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U.S. FDA Submission of New Drug Application for NS-065/NCNP-01 (Viltolarsen)

Nippon Shinyaku Co., Ltd. announced that it has completed the submission of its rolling New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for NS-065/NCNP-01 (viltolarsen), which is under development for the treatment of Duchenne muscular dystrophy (DMD) amenable to exon 53 skipping.

DMD is an inherited muscle disorder that male children develop. It causes a loss of muscle power due to a deficiency of normal dystrophin, a protein involved in constructing the framework of muscle cells. Because there is no effective treatment for patients with DMD amenable to exon 53 skipping other than steroids, it is expected that this would be the first effective new treatment for this population.

Viltolarsen is a drug candidate which is expected to generate functional dystrophin proteins and be effective for DMD amenable to dystrophin exon 53 skipping.

U.S. FDA Submission Of New Drug Application For NS-065/NCNP-01 (Viltolarsen)