Dudsy Muscular Dystrophy (DMD) Gene Therapy! FDA grants Sarepta/Roche SRP-9001 Fast Track qualification!

26 July 2020 /PRNewswire/ -- Sarepta Therapeutics is a leader in precision genetic medicine for rare diseases., the company announced that the U.S. Food and Drug Administration (FDA) has granted SRP-9001 (AAVrh74.MHCK7.micro-dystrophin) fast-track (FTD) for the treatment of Duchs muscular dystrophy (DMD).SRP-9001 is a gene transfer therapy designed to transfer genes encoded micro-dystrophin to muscle tissue to produce the target protein, the micromaltrophin.December 2019, Roche and Sarepta signed a $2.85 billion ($750 million in cash, $400 million equity investment, $1.7 billion potential milestone payment) license, acquiring exclusive rights to SRP-9001 outside the United States.Sarepta, which is responsible for the global development and manufacture of SRP-9001 and plans to commercialize SRP-9001 in the United States, has exclusive rights to the micro-malnutrition gene therapy program originally developed by the Abigail-Wexner Institute of the National Children's Hospital of the United States.Fast Track Qualification (FTD) is designed to accelerate the development and rapid review of new drugs for serious diseases to address critically unmet medical needs in key areas.'s FTD recognition for the drug in research means that drug companies can meet more frequently with the FDA during the development phase, be eligible for expedited approval and priority reviews if they meet the relevant criteria after submitting a listing application, and the opportunity for rolling reviews.in addition to FTD, SRP-9001 has been granted rare pediatric disease (RPD) status by the FDA.SRP-9001 was previously granted Orphan Medicine (ODD) in the United States, the European Union and Japan., the journal JAMAy recently published one-year safety and tolerance data for four clinical trial subjects treated with SRP-9001 in the Study 101 trial, a randomized, double-blind, placebo-controlled study evaluating SRP-9001, which is currently under way and is expected to be published in early 2021.Du's muscular dystrophy (DMD) is a rare and fatal neuromuscular genetic disease that occurs in one in every 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 stunting, 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 arms, neck and other parts.most patients need full-time use of a wheelchair in their teens 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. at the end of June, Sarepta announced that it had completed rolling applications for new drugs (NDA) to the FDA to seek accelerated approval of Casimersen (SRP-4045), a phosphoric acid diamide protomelobody (PMD) for dMD patients with genetically confirmed mutations that are suitable for skipping exon 45 treatment. 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 the myotrophic pre mRNA to exclude or "skip" the exon during mRNA treatment in DMD patients carrying a genetic mutation that is suitable for skipping the exon No. 45. exon skips to allow the production of an internally cut-out myotrophic protein. note, casimersen is the third exon skipping drug using Sarepta's proprietary PMO RNA platform, specifically designed to treat children with DMD who are suitable for skipping extrinsic no. 45, which accounts for about 8 percent of the total number of DMD patients. previously, Sarepta obtained FDA approval for two exosome skipping drugs developed using its proprietary PMO RNA chemistry and exosome skip technology: (1) In 2019, Vyondys 53 (golodirsen) was approved to treat DMD patients who were tested and tested for treatment with exon 53 skipping, which accounts for about 8 percent of the total number of DMD patients. (2) 2016, Exondys 51 (eteplirsen) was approved for the treatment of DMD patients who have been tested and tested for treatment with skip 51, about 12% of the total number of DMD patients. unlike dMD patients with specific genetic mutations, SRP-9001 is a potentially curable treatment for all DMD patients, unlike the three exosome skip therapies mentioned above. its advantage is that SRP-9001 has the potential to have therapeutic effects wherever the gene mutation in DMD patients is present in the anti-mytrophin gene. () Original source: Sarepta Therapeutics Receivs Fast Track Mar for SRP-9001 Micro-Dystrophin Gene for Treatment of Duchenne Dystrophy.