Drugs / ULEFNERSEN
Trials 1
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 3 | NCT04768972 | Jun 2021 → Jun 2026 overdue | amyotrophic lateral sclerosis | Ionis Pharmaceuticals, Inc. | Active not recruiting | No outcome recorded |
Press releases naming this drug 4 releases
| Date | Issuer | Release |
|---|---|---|
| 2026-09-22 | Otsuka Pharmaceutical Development & Commercialization, Inc. | Otsuka Announces Transformative Phase 3 FUSION Results for Ulefnersen, Bringing the FUS-ALS Community Closer to a Potential Targeted Treatment otsuka.co.jp ↗ |
| 2026-09-22 | Otsuka Pharmaceutical Development & Commercialization, Inc. | Otsuka Establishes Ulefnersen Early Access Program (EAP) for Eligible People Living with FUS-ALS otsuka-us.com ↗ |
| 2026-09-22 | Otsuka Pharmaceutical Development & Commercialization, Inc. | Otsuka Announces Transformative Phase 3 FUSION Results for Ulefnersen, Bringing the FUS-ALS Community Closer to a Potential Targeted Treatment otsuka-us.com ↗ |
| 2024-11-22 | Otsuka Pharmaceutical Development & Commercialization, Inc. | Otsuka Obtains Exclusive Global Licensing Rights from Ionis to Ulefnersen, a Potential Treatment for Amyotrophic Lateral Sclerosis Caused by Mutations in the Fused in Sarcoma Gene (FUS-ALS) otsuka.co.jp ↗ |
Evidence & citations 3 cited values
Every value below carries the sentence it was read from. 2 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | ULEFNERSEN | ChEMBL registry synonym — accepted as the source's own label CHEMBL5095349 ↗ |
| Known as | ION363 | ClinicalTrials.gov intervention name — accepted as the source's own label NCT04768972 ↗ |
| Route | Intrathecal | “ION363 will be administered by IT bolus injection.” NCT04768972 ↗ |