Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
AVXS-101 · 1 trial · 1 indication
| Arm | Type | Description |
|---|---|---|
| Cohort 1 | EXPERIMENTAL | 6.7 X 10\^13 vg/kg of AVXS-101 delivered one-time through a venous catheter inserted into a peripheral vein (n=3) |
| Cohort 2 | EXPERIMENTAL | 2.0 X 10\^14 vg/kg of AVXS-101 delivered one-time through a venous catheter inserted into a peripheral vein (n=12) |
| Name | Type | Description |
|---|---|---|
| AVXS-101 | BIOLOGICAL | Self-complementary AAV9 carrying the SMN gene under the control of a hybrid CMV enhancer/chicken-β-actin promoter |
Inclusion Criteria: * Six or nine months of age and younger (depending on cohort) on day of vector infusion with Type 1 SMA as defined by the following features: * Diagnosis of SMA based on gene mutation analysis with bi-allelic SMN1 mutations (deletion or point mutations) and 2 copies of SMN2. ...
| Company | Ticker | Trials | Lead Phase | Drugs |
|---|---|---|---|---|
| Biogen Inc. | BIIB | 13 | PHASE3 | Nusinersen |
| Novartis AG Sponsored ADR | NVS | 5 | PHASE3 | onasemnogene abeparvovec |
| Biohaven Ltd. | BHVN | 1 | PHASE3 | taldefgrobep alfa |
| Scholar Rock Holding Corp. | SRRK | 2 | PHASE3 | Apitegromab |
| argenx SE Sponsored ADR | ARGX | 1 | PHASE2 | ARGX-119 |
| Illumina, Inc. | ILMN | 1 | - | Undisclosed |
AVXS-101 is an investigational gene therapy being developed for the treatment of Spinal Muscular Atrophy 1, a severe genetic disorder affecting motor neurons. It is currently in clinical development and has not been approved by regulatory authorities.
AVXS-101 is developed by Novartis AG, a multinational pharmaceutical company listed on the New York Stock Exchange under the ticker symbol NVS. The drug is being studied for Spinal Muscular Atrophy 1.
AVXS-101 is in Phase 1 clinical development. It is an investigational drug, meaning it has not yet been approved by regulatory agencies. The Phase 1 trial for Spinal Muscular Atrophy 1 has been completed.
AVXS-101 has one completed clinical trial, identified as NCT02122952, titled 'Gene Transfer Clinical Trial for Spinal Muscular Atrophy Type 1'. This Phase 1 study enrolled 15 participants in the United States and evaluated the drug for Spinal Muscular Atrophy 1.
Yes, AVXS-101 is classified as a monoclonal antibody. It is being developed for the treatment of Spinal Muscular Atrophy 1, a neurological condition. The drug is currently in Phase 1 clinical development.