Good news for Gaucher's patients!

▎The editor of WuXi AppTec's content team recently, Takeda issued a press release stating that its Shire company's injection-use velaglucerase alfa (velaglucerase alfa, trade name: VEGRA ®, English trade name: Vpriv) was launched The application has been approved by the National Medical Products Administration (NMPA) of China and is suitable for long-term enzyme replacement therapy (ERT) for patients with Gaucher disease type 1.

Screenshot source: NMPA official website about Gaucher disease Gaucher disease is an autosomal recessive inherited disease, a lysosomal storage disease.

In 2018, the disease was included in China's "First Batch of Rare Disease Catalog.
"
To learn more about rare disease related knowledge and research progress, please click to visit the rare disease think tank applet.
The prevalence of Gaucher disease varies from region to region in the world.

A systematic analysis statistics show that the number of cases per 100,000 people in the world is 0.
7-1.
75, which is one of the most common lysosomal storage diseases in the world.

The incidence of Gaucher disease in China is 1:80844.

A Chinese demographic study found that Gaucher disease is the fourth-ranked lysosomal storage disease in the population of eastern China.

Due to gene mutations, the body's glucocerebrosidase activity is lacking or decreased, causing its substrate glucocerebrosid to accumulate in the lysosomes of macrophages in the liver, spleen, bones, lungs, and even brain, forming a typical storage Accumulative cells are "Gaucher cells", which in turn causes the body tissues and organs to appear disease and gradually worsen.

According to whether the nervous system is involved, Gaucher's disease is mainly divided into non-neuropathy (type Ⅰ) and neuropathy (type Ⅱ and Ⅲ).

Other subtypes, such as perinatal fatal type and cardiovascular type, are relatively rare.

Relevant data show that type I Gaucher disease can occur at all ages (mainly childhood onset), and its symptoms vary greatly.
Generally speaking, the earlier the onset, the more severe the symptoms.

In terms of organs, the symptoms are mainly hepatosplenomegaly, especially splenomegaly, which is often accompanied by hypersplenism, and even splenic infarction, splenic rupture and other complications are life-threatening; in blood, thrombocytopenia and anemia In some patients, leukopenia may be accompanied by abnormal blood coagulation.

In addition, patients may also experience paleness, fatigue, skin and gum bleeding, increased menstrual flow, and even life-threatening bleeding.

Image source: 123RF Regarding Veracilase Alpha for Injection At present, there are two main treatment methods for Gaucher's disease: enzyme replacement therapy (ERT) and substrate removal therapy (SRT).

According to Takeda Shimbun, Enzyme Replacement Therapy (ERT) has been recommended by authoritative guidelines/consensus at home and abroad as the standard treatment for patients with type I Gaucher disease.
By specifically supplementing the lack of enzymes in the patient’s body, it reduces the presence of glucocerebrosides in the body.
The accumulation of serotonin can improve the clinical symptoms and signs of patients and maintain normal growth and development.

The approved Verazilase Alpha for injection belongs to enzyme replacement therapy.

Velaglucerase α for injection is a glucocerebrosidase produced by gene activation technology in a continuous human cell line.
It has the same amino acid sequence as natural human glucocerebrosidase, which can improve children and adult patients in the early treatment.
Symptoms such as anemia and thrombocytopenia, reduce the size of the liver and spleen due to disease, improve related bone diseases, and continue to improve or stabilize the patient’s symptoms in the later stage.

The results of a phase 3 study named TKT032 showed that after 12 months of treatment with velaglucerase alpha for newly-treated patients with type I Gaucher disease, key clinical parameters were significantly improved, including an increase in hemoglobin concentration (+23.
3%) ), increased platelet count (+65.
9%), decreased liver volume (-17.
0%) and decreased spleen volume (-50.
4%), which continued during the subsequent study period.

Another 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, a phase 3 study named TKT034 also showed that patients can safely switch from other enzyme replacement therapies to equal-dose velaglucerase alpha treatment, and that key clinical parameters of velaglucerase alpha are maintained within 12 months of treatment stable.

Image source: 123RF At present, velaglucerase alpha for injection has been approved in more than 50 countries and regions including the United States, the European Union, and Japan.

This time, the approval of the drug in China will bring new hope to the lives of Chinese patients with Gaucher disease and help more patients enjoy a healthy life and a better future.

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start up! Reference materials [1] Government Service Portal of China National Medical Products Administration.
Retrieved May 10, 2021, from [2] Takeda Verlaglucerase for Injection α is approved to enter China.
Retrieved May 10, 2021, from https://mp.
weixin.
qq.
com/s/SpltjnP_8Ont-YWm56N31Q[3] Expert consensus on diagnosis and treatment of Gaucher disease in China (2015)[4] Guidelines for the diagnosis and treatment of rare diseases (2019 edition) Note: This article aims to introduce the progress of medical and health research, not a treatment plan recommendation.

If you need guidance on treatment plans, please go to a regular hospital for treatment.