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▎WuXi AppTec Content Team Editor Today, Sanofi announced that the Japanese Ministry of Health, Labour and Welfare (MHLW) has approved the company's innovative enzyme replacement therapy Xenpozyme (olipudase alfa) for the treatment of acid sphingomyelinase deficiency ( acid sphingomyelinase deficiency, ASMD) non-central nervous system symptoms in adult and pediatric patients
.
This is a rare and potentially fatal genetic disorder
.
The release notes that Xenpozyme is the first approved treatment for ASMD
.
Xenpozyme is a recombinant human acid sphingomyelinase protein used to replace missing or defective acid sphingomyelinase (ASM)
.
It is responsible for degrading sphingomyelin
.
ASMD patients have mutations in the gene encoding this metabolic enzyme, resulting in the accumulation of sphingomyelin in cells, causing damage to the lungs, spleen, liver and other organs, which may lead to premature death
.
The approval of Xenpozyme in Japan is based on clinical trials conducted in adult and pediatric patients, which showed that Xenpozyme improved lung function and reduced spleen and liver volume, while demonstrating good safety and tolerance in both adult and pediatric patients.
acceptability
.
Outside of Japan, the Biologics License Application (BLA) for olipudase alfa has been granted priority review status by the US FDA, and a review decision is expected in the third quarter of this year
.
It has been granted Breakthrough Therapy Designation by the US FDA
.
Image credit: 123RF Sanofi Executive Vice President and Global Head of R&D John Reed, PhD, said: "Today's approval of Xenpozyme is a watershed moment for ASMD patients and their families
.
It represents 20 years of scientific research and the combined efforts of patient organizations, clinicians and patients
.
As the world's first approved treatment for ASMD, Xenpozyme offers a potentially transformative treatment option for this patient population
.
We will work with global regulators to bring this important medicine to ASMD patients around the world as quickly as possible
.
"Reference: [1] Xenpozyme® (olipudase alfa) approved in Japan, first and only approved therapy indicated to treat acid sphingomyelinase deficiency.
Retrieved March 28, 2022, from https:// 2022/03/28/2410648/0/en/Press-Release-Xenpozyme-olipudase-alfa-approved-in-Japan-first-and-only-approved-therapy-indicated-to-treat-acid-sphingomyelinase-deficiency.
html Disclaimer: The WuXi AppTec content team focuses on introducing the progress of global biomedical health research
.
This article is for information exchange purposes only.
The views in this article do not represent WuXi AppTec's position, nor do they support or oppose the views in the article
.
This article is not Recommended treatment plan
.
If you need treatment plan guidance, please go to a regular hospital for treatment
.
.
This is a rare and potentially fatal genetic disorder
.
The release notes that Xenpozyme is the first approved treatment for ASMD
.
Xenpozyme is a recombinant human acid sphingomyelinase protein used to replace missing or defective acid sphingomyelinase (ASM)
.
It is responsible for degrading sphingomyelin
.
ASMD patients have mutations in the gene encoding this metabolic enzyme, resulting in the accumulation of sphingomyelin in cells, causing damage to the lungs, spleen, liver and other organs, which may lead to premature death
.
The approval of Xenpozyme in Japan is based on clinical trials conducted in adult and pediatric patients, which showed that Xenpozyme improved lung function and reduced spleen and liver volume, while demonstrating good safety and tolerance in both adult and pediatric patients.
acceptability
.
Outside of Japan, the Biologics License Application (BLA) for olipudase alfa has been granted priority review status by the US FDA, and a review decision is expected in the third quarter of this year
.
It has been granted Breakthrough Therapy Designation by the US FDA
.
Image credit: 123RF Sanofi Executive Vice President and Global Head of R&D John Reed, PhD, said: "Today's approval of Xenpozyme is a watershed moment for ASMD patients and their families
.
It represents 20 years of scientific research and the combined efforts of patient organizations, clinicians and patients
.
As the world's first approved treatment for ASMD, Xenpozyme offers a potentially transformative treatment option for this patient population
.
We will work with global regulators to bring this important medicine to ASMD patients around the world as quickly as possible
.
"Reference: [1] Xenpozyme® (olipudase alfa) approved in Japan, first and only approved therapy indicated to treat acid sphingomyelinase deficiency.
Retrieved March 28, 2022, from https:// 2022/03/28/2410648/0/en/Press-Release-Xenpozyme-olipudase-alfa-approved-in-Japan-first-and-only-approved-therapy-indicated-to-treat-acid-sphingomyelinase-deficiency.
html Disclaimer: The WuXi AppTec content team focuses on introducing the progress of global biomedical health research
.
This article is for information exchange purposes only.
The views in this article do not represent WuXi AppTec's position, nor do they support or oppose the views in the article
.
This article is not Recommended treatment plan
.
If you need treatment plan guidance, please go to a regular hospital for treatment
.
