Rare Daily Staff

Nippon Shinyaku said that it has completed the submission of its rolling new drug application to the U.S. Food and Drug Administration for viltolarsen, its experimental therapy for 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 an experimental drug that is expected to generate functional dystrophin proteins and be effective for DMD amenable to dystrophin exon 53 skipping.

The filing comes just months after Sarepta received a complete response letter from the FDA for its exon 53 skipping DMD therapy Vyondys because of safety concerns.

Viltolarsen was granted a Rare Pediatric Disease designation, Orphan Drug designation, and Fast Track designation in the United States and SAKIGAKE designation and Orphan Drug designation in Japan.

The company also said it submitted a new drug application to the Ministry of Health, Labour and Welfare in Japan for vitolarsen at the end of September.

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