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.
News from the official website: Recently, the U.
S.
Food and Drug Administration (FDA) approved the injectable preparation Voxzogo for use in children with "open" achondroplasia (growth plates) 5 years of age and older
.
Achondroplasia is the most common cause of dwarfism, and the approval of the drug means that these children are still likely to grow taller
.
Chondroplasia-a genetic disease that makes people grow taller.
Chondroplasia is a genetic disease that causes severe short stature and unbalanced growth
.
It is caused by mutations in the FGFR3 gene .
FGFR3 is a negative regulator of bone growth.
Mutations in this gene can inhibit the proliferation and differentiation of chondrocytes in the growth plate of patients, leading to long bone growth disorders.
As a result, children's growth slows down.
The average height of adult patients is about 1.
2 meters
.
Voxzogo's mechanism of action Vosoritide is a C-type natriuretic peptide (CNP) analog derived from natural human peptide.
Natural human peptide is a positive regulator of bone growth and can stimulate endochondral ossification
.
By inhibiting the overactive FGFR3 pathway, CNP can stimulate the proliferation and differentiation of chondrocytes in the growth plate, promote the growth of long bones caused by cartilage internalization, and help patients restore normal growth
.
The safety and effectiveness of Voxzogo were evaluated in a one-year, double-blind, placebo-controlled Phase 3 study of 121 participants who were five and older with achondroplasia and open epiphyses
.
At 52 weeks, the annualized growth rate (AGV) of the Vosoritide treatment group was 1.
57 cm higher than that of the placebo group, which was statistically significant
.
Safety of Voxzogo The most common side effects of Voxzogo include injection site reactions, vomiting and lowered blood pressure
.
The lowering of blood pressure is listed as a warning in the drug insert, which means that this is a potentially serious side effect
.
Dr.
Teresa Keho, MD, Director of the Department of General Endocrinology, FDA Drug Evaluation and Research Center, said: “There are tens of thousands of related pediatric patients in the United States, and the approval of the drug meets this clinical need
.
Chondrodysplasia leads to children with short stature.
Treatment options
.
" Yimaitong compiled and compiled from: FDA.
FDA Approves First Drug to Improve Growth in Children with Most Common Form of Dwarfism[EB/OL].
[2021-11-19].
FDA Approves First Drug to Improve Growth in Children with Most Common Form of Dwarfism.
.
News from the official website: Recently, the U.
S.
Food and Drug Administration (FDA) approved the injectable preparation Voxzogo for use in children with "open" achondroplasia (growth plates) 5 years of age and older
.
Achondroplasia is the most common cause of dwarfism, and the approval of the drug means that these children are still likely to grow taller
.
Chondroplasia-a genetic disease that makes people grow taller.
Chondroplasia is a genetic disease that causes severe short stature and unbalanced growth
.
It is caused by mutations in the FGFR3 gene .
FGFR3 is a negative regulator of bone growth.
Mutations in this gene can inhibit the proliferation and differentiation of chondrocytes in the growth plate of patients, leading to long bone growth disorders.
As a result, children's growth slows down.
The average height of adult patients is about 1.
2 meters
.
Voxzogo's mechanism of action Vosoritide is a C-type natriuretic peptide (CNP) analog derived from natural human peptide.
Natural human peptide is a positive regulator of bone growth and can stimulate endochondral ossification
.
By inhibiting the overactive FGFR3 pathway, CNP can stimulate the proliferation and differentiation of chondrocytes in the growth plate, promote the growth of long bones caused by cartilage internalization, and help patients restore normal growth
.
The safety and effectiveness of Voxzogo were evaluated in a one-year, double-blind, placebo-controlled Phase 3 study of 121 participants who were five and older with achondroplasia and open epiphyses
.
At 52 weeks, the annualized growth rate (AGV) of the Vosoritide treatment group was 1.
57 cm higher than that of the placebo group, which was statistically significant
.
Safety of Voxzogo The most common side effects of Voxzogo include injection site reactions, vomiting and lowered blood pressure
.
The lowering of blood pressure is listed as a warning in the drug insert, which means that this is a potentially serious side effect
.
Dr.
Teresa Keho, MD, Director of the Department of General Endocrinology, FDA Drug Evaluation and Research Center, said: “There are tens of thousands of related pediatric patients in the United States, and the approval of the drug meets this clinical need
.
Chondrodysplasia leads to children with short stature.
Treatment options
.
" Yimaitong compiled and compiled from: FDA.
FDA Approves First Drug to Improve Growth in Children with Most Common Form of Dwarfism[EB/OL].
[2021-11-19].
FDA Approves First Drug to Improve Growth in Children with Most Common Form of Dwarfism.
