Drugs / CLEMIDSOGENE LANPARVOVEC
CLEMIDSOGENE LANPARVOVEC
Regulatory milestones approvals, filings & regulatory actions · 4 recorded
| Milestone | Jurisdiction | Brand | Indication | Date | Sentence it was read from |
|---|---|---|---|---|---|
| Clinical hold | US (FDA) | — | mucopolysaccharidosis type 2 | 2026-08-24 | “The U.S. Food and Drug Administration (FDA) placed a clinical hold on RGX-121 following the discovery of asymptomatic spine MRI findings” regenxbio.com ↗ |
| Rejected (CRL) | US (FDA) | — | mucopolysaccharidosis type 2 | 2026-02-07 | “Ultimately, the FDA did not agree the data set provided substantial evidence of effectiveness to support approval of RGX-121 for the treatment of MPS II.” regenxbio.com ↗ “FDA issues Complete Response Letter for RGX-121 (clemidsogene lanparvovec) for treatment of Mucopolysaccharidosis II (MPS II)” regenxbio.com ↗ |
| Clinical hold | US (FDA) | — | mucopolysaccharidosis type 2 | 2026-01-28 | “The FDA also placed a clinical hold on RGX-121, for the treatment of MPS II, also known as Hunter Syndrome” regenxbio.com ↗ |
| Filed | US (FDA) | — | mucopolysaccharidosis type 2 | — | “In May 2025, the FDA accepted the RGX-121 BLA under the accelerated approval pathway.” regenxbio.com ↗ |
Trials 2
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 1/21 trial | ||||||
| Phase 1/2 | NCT04571970 | Mar 2021 → May 2023 | mucopolysaccharidosis type 2 | REGENXBIO Inc. | Completed | No outcome recorded |
| Phase 2/31 trial | ||||||
| Phase 2/3 | NCT03566043 | Sep 2018 → Nov 2023 overdue | mucopolysaccharidosis type 2 | REGENXBIO Inc. | Active not recruiting | No outcome recorded |
News releases announcing trial results or a regulatory action · 8
| Date | Issuer | Release |
|---|---|---|
| 2026-08-25 | Nippon Shinyaku Co., Ltd. | Regulatory REGENXBIO社のムコ多糖症Ⅱ型治療薬「RGX-121」に関する規制当局対応の最新情報についてのお知らせ nippon-shinyaku.co.jp ↗
日本新薬株式会社（本社：京都市南区、代表取締役社長：中井 亨、以下「当社」）は、ムコ多糖症Ⅱ型（MPSⅡ、ハンター症候群）治療薬として開発中のRGX-121について、REGENXBIO Inc.（本社：米国メリーランド州ロックビル、最高経営責任者（CEO）：Curran M. Simpson、以下「REGENXBIO社」）が米国食品医薬品局（FDA）より、臨床試験の実施保留命令（クリニカル・ホールド）を受けたことをお知らせいたします。 |
| 2026-08-24 | REGENXBIO Inc. | Regulatory REGENXBIO Announces Regulatory Update on RGX-121 for MPS II regenxbio.com ↗
REGENXBIO does not expect to resubmit the RGX-121 Biologics License Application (BLA) in the near term. |
| 2026-06-22 | REGENXBIO Inc. | Regulatory REGENXBIO Announces Alignment with FDA on Path Forward for NAVSUNLI™ BLA Resubmission for Accelerated Approval; First Potential Gene Therapy for MPS II regenxbio.com ↗
The FDA asked the Company to request a Type A meeting to review existing longer-term biomarker and clinical data and to resubmit the Biologics License Application (BLA) following this meeting. |
| 2026-05-14 | REGENXBIO Inc. | Regulatory REGENXBIO Reports First Quarter 2026 Financial Results and Operational Highlights regenxbio.com ↗
The FDA has lifted the partial clinical hold on RGX-121. |
| 2026-02-10 | Nippon Shinyaku Co., Ltd. | Regulatory RGX-121の生物製剤承認申請に関する審査完了報告通知の受領について nippon-shinyaku.co.jp ↗
日本新薬株式会社（本社：京都市南区、代表取締役社長：中井 亨、以下「当社」）は、ムコ多糖症Ⅱ型（以下「MPSⅡ」）を期待適応症とするRGX-121(clemidsogene lanparvovec、以下「本剤」）について、REGENXBIO Inc.（本社：米国メリーランド州ロックビル、最高経営責任者（CEO）：Curran M. Simpson、以下「REGENXBIO社」）が米国FDA（食品医薬局）から審査完了報告通知（Complete Response Letter、以下「CRL」）を受領したことをお知らせします。 |
| 2026-02-09 | REGENXBIO Inc. | Regulatory REGENXBIO Announces Regulatory Update on RGX-121 BLA for MPS II regenxbio.com ↗
FDA issues Complete Response Letter for RGX-121 (clemidsogene lanparvovec) for treatment of Mucopolysaccharidosis II (MPS II) |
| 2026-01-28 | REGENXBIO Inc. | Regulatory REGENXBIO Announces Regulatory Update on Ultra Rare MPS Programs regenxbio.com ↗
The FDA also placed a clinical hold on RGX-121, for the treatment of MPS II, also known as Hunter Syndrome, citing the similarities in products, study populations, and shared risk between the clinical studies. |
| 2025-08-19 | Nippon Shinyaku Co., Ltd. | Regulatory RGX-121（clemidsogene lanparvovec）： 米国FDAによる審査期間延長に関するお知らせ nippon-shinyaku.co.jp ↗
本剤の審査終了目標日（PDUFA date）は当初の2025年11月9日（米国時間）から2026年2月8日（米国時間）に変更されました。 |
All press releases naming this drug 11 releases
Evidence & citations 5 cited values
Every value below carries the sentence it was read from. 3 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | CLEMIDSOGENE LANPARVOVEC | ChEMBL registry synonym — accepted as the source's own label CHEMBL5314820 ↗ |
| Known as | Clemidsogen lanparvovec | ChEMBL registry synonym — accepted as the source's own label CHEMBL5314820 ↗ |
| Known as | RGX-121 | ClinicalTrials.gov intervention name — accepted as the source's own label NCT03566043 ↗ |
| Modality | Gene therapy (AAV / viral vector) | “RGX-121 is a gene therapy” NCT03566043 ↗ |
| Route | Other | “administered by IC or ICV injection” NCT04571970 ↗ |