Sarepta Therapeutics Completes Submission of New Drug Application Seeking Approval of Casimersen (SRP-4045) for Patients with Duchenne Muscular Dystrophy Amenable to Skipping Exon 45
- Casimersen is designed for the treatment of exon 45 amenable patients, approximately eight percent of patients with Duchenne
- Casimersen is the third exon-skipping medicine using the Company’s proprietary PMO RNA-based platform.
CAMBRIDGE, Mass., June 26, 2020 (GLOBE NEWSWIRE) >Original press release here
Sarepta Therapeutics, Inc. has completed the submission of a rolling New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) seeking accelerated approval for casimersen (SRP-4045). Casimersen, a phosphorodiamidate morpholino oligomer (PMO), is engineered to treat patients with Duchenne muscular dystrophy (DMD) 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.
Doug Ingram, president and chief executive officer, Sarepta Therapeutics. – “The completion of our casimersen submission is an important milestone in our journey to advance treatments for the greatest possible number of people living with Duchenne muscular dystrophy. If approved, casimersen will be our third approved therapy for sub-populations of Duchenne. Together with our other approved therapies, we have the potential to treat nearly 30% of Duchenne patients in the United States. Our proprietary PMO platform is an important focus of our pipeline, and we owe our clinical progress to the patients and families participating in our studies.”
About Casimersen
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 the production of an internally truncated dystrophin protein.
What about Canada?
At this moment, casimersen is not available in Canada. We hope that shortly, Sarepta Therapeutics will file a request for marketing approval with Health Canada.
For a better understanding
- * The western blot is a widely used analytical technique in molecular biology, immunogenetics and other molecular biology disciplines to detect specific proteins in a sample of tissue homogenate or extract.
- * Ribonucleic acid (RNA) is a polymeric molecule essential in various biological roles in coding, decoding, regulation and expression of genes. RNA and DNA are nucleic acids and, along with lipids, proteins and carbohydrates, constitute the four major macromolecules essential for all known forms of life. Like DNA, RNA is assembled as a chain of nucleotides, but unlike DNA, it is more often found in nature as a single-strand folded onto itself, rather than a paired double-strand.
- Read more here
- Grounded in the DMD community
About DMD
Duchenne muscular dystrophy (DMD) is a disease that almost exclusively affects boys and whose incidence is 1 in 3,500. It is sporadic that Duchenne muscular dystrophy (DMD) will affect girls. Those affected are usually diagnosed around the age of five, but symptoms may be visible from early childhood. It is a degenerative disease of the muscles caused by a genetic mutation. The Duchenne muscular dystrophy (DMD) – for which no treatment is currently available – directly affects skeletal muscles. Without treatment, the consequences of the disease are dire for those afflicted and their families.
About Sarepta Therapeutics
The Company has built an impressive position in Duchenne muscular dystrophy (DMD) and in gene therapies for limb-girdle muscular dystrophies (LGMDs), mucopolysaccharidosis type IIIA, Charcot-Marie-Tooth (CMT), and other CNS-related disorders, with more than 40 programs in various stages of development. The Company’s programs and research focus span several therapeutic modalities, including RNA, gene therapy and gene editing. For more information, please visit www.sarepta.com or follow them on Twitter, LinkedIn, Instagram and Facebook.
