API | onasemnogene abeparvovec

onasemnogene abeparvovec (Zolgensma) Report issue

Gene therapy Orphan Drug ^FDA Approved ^FDA Breakthrough Therapy ^FDA

AACT

Active Ingredient History

NOW

Onasemnogene abeparvovec, sold under the brand name Zolgensma, is a gene therapy medication used to treat spinal muscular atrophy (SMA). It is used as a one-time infusion into a vein. ^Wikipedia

Chemistry

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More Chemistry

Pharmacology

Mechanism of Action:
- Survival motor neuron protein (Homo sapiens) ^UniProt
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avxs-101

Organizations (6)

Research & Development (6)

Organization	Org Type	FDA approvals	Clinical Trials involvement	Org ID	Force Sort

Marketing (1)

Organization	Org Type	FDA approvals	Clinical Trials involvement	Org ID	Force Sort

Indications (4)

Approved Indications (1)

Spinal Muscular Atrophies of Childhood (approved 2019)

Experimental Indications - Clinical Trial Phases 1-4 (4)

Genetic Therapy (Phase 3)

Muscular Atrophy, Spinal (Phase 4)

Spinal Diseases (Phase 3)

Spinal Muscular Atrophies of Childhood (Phase 4)

Overview

Highest Phase: Phase 4
# of Trials: 9
# of Trial Organizations: 6

Trials by Phase

Trial	Phase	Start Date	Organizations	Indications

Feedback

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