Phase IIIb, Open-label, Multi-center Study to Evaluate Safety, Tolerability and Efficacy of OAV101 Administered Intrathecally to Participants With SMA Who Discontinued Treatment With Nusinersen or Risdiplam

NCTID	NCT05386680 (View at clinicaltrials.gov)
Description	This was a Phase IIIb open-label, single arm, multi-center study to evaluate the safety, tolerability and efficacy of OAV101B in participants with SMA aged 2 to \<18 years after the discontinuation of treatment with nusinersen or risdiplam. The study aimed to enroll approximately 28 participants across each of 2 age brackets (2 to \<6 years, and 6 to \<18 years). (Show More)
Development Status	Approved
Indication	Spinal Muscular Atrophy
Disease Ontology Term	DOID:13137
Compound Name	ITVISMA
Compound Alias	onasemnogene abeparvovec, OAV101, AVXS-101
Sponsor	Novartis Pharmaceuticals
Funder Type	Industry
Recruitment Status	Completed
Enrollment Count	27 (ACTUAL)
Results Posted	View Results

Target Gene/Variant	SMN1
Therapy Type	Gene transfer
Therapy Route	In-vivo
Mechanism of Action	Functional gene replacement
Route of Administration	Intrathecal
Drug Product Type	Viral vector
Target Tissue/Cell
Delivery System	Viral transduction
Vector Type	AAV9
Editor Type
Dose 1	Phase 1/2: 6.0E13 vg, 1.2E14 vg, 2.4E14 vg
Dose 2	Phase 3: 1.2E14 vg
Dose 3	Approved Dose: 1.2E14 vg
Dose 4
Dose 5

No.of Trial Sites	13
Locations	Canada,Netherlands,Belgium,United States,Japan,Italy,Australia,France,Spain

Has US IND	True
FDA Designations	Breakthrough Therapy, Orphan Drug Designation
Recent Updates	Same drug product as Zolgensma (dosing and route of administration differs), FDA approved 11/24/25 for list price $2.59M