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VCA-894A · 1 trial · 2 indications
Safety will be assessed by determining the incidence, severity, and dose relationship of adverse events that are related to treatment with VCA-894A.
The Revised Upper Limb Module for Spinal Muscular Atrophy (RULM) scores range from a minimum of 0 to a maximum of 37 points, with higher scores indicating better upper limb function.
The Hammersmith Functional Motor Scale - Expanded (HFMSE) scores range from a minimum of 0 to a maximum of 66 points, with higher scores indicating greater motor functioning.
| Arm | Type | Description |
|---|---|---|
| VCA-894A | EXPERIMENTAL | - |
| Name | Type | Description |
|---|---|---|
| VCA-894A | DRUG | intrathecal antisense oligonucleotide injection |
Inclusion Criteria: * Ability and acceptance to provide written informed consent. * Genetically confirmed diagnosis of CMT2S with confirmed IGHMBP2 intronic cryptic splice variant c. 1235+894C\>A. Exclusion Criteria: * Significant clinical deterioration of the patient's neurologic status, as judg...
VCA-894A is an investigational small molecule being developed for the treatment of Charcot-Marie-Tooth disease (CMT), a group of inherited neuromuscular disorders. It is specifically being studied in a Phase 1 clinical trial for CMT type 2S, an axonal form of the disease. The drug is in early clinical development and has not been approved by regulatory authorities.
VCA-894A is being developed by Vanda Pharmaceuticals Inc., a biopharmaceutical company traded on the NASDAQ under the ticker symbol VNDA. The company is conducting a Phase 1 clinical trial to evaluate the drug in patients with Charcot-Marie-Tooth disease type 2S.
VCA-894A is currently in Phase 1 clinical development. It is an investigational drug being studied for the treatment of Charcot-Marie-Tooth disease type 2S. The drug has received Orphan Drug and Rare Pediatric Disease designations from the FDA, but it is not yet approved for any use.
VCA-894A is being evaluated in a single Phase 1 clinical trial registered as NCT07223632, titled 'Treatment of Charcot-Marie-Tooth Disease, Axonal, Type 2S (CMT2S) in an Individual Patient.' The trial is active but not recruiting, and it is an uncontrolled, open-label study enrolling one female patient in the United States.
No, VCA-894A is not FDA approved. It is an investigational drug currently in Phase 1 clinical development for Charcot-Marie-Tooth disease type 2S. The FDA has granted it Orphan Drug and Rare Pediatric Disease designations, which provide certain development incentives, but the drug has not completed clinical trials or received marketing approval.
No alternative names for VCA-894A have been disclosed. It is a unique investigational small molecule developed by Vanda Pharmaceuticals. The drug is being studied specifically for the treatment of Charcot-Marie-Tooth disease type 2S, and no other names or aliases are associated with it in the available clinical trial information.