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onasemnogene abeparvovec

Phase 3

Spinal Muscular Atrophy (SMA) | Monoclonal antibody | Neurology |Novartis AG|Last Updated: Jan 20, 2026

Success Probability
Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
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Market & Valuation
rNPV $3.2B
Market Size $9.4B
Revenue Basis $1.6B
Competitors 6
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Trial Design
UNCONTROLLEDDMC
Total Trials1
Total Enrollment175
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT05335876Long-term Follow-up of Patients With Spinal Muscular Atrophy Treated With OAV101 in Clinical TrialsPHASE3 RECRUITING 175Dec 19, 2022Feb 27, 2031Jan 20, 202632 United States, Australia +17
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Study Endpoints
Primary Endpoints
Number of participants with treatment-emergent serious adverse events (SAEs)
Up to Year 5

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

Number of participants with treatment emergent Adverse Events of Special Interest (AESI)
Up to Year 5

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.

Secondary Endpoints
The number of participants demonstrating each developmental milestone according to the Developmental Milestone Checklist
Up to Year 5
The number of participants demonstrating maintenance of each developmental milestone according to the Developmental Milestone Checklist
Up to Year 5
Change from Baseline in the Hammersmith Functional Motor Scale - Expanded (HFMSE) total score
Up to Year 5
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Study Design & Arms
AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
Intravenous (IV) & Intrathecal (IT) Onasemnogene AbeparvovecEXPERIMENTALPatients who received OAV101 IT or OAV101 IV in clinical trials (COAV101A12306, COAV101B12301 and COAV101B12302)
Interventions
NameTypeDescription
onasemnogene abeparvovecBIOLOGICALOnasemnogene 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.
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Eligibility Criteria
Age Range0 Years — 100 Years
SexALL
Healthy VolunteersNo
Study Sites32

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.

Countries:United StatesAustraliaBelgiumBrazilCanadaChinaDenmarkFranceItalyJapanMalaysiaNetherlandsSaudi ArabiaSingaporeSpainTaiwanThailandUnited KingdomVietnam
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Recent Changes (Last 90 Days)
LOWMay 26, 2026NCT05335876primaryCompletionDate: changed
LOWMay 24, 2026NCT05335876studyFirstPostDate: changed