PPMD is thrilled to learn from Nippon Shinyaku Co., Ltd. and NS Pharma, Inc. that the FDA has filed a New Drug Application (NDA) for their antisense oligonucleotide drug NS-065/NCNP-01 (viltolarsen). Viltolarsen is a drug candidate that hopes to treat people with Duchenne who have a mutation in the DMD gene amenable to exon 53 skipping. 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. As you will recall, because a NDA has been filed, the review for regulatory approval will be accelerated according to Fast Track Designation by FDA. In addition, under the Prescription Drug User Fee Act (PDUFA), the FDA has set a target action date of 3Q of 2020 (July-September).
We look forward to working with the NS Pharma team to learn more about next steps, including how PPMD can help, and we are so grateful to all of the families in the US and overseas who have participated in this trial. We are hopeful that viltolarsen will be another approved therapeutic option to treat Duchenne for those amenable to exon 53 skipping.
Read NS Pharma’s Press Release
NDA Accepted for Filing by the FDA for Antisense Oligonucleotide Viltolarsen (NS-065/NCNP-01)
KYOTO, Japan and PARAMUS, NJ: February 7, 2020 – Nippon Shinyaku Co., Ltd. (President, Shigenobu Maekawa) and NS Pharma, Inc. (President, Tsugio Tanaka), a wholly owned subsidiary of Nippon Shinyaku, announced today that the U.S. Food & Drug Administration (FDA) has accepted the filing of our New Drug Application (NDA) under the priority review for viltolarsen in patients with Duchenne Muscular Dystrophy (DMD) who are amenable to exon 53 skipping therapy. In addition to priority review, the FDA previously granted viltolarsen with Fast Track, Orphan Drug and Rare Disease designations.
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 3 rd 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.