may think that it is a "minor disease" and does not need to be concerned
.

The "Clinical Practice Guidelines for β-thalassemia" pointed out that so far, more than 300 hemoglobin gene mutations related to β-thalassemia have been found in the world, but more than 90% of patients are caused by more than 40 mutations
.

Based on clinical phenotype and dependence on blood transfusions, individuals with beta-thalassemia can be classified as:

Carriers, usually asymptomatic and without body weight, may show mild microcytic hypochromic anemia (ie, red blood cells become smaller with an enlarged central pale area) on hematology
.

Patients are divided into β-thalassemia intermediate and β-thalassemia major
.

Southern China is one of the regions with the highest incidence of the disease in the world.
The three provinces with the highest β-thalassemia mutation carrier rate are Guangxi (6.
66%), Hainan (5.
11%) and Guizhou (4.
13%), Guangdong, Yunnan, Hunan, Jiangxi and other places are also relatively high
.

For the treatment of beta-thalassemia, patients rely on lifelong blood transfusions, iron removal therapy, or hematopoietic stem cell transplantation to stay alive
.

Without effective treatment, patients can cause a series of complications, including hepatosplenomegaly, special facial features (maxillary protrusion, zygomatic bulge, widened eye distance, nasal bridge collapse), osteoporosis, joint disease, developmental delay, Short stature, and anemia, heart disease,
etc.

Without blood transfusions, most patients will die of infection-induced heart failure by age 5-10
.

About Rottership

Rotecept is a soluble fusion protein composed of the Fc domain of human immunoglobulin G1 (IgG1) fused to the extracellular domain of activin receptor IIB (ActRIIB)
.

Public information shows that the new mechanism of rotacept will be expected to reduce the patient's sensitivity to blood transfusion and iron removal.
Differentiation and maturation of red blood cells, increasing hemoglobin levels
.

The approval of Rottecept in China this time is based on a randomized, double-blind, placebo-controlled, multi-center global Phase 3 clinical trial called BELIEVE, which has 65 clinical trials in 15 countries.

The patients were randomized 2:1 to receive rotacept + best supportive care (including blood transfusion, iron removal, anti-infective therapy and nutritional support) or placebo + best supportive care

research shows:

From week 13 to week 24 of treatment, 21.

During any 24-week period, the proportion of patients with a 33% reduction in transfusion burden in the rotercept group was 15 times that in the placebo group (41.

At 48 weeks of treatment, serum ferritin levels in the rotercept group decreased by an average of 248 μg/L from baseline, while the placebo group increased by an average of 107 μg/L;

Rotecept was well tolerated, and the vast majority of adverse events were grade 3 or lower

Rottecept is currently approved in the United States, Canada and the European Union for the treatment of adult patients with beta-thalassemia who require regular red blood cell transfusions, and in the United States and the European Union for the treatment of anemia in patients with certain blood disorders

We expect that after the approval of rotacept for injection in China, it will come to patients as soon as possible, so that they can have new treatment options, reduce the incidence of anemia, iron overload and blood transfusion complications, and improve the quality of life of patients