Zolgensma

Phase 3Small molecule

Novartis

Zolgensma is one of 13 small molecule programs targeting Spinal Muscular Atrophy. It is currently in Phase 3.

Indications

Total Trials

Active Trials

Competitors

Pipeline

Status by Indication[3][1]

Indication

Spinal Muscular AtrophyActive, not recruiting

Spinal Muscular AtrophyPhase 3

Active, not recruiting

Competition

Competitive Landscape

14 competitors in Spinal Muscular Atrophy

#DrugCompanyPhaseTrials

Spinraza

Biogen

Approved

18 2

Evrysdi

Roche

Approved

7 3

Prilosec

DDexcel Pharma Technologies Ltd.

Amondys 45

Onasemnogene Abeparvovec

Novartis

Phase 3

7 6

Salanersen

Biogen

Phase 3

1 7

Apitegromab

SScholar Rock, Inc.

Phase 3

4 8

Taldefgrobep Alfa

BBiohaven Therapeutics Ltd.

Phase 3

1 9

Vesemnogene Lantuparvovec

LLantu Biopharma

Phase 3

2 10

Zolgensma

Novartis

Phase 3

Ranked by pipeline maturity and clinical activity. Current drug highlighted in teal.

Clinical

Trials by Indication[1]

Spinal Muscular Atrophy1 trial

FIREFLY-2: Risdiplam in Spinal Muscular Atrophy

Active, not recruiting· Roche logo

Roche·Phase 3·NCT04174157·180 enrolled

Sources

References

[1]
ClinicalTrials.gov — 1 trial record for Zolgensma. U.S. National Library of Medicine, National Institutes of Health.
[2]
ChEMBL — Zolgensma compound record. European Bioinformatics Institute (EMBL-EBI).
[3]
Open Targets Platform — Drug–disease associations for Zolgensma. EMBL-EBI & Wellcome Sanger Institute.
[4]
SEC EDGAR — Novartis company filings. U.S. Securities and Exchange Commission.

Details

Drug Info

Quick Facts

Modality: Small molecule[2]
Highest Phase: Phase 3[1]
Company: Novartis

Zolgensma

Status by Indication[3][1]

Competitive Landscape

Trials by Indication[1]

Spinal Muscular Atrophy1 trial

References

Drug Info

Quick Facts

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