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onasemnogene abeparvovec · 1 trial · 1 indication
An SAE is defined as any adverse event \[appearance of (or worsening of any pre-existing)\] undesirable sign(s), symptom(s), or medical conditions(s) which meets any one of the following criteria: * fatal * life-threatening * results in persistent or significant disability/incapacity * constitutes a congenital anomaly/birth defect, fetal death or congenital abnormality or birth defect * requires in-patient hospitalization or prolongation of existing hospitalization, unless hospitalization is for routine treatment or monitoring of the studied indication, not associated with any deterioration in condition * is medically significant, e.g. defined as an event that jeopardizes the participant or may require medical or surgical intervention to prevent one of the outcomes listed above
The following are important identified and important potential risks (AESI) associated with OAV101: Hepatotoxicity, Transient Thrombocytopenia, Cardiac adverse events, Sensory abnormalities suggestive of ganglionopathy, and Thrombotic microangiopathy. These will be assessed by the investigator.
| Arm | Type | Description |
|---|---|---|
| Intravenous (IV) & Intrathecal (IT) Onasemnogene Abeparvovec | EXPERIMENTAL | Patients who received OAV101 IT or OAV101 IV in clinical trials (COAV101A12306, COAV101B12301 and COAV101B12302) |
| Name | Type | Description |
|---|---|---|
| onasemnogene abeparvovec | BIOLOGICAL | Onasemnogene abeparvovec is a non-replicating recombinant adeno-associated virus serotype 9 containing the human survival motor neuron gene under the control of the ytomegalovirus enhancer/chicken β-actin-hybrid promoter. Onasemnogene abeparvovec is administered as a one-time intravenous (IV) infusion or intrathecal (IT) injection. Dosage determined by participant weight. |
Inclusion Criteria: 1. Participated in an OAV101 clinical trial. 2. Written informed consent must be obtained before any assessment is performed. 3. Patient/Parent/legal guardian willing and able to comply with study procedures. Exclusion Criteria: There are no exclusion criteria for this study.
| 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 |
Onasemnogene abeparvovec is used for the treatment of Spinal Muscular Atrophy (SMA), a genetic neuromuscular disorder. It is currently in Phase 3 clinical development and is being studied in a long-term follow-up trial to assess outcomes in patients who previously received the treatment in clinical trials.
Onasemnogene abeparvovec targets the SMN1 and SMN2 genes, which are involved in the production of the survival motor neuron protein. It is an exogenous gene therapy designed to address the genetic cause of Spinal Muscular Atrophy by delivering a functional copy of the SMN1 gene.
Onasemnogene abeparvovec is developed by Novartis AG, a global healthcare company traded on the New York Stock Exchange under the ticker symbol NVS. The drug is being evaluated for the treatment of Spinal Muscular Atrophy in an ongoing Phase 3 clinical trial.
Onasemnogene abeparvovec is in Phase 3 clinical development. It is an investigational therapy for Spinal Muscular Atrophy and is not yet approved by regulatory authorities. The current Phase 3 trial is a long-term follow-up study of patients who received the treatment in earlier clinical trials.
Onasemnogene abeparvovec is being studied in the clinical trial NCT05335876, titled "Long-term Follow-up of Patients With Spinal Muscular Atrophy Treated With OAV101 in Clinical Trials." This Phase 3 trial is active but not recruiting, with an enrollment of 20 participants across multiple countries including the United States, Australia, and Japan.