Drugs / Viltolarsen
Viltolarsen
Regulatory milestones approvals, filings & regulatory actions · 1 recorded
| Milestone | Jurisdiction | Brand | Indication | Date | Sentence it was read from |
|---|---|---|---|---|---|
| Approved | US (FDA) | VILTEPSO | — | 2020-08-12 | fda.gov ↗ |
Trials 8
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 11 trial | ||||||
| Phase 1 | NCT02081625 | Jun 2013 → Nov 2014 | Duchenne muscular dystrophy | National Center of Neurology and Psychiatry, Japan | Completed | No outcome recorded |
| Phase 23 trials · 1 met primary | ||||||
| Phase 2 | NCT04956289 | Jul 2021 → Jun 2023 | Duchenne muscular dystrophy | NS Pharma, Inc. | Completed | No outcome recorded |
| Phase 2 | NCT03167255 | Jul 2017 → Oct 2021 | Duchenne muscular dystrophy | NS Pharma, Inc. | Completed | No outcome recorded |
| Phase 2 | NCT02740972 | Dec 2016 → Mar 2018 | Duchenne muscular dystrophy | NS Pharma, Inc. | Completed | Met primary |
| Phase 32 trials | ||||||
| Phase 3 | NCT04768062 | Apr 2021 → Oct 2025 | Duchenne muscular dystrophy | NS Pharma, Inc. | Unknown | No outcome recorded |
| Phase 3 | NCT04060199 | Apr 2020 → Oct 2023 | Duchenne muscular dystrophy | NS Pharma, Inc. | Completed | No outcome recorded |
| Phase 41 trial | ||||||
| Phase 4 | NCT04687020 | Jun 2021 → Sep 2032 expected | Duchenne muscular dystrophy | NS Pharma, Inc. | Active not recruiting | No outcome recorded |
| Phase not stated1 trial | ||||||
| — | NCT04337112 | — | Duchenne muscular dystrophy | NS Pharma, Inc. | Approved for marketing | No outcome recorded |
News releases announcing trial results or a regulatory action · 9
| Date | Issuer | Release |
|---|---|---|
| 2024-05-27 | NS Pharma, Inc. | Results NS Pharma Shares Preliminary Results of Viltolarsen (NS-065 / NCNP-01) Phase 3 Clinical Trial (RACER53 Study) nspharma.com ↗
NS Pharma, Inc. (NS Pharma), a subsidiary of Nippon Shinyaku Co., Ltd., announced today that it has received preliminary analysis results from the global Phase 3 clinical trial (RACER53 study, NCT04060199) of NS-065/NCNP-01 (generic name: viltolarsen). |
| 2024-03-06 | NS Pharma, Inc. | Results NS Pharma, Inc. Shares, New VILTEPSO® (Viltolarsen) Data at the MDA Clinical & Scientific Conference 2024 nspharma.com ↗
The study also found that the upper limb motor function of participants was stabilized over 49 weeks in both ambulatory and non-ambulatory patients. |
| 2023-05-09 | NS Pharma, Inc. | Results VILTEPSO® (viltolarsen) injection Four-Year Clinical Trial Data Published in the Journal of Neuromuscular Diseases nspharma.com ↗
The article, “Efficacy and Safety of Viltolarsen in Boys With Duchenne Muscular Dystrophy: Results From the Phase 2, Open-Label, 4-Year Extension Study,” is freely available under open access (click here). |
| 2022-10-24 | NS Pharma, Inc. | Results NS Pharma Reports on Results of VILTEPSO® (viltolarsen) injection After Four Years of Treatment in Open-Label Extension Trial in Duchenne Muscular Dystrophy nspharma.com ↗ |
| 2021-04-01 | NS Pharma, Inc. | Regulatory NS Pharma’s VILTEPSO® (viltolarsen) Receives Permanent J-code from Centers for Medicare and Medicaid Services nspharma.com ↗
NS Pharma, Inc. (NS Pharma; President, Tsugio Tanaka), a wholly owned subsidiary of Nippon Shinyaku Co., Ltd. (Nippon Shinyaku; President, Shigenobu Maekawa), announced that the J-code assigned by the Centers for Medicare and Medicaid Services (CMS) for VILTEPSO® (viltolarsen) injection is effective today. |
| 2020-08-12 | NS Pharma, Inc. | Regulatory NS Pharma’s VILTEPSOTM (viltolarsen) injection Now FDA-Approved in the U.S. for the Treatment of Duchenne Muscular Dystrophy in Patients Amenable to Exon 53 Skipping Therapy nspharma.com ↗
NS Pharma, Inc. (NS Pharma; President, Tsugio Tanaka), a wholly owned subsidiary of Nippon Shinyaku Co., Ltd. (Nippon Shinyaku; President Shigenobu Maekawa), announced today that the U.S. Food & Drug Administration (FDA) has approved VILTEPSO™ (viltolarsen) injection for patients with Duchenne muscular dystrophy (DMD) who are amenable to exon 53 skipping therapy. |
| 2016-11-09 | Nippon Shinyaku Co., Ltd. | Regulatory 米国で開発中の核酸医薬品NS-065/NCNP-01、米国FDAよりファストトラック指定受理のお知らせ nippon-shinyaku.co.jp ↗
日本新薬株式会社は、米国で開発を進める国産初の核酸医薬品NS-065/NCNP-01（以下、本剤） ※1 について、その適応疾患となるデュシェンヌ型筋ジストロフィー（DMD）に対し、米国食品医薬品局（FDA）からファストトラック指定を受理しましたのでお知らせします。 |
| 2015-10-27 | Nippon Shinyaku Co., Ltd. | Regulatory 核酸医薬品「NS-065/NCNP-01」が、厚生労働省より先駆け審査指定制度の対象品目に指定されました nippon-shinyaku.co.jp ↗
本日、厚生労働省より、患者さんに世界で最先端の治療薬を最も早く提供することを目指し、一定の要件を満たす画期的な新薬等を指定する「先駆け審査指定制度」の対象品目が6品目発表され、そのひとつに日本新薬が開発中である核酸医薬品、デュシェンヌ型筋ジストロフィー治療剤「NS-065/NCNP-01」が指定されました。 |
| 2015-03-23 | Nippon Shinyaku Co., Ltd. | Results デュシェンヌ型筋ジストロフィー治療剤NS-065/NCNP-01の早期探索的臨床試験の終了のお知らせ nippon-shinyaku.co.jp ↗
国産初のアンチセンス核酸医薬品であるデュシェンヌ型筋ジストロフィー治療剤NS-065/NCNP-01の早期探索的臨床試験のすべての投与が終了し、初期の解析において、本剤の治療効果を予測するジストロフィンタンパク質の発現が確認されました。 |
All press releases naming this drug 17 releases
Evidence & citations 7 cited values
Every value below carries the sentence it was read from. 9 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | viltolarsen | “INTERVENTIONS: Participants received 40 mg/kg (low dose) or 80 mg/kg (high dose) of viltolarsen administered by weekly intravenous infusion.” PMID 32453377 ↗ Aug 2020 |
| Known as | NCNP-01 | ChEMBL registry synonym — accepted as the source's own label CHEMBL4298062 ↗ |
| Known as | NS-065 | ChEMBL registry synonym — accepted as the source's own label CHEMBL4298062 ↗ |
| Known as | NS-065/NCNP-01 | ClinicalTrials.gov intervention name — accepted as the source's own label NCT02081625 ↗ |
| Known as | Viltepso | ChEMBL registry synonym — accepted as the source's own label CHEMBL4298062 ↗ |
| Action | Restore | “Systemic treatment of participants with DMD with viltolarsen induced de novo dystrophin production” PMID 32453377 ↗ Aug 2020 |
| Route | Intravenous | “Study dosages will be infused over a 1 hour period” NCT02081625 ↗ |