Feb. 7, 2020 - KYOTO, Japan and PARAMUS, NJ:
Just a portion of press release: "The viltolarsen NDA includes results from a Phase 2 study and its long-term extension study in North America -- as well as a Phase 1 and a Phase 1/2 study in Japan. Both the Phase 1/2 and Phase 2 studies evaluated changes in dystrophin levels and motor function across two doses (40 mg/kg and 80 mg/kg weekly dose groups).
The PDUFA date for viltolarsen is within the 3rd quarter (July-September) of 2020. The PDUFA date is the target date the FDA provides a decision on the approval of a new drug.
Viltolarsen represents one of the most extensively studied antisense therapies in DMD. Viltolarsen, if approved by the FDA, would represent a new treatment option for DMD patients amenable to exon 53 skipping in the United States."