Dushi muscular dystrophy (DMD) new drug! Sarepta's 3rd exomer jump therapy casimersen is listed in the United States!

, June 28, 2020 /PR
Newswire/ -- Sarepta Therapeutics is a biotechnology company focused on developing precision gene therapy for the treatment of rare diseasesRecently, the company announced that it has completed a rolling submission of new drug applications (NDA) to the U.SFood and Drug Administration (
FDA) to seek accelerated approval of Casimersen (SRP-4045), a phosphoric acid diamide protoolinate (PMD) that is genetically proven to be a mutation and is suitable for patients with Douemuscular dystrophy (DMD) that has been identified by genetic testing to skip the treatment of the 45th exon ( skipping exon 45)note, casimersen is the third exon to skip the drugusing Sarepta's proprietary PMO RNA platform, specifically designed for the treatment of DMD patients who are suitable for skipping exempat No45, which accounts for about 8% of the total number of DMD patientsthe completion of the rolling submission includes data from the CLASS of studies (also known as the 4045-301 study) casimersen therapy groupThis is a global, randomized, double-blind, placebo-controlled Phase III study that evaluates the efficacy and safety of DMD patients who are treated with treatments suitable for skipping exnomes45 and 53an interim analysis from the study showed that patients treated with casimersen were treated with a statistically significant increase in the production of myotrophic protein sein compared to baselines and placebosThe study is still ongoing and remains blind to collect more efficacy and safety dataIf the casimersen NDA is accepted and accelerated, the completed ESSENCE study will be used as a post-ipo confirmation studyDudsiyomy (DMD) is a rare and fatal neuromusculargenetic disease that occurs in one in 3,500-5,000 men worldwide DMD is caused by changes or mutations in genes that encode myotrophic proteins Symptoms of DMD usually appear in infants and young children Affected children may experience developmental delays, such as walking, climbing stairs or standing from a sitting position With the development of the disease, the lower limb muscle weakness spread to the arm, neck and other parts Most patients need full-time use of a wheelchair as a teenager and then gradually lose the ability to perform daily activities independently, such as using the restroom, bathing and feeding Eventually, increased breathing difficulties due to insufficiency of the respiratory muscles require ventilation support, and heart failure can lead to heart failure The disease is widespread and fatal, and patients usually die of the disease in their 20s casimersen is an antisense oligonucleotide drug that uses Sarepta's proprietary phosphoric lydiatidydia (PMO) chemical and exon skipping techniques, skipping the DMD gene No 45 exon Casimersen is designed to bind the exon No 45 of myotrophic protein pre mRNA to exclude or "skip" the exon during mRNA treatment in DMD patients who carry a genetic mutation that is suitable for skipping the exon No 45 exon Exon skipping is intended to allow the production of an internally cut myotrophic protein so far, Sarepta has won u.S using two drugs developed using PMO chemistry and exon skip technology FDA approval: (1) In 2019, Vyondys 53 (golodirsen) was approved for the treatment of DMD patients who have been tested and tested for treatment with exon 53 skipping, which accounts for approximately 8% of the total number of DMD patients (2) In 2016, Exondys 51 (eteplirsen) was approved for the treatment of DMD patients who have been tested and tested for treatment with skip 51 exskipp51, which accounts for approximately 12% of the total number of DMD patients "Completing the Casimersen NDA submission is an important milestone for us to provide treatment to as many DMD patients as possible," said Doug Ingram, President and CEO of Sarepta Therapeutics If approved, casimersen will be our third approved treatment for DMD Along with our other approved therapies, we have the potential to treat nearly 30% of DMD patients in the United States "
(biovalleybioon.com) original origin: Sarepta Therapeutics Completes Submission sss op seeking approval of Casimersen (SRP-4045) for Patients with Duchenne Dystrophy Amenable to Skipping Ex45
.