Casimersen - Drug Insight and Market Forecast - 2030
発行: DelveInsight Business Research LLP
ページ情報: 英文 80 Pages
Casimersenは、Sarepta独自のホスホロジアミデートモルホリノオリゴマー（PMO）とエクソンスキッピング技術を使って、DMD遺伝子のエクソン45をスキップするための治療薬です。ジストロフィンプレmRNAのエクソン45に結合するように設計されており、エクソン45のスキップに影響されやすい遺伝子変異を有する患者のmRNA処理中にこのエクソンを除外または「スキップ」することで、内部で切断されたジストロフィンタンパク質の生産が可能になります。 2020年6月、Sarepta Therapeuticsは、エクソン45スキップを受け入れやすい遺伝子変異を有するデュシェンヌ型筋ジストロフィー（DMD）患者を治療するCasimersen（SRP-4045）の新薬承認申請（NDA）を行っています。
"Casimersen - Emerging Insight and Market Forecast - 2030" report by DelveInsight outlays comprehensive insights of the product indicated for the treatment of its approved condition. A detailed picture of the Casimersen in Seven Major Markets, i.e., United States, EU5 (Germany, France, Italy, Spain, and the United Kingdom), and Japan, for the study period 2017-2030 is provided in this report along with a detailed description of the product. The product details covers mechanism of action, dosage and administration, route of synthesis, and pharmacological studies, including product marketed details, regulatory milestones, and other development activities. Further, it also consists of market assessments inclusive of the market forecast, SWOT analysis, and detailed analyst views. It further highlights the market competitors, late-stage emerging therapies, and patent details in the global space.
Casimersen uses Sarepta's proprietary phosphorodiamidate morpholino oligomer (PMO) chemistry and exon-skipping technology to skip exon 45 of the DMD gene. Casimersen is designed to bind to exon 45 of dystrophin pre-mRNA, resulting in exclusion, or "skipping," of this exon during mRNA processing in patients with genetic mutations that are amenable to exon 45 skipping. Exon skipping is intended to allow for production of an internally truncated dystrophin protein. In June 2020, Sarepta Therapeutics has completed the submission of the casimersen (SRP-4045) New Drug Application (NDA) for the treatment of Duchenne muscular dystrophy (DMD) in patients who have genetic mutations that are amenable to skipping exon 45 of the Duchenne gene.The completion of the rolling submission includes data from the casimersen arm of the ESSENCE study (also known as study 4045-301), a global, randomized, double-blind, placebo-controlled Phase 3 study evaluating efficacy and safety in patients amenable to skipping exons 45 and 53. An interim analysis from ESSENCE demonstrated a statistically significant increase in dystrophin production as measured by western blot* in patients who received casimersen compared to baseline and placebo. The study is ongoing and remains blinded to collect additional efficacy and safety data. If the casimersen NDA is accepted and granted accelerated approval, the completed ESSENCE study will serve as a post-marketing confirmatory study.
The report provides insights into:
The report is built using data and information sourced primarily from internal databases, primary and secondary research and in-house analysis by DelveInsight's team of industry experts. Information and data from the secondary sources have been obtained from various printable and nonprintable sources like search engines, news websites, global regulatory authorities websites, trade journals, white papers, magazines, books, trade associations, industry associations, industry portals and access to available databases.
This report provides a detailed market assessment of Casimersen in Seven Major Markets, i.e., United States, EU5 (Germany, France, Italy, Spain, and the United Kingdom), and Japan. This segment of the report provides historical and forecasted sales data from 2017 to 2030.
The report provides the clinical trials information of Casimersen covering trial interventions, trial conditions, trial status, start and completion dates.