On April 30, Takeda China announced that its subsidiary Vedex® (velaglucerase alpha for injection) has been approved by the China National Medical Products Administration for long-term enzyme replacement therapy (ERT) for patients with type 1 Gaucher disease.
Vedex® (velaglucerase alpha for injection) is glucocerebrosidase produced by gene activation technology in a continuous human cell line. It has the same amino acid sequence as natural human glucocerebrosidase. It can improve the symptoms of anemia and thrombocytopenia in children and adult patients, reduce the size of the enlarged liver and spleen, improve related bone diseases, and continue to improve or stabilize the symptoms of patients in the later stage.
As an autosomal recessive genetic metabolic disease, Gaucher disease is a lysosomal storage disease. Due to gene mutations, the body's glucocerebrosidase activity is lacking or reduced, causing its substrate glucocerebrosid to be in the liver, spleen, and spleen. Macrophages in bones, lungs and even brains accumulate in lysosomes, forming typical storage cells called "Gaucher cells", which leads to the appearance of pathological changes in body tissues and organs and gradually worsens. The main manifestation of the patient’s organ disease is hepatosplenomegaly: the liver can be enlarged to 2-3 times the normal volume; the spleen is enlarged to 15.2 times the normal volume on average, up to 75 times the normal volume, and even splenic infarction and spleen rupture may occur. Wait for the situation. In addition, the patient is also accompanied by growth retardation, skeletal abnormalities, anemia, thrombocytopenia, and even life-threatening bleeding. In 2018, Gaucher's disease was included in my country's "First Batch of Rare Disease Catalog."
Enzyme replacement therapy (ERT) is recommended by authoritative guidelines/consensus at home and abroad as the standard method for the treatment of patients with type 1 Gaucher disease. By specifically supplementing the lack of enzymes in the patient's body, it reduces the accumulation of glucocerebrosid in the body and improves the patient The clinical symptoms and signs, to maintain normal growth and development5.
Vedex® (velaglucerase alpha for injection) has passed multiple ERT clinical development projects and new drug clinical trial project evaluations. A total of 305 patients have received partial treatment for up to 7 years. The results of the TKT032 Phase III study showed that after 12 months of velaglucerase alpha treatment in newly-treated patients, key clinical parameters showed significant improvement compared with baseline values: increased hemoglobin concentration (+ 23.3%) and increased platelet count (+ 65.9%), liver volume reduction (-17.0%) and spleen volume reduction (-50.4%), which persisted during the subsequent study period; the HGT-GCB-044 Phase III expansion study confirmed the Vedex® The efficacy and safety of (velaglucerase α for injection) in pediatric patients are the same as those in adult patients. A post-treatment analysis showed that after 4 years of treatment with velaglucerase alpha, the hematological parameters, liver and spleen volume, and bone density of most patients reached normal levels. In addition, the Phase III study of TKT034 showed that patients can safely switch from other enzyme replacement therapies to equal-dose velaglucerase alpha treatment, and the key clinical parameters of velaglucerase alpha remain stable for 12 months.
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