Drugs / VCA-894A
Trials 1
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 1/2 | NCT07223632 | May 2025 → Mar 2026 overdue | Charcot-Marie-Tooth disease | Vanda Pharmaceuticals | Active not recruiting | No outcome recorded |
News releases announcing trial results or a regulatory action · 5
| Date | Issuer | Release |
|---|---|---|
| 2026-07-07 | Vanda Pharmaceuticals | Regulatory Vanda Pharmaceuticals Announces FDA Rare Pediatric Disease Designation for Investigational Therapy for Charcot-Marie-Tooth Disease Type 2S vandapharma.com ↗
The designation was granted by the FDA's Office of Orphan Products Development and Office of Pediatric Therapeutics . |
| 2025-04-07 | Vanda Pharmaceuticals | Results Vanda Pharmaceuticals Announces Presentation at 2025 AAN Annual Meeting vandapharma.com ↗
The effects of VCA-894A were confirmed in Hesperos' Human-on-a-Chip ® neuromuscular junction model with patient-derived cells where VCA-894A demonstrated significant improvements in neuromuscular function, including reduced muscle fatigue and improved synaptic transmission between motor neurons and skeletal muscle myotubes. |
| 2025-02-24 | Vanda Pharmaceuticals | Results Vanda Pharmaceuticals announces the publication of an article titled "Potential ASO-based personalized treatment for Charcot-Marie-Tooth disease type 2S" vandapharma.com ↗
VCA-894A targets a patient-specific IGHMBP2 variant and restores expression levels of IGHMBP2, highlighting the exciting potential of this approach for personalized therapeutics for rare diseases. |
| 2024-01-23 | Vanda Pharmaceuticals | Regulatory Vanda Pharmaceuticals Receives FDA Approval to Proceed with Investigational New Drug VCA-894A, a Novel Antisense Oligonucleotide Candidate for the Treatment of Charcot-Marie-Tooth Disease, Type 2S vandapharma.com ↗ |
| 2023-06-01 | Vanda Pharmaceuticals | Regulatory Vanda Pharmaceuticals Announces Orphan Drug Designation Granted for VCA-894A, a Novel Antisense Oligonucleotide Candidate for the Treatment of Charcot-Marie-Tooth Disease, Type 2S vandapharma.com ↗
Vanda Pharmaceuticals Inc. (Vanda) (Nasdaq: VNDA) today announced the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation for VCA-894A for the treatment of Charcot-Marie-Tooth disease, axonal, type 2S (CMT2S), caused by cryptic splice site variants within IGHMBP2. |
All press releases naming this drug 6 releases
Evidence & citations 5 cited values
Every value below carries the sentence it was read from. 1 source stands behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | VCA-894A | ClinicalTrials.gov intervention name — accepted as the source's own label NCT07223632 ↗ |
| Action | Restore | “rescue and restore the activity of IGHMBP2” NCT07223632 ↗ |
| Modality | Antisense oligonucleotide | “an ASO designed to rescue and restore the activity of IGHMBP2” NCT07223632 ↗ |
| Route | Intrathecal | “administered by intrathecal injection” NCT07223632 ↗ |
| Target | IGHMBP2 | “restore the activity of IGHMBP2” NCT07223632 ↗ |