-
Categories
-
Pharmaceutical Intermediates
-
Active Pharmaceutical Ingredients
-
Food Additives
- Industrial Coatings
- Agrochemicals
- Dyes and Pigments
- Surfactant
- Flavors and Fragrances
- Chemical Reagents
- Catalyst and Auxiliary
- Natural Products
- Inorganic Chemistry
-
Organic Chemistry
-
Biochemical Engineering
- Analytical Chemistry
-
Cosmetic Ingredient
- Water Treatment Chemical
-
Pharmaceutical Intermediates
Promotion
ECHEMI Mall
Wholesale
Weekly Price
Exhibition
News
-
Trade Service
On February 15, the FDA approved the Casimersen (trade name: Ammondys 45) developed by Sarepta Therapeutics for the treatment of patients with Duchenne progressive muscular dystrophy (DMD) with a gene mutation exon 45 skipping type.
This is the first drug approved for this type of genetic mutation DMD.
DMD is a recessive genetic disease of the X chromosome.
It is a muscular wasting disease caused by mutations in the dystrophin gene.
There are 79 exons in the dystrophin gene.
Exon mutations in DMD patients are mainly concentrated in the exons.
Exons 45-55.
The frameshift mutation of DMD gene directly causes the generated messenger nucleic acid (mRNA) to not encode the working dystrophin protein, and the exon deletion and frameshift cause severe symptoms of DMD.
Casimersen is an antisense oligonucleotide therapy.
It is Sarepta Therapeutics' third exon skipping drug developed using the PMO RNA platform to target Dystroglycan.
The drug is a phosphodiamide morpholino oligomer (PMD), specifically designed to treat patients with DMD who are suitable for skipping exon 45.
Casimersen was approved to use the accelerated approval channel this time.
The drug has obtained the fast-track qualification granted by the FDA, the priority review qualification and the orphan drug qualification.
Three antisense oligonucleotide therapies for the treatment of exon skip mutation DMD have been listed before, namely Eteplirsen (exon 51 skip mutation DMD), Viltolarsen (53 exon skip mutation DMD) and Golodirsen (53 exon Sub-jumping mutation (DMD).
Competition Casimersen details and Dystroglycan regulators see below :( click to enlarge) Author: Sea Editor: drugs crossing the sea of data produced, reproduced without permission prohibited fourth paragraph 1.
Global CAR-T therapy, BMS Breyanzi approved by the FDA 2.
In January 2021, the world's first batch of 3 new drugs, Bayer's new anti-heart failure drug Vericiguat won the FDA's first batch 3.
The world's first new chemotherapy drug for bone marrow protection Trilaciclib, a different CDK4/6 inhibitor 4.
Regeneration First-in -class lipid-lowering new drug Evinacumab received FDA approval 5.
FDA accelerated approval of new lymphoma drug Umbralisib
This is the first drug approved for this type of genetic mutation DMD.
DMD is a recessive genetic disease of the X chromosome.
It is a muscular wasting disease caused by mutations in the dystrophin gene.
There are 79 exons in the dystrophin gene.
Exon mutations in DMD patients are mainly concentrated in the exons.
Exons 45-55.
The frameshift mutation of DMD gene directly causes the generated messenger nucleic acid (mRNA) to not encode the working dystrophin protein, and the exon deletion and frameshift cause severe symptoms of DMD.
Casimersen is an antisense oligonucleotide therapy.
It is Sarepta Therapeutics' third exon skipping drug developed using the PMO RNA platform to target Dystroglycan.
The drug is a phosphodiamide morpholino oligomer (PMD), specifically designed to treat patients with DMD who are suitable for skipping exon 45.
Casimersen was approved to use the accelerated approval channel this time.
The drug has obtained the fast-track qualification granted by the FDA, the priority review qualification and the orphan drug qualification.
Three antisense oligonucleotide therapies for the treatment of exon skip mutation DMD have been listed before, namely Eteplirsen (exon 51 skip mutation DMD), Viltolarsen (53 exon skip mutation DMD) and Golodirsen (53 exon Sub-jumping mutation (DMD).
Competition Casimersen details and Dystroglycan regulators see below :( click to enlarge) Author: Sea Editor: drugs crossing the sea of data produced, reproduced without permission prohibited fourth paragraph 1.
Global CAR-T therapy, BMS Breyanzi approved by the FDA 2.
In January 2021, the world's first batch of 3 new drugs, Bayer's new anti-heart failure drug Vericiguat won the FDA's first batch 3.
The world's first new chemotherapy drug for bone marrow protection Trilaciclib, a different CDK4/6 inhibitor 4.
Regeneration First-in -class lipid-lowering new drug Evinacumab received FDA approval 5.
FDA accelerated approval of new lymphoma drug Umbralisib
