Amondys 45

(Casimersen)

ApprovedRNA therapy

Amondys 45 is a rna therapy that works as a Exon 45 Skipping ASO.[2] It is being used across 1 indication, with its most advanced program in Approved.[1]

Amondys 45 is one of 12 aso programs targeting Spinal Muscular Atrophy. It is currently in Approved.

Indications

Total Trials

Active Trials

Competitors

FDA Applications

NDA/BLA/ANDA regulatory filings

Patents

Pipeline

Status by Indication[3][1]

Indication

Spinal Muscular AtrophyApproved

Approved

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

2 5

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

NMD670

NNMD Pharma A/S

Phase 2

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

Clinical

Trials by Indication[1]

Observational Study1 trial

A Long-term Observational Study Evaluating Eteplirsen, Golodirsen, or Casimersen in Routine Clinical Practice

Enrolling by invitation· Sarepta Therapeutics logo

Sarepta Therapeutics··NCT06606340·300 enrolled

Duchenne Muscular Dystrophy1 trial

An Extension Study to Evaluate Casimersen or Golodirsen in Patients With Duchenne Muscular Dystrophy

Terminated· Sarepta Therapeutics logo

Sarepta Therapeutics·Phase 3·NCT03532542·171 enrolled

Regulatory

FDA Approval Timeline[4]

Feb 25, 2021

AMONDYS 45

NDA NDA213026

Feb 25, 2021

AMONDYS 45

NDA NDA213026

Mar 23, 2023

AMONDYS 45

NDA NDA213026

Jul 11, 2024

AMONDYS 45

NDA NDA213026

Jan 6, 2026

AMONDYS 45

NDA NDA213026

Sources

References

[1]
ClinicalTrials.gov — 2 trial records for Casimersen. U.S. National Library of Medicine, National Institutes of Health.
[2]
ChEMBL — Casimersen compound record. European Bioinformatics Institute (EMBL-EBI).
[3]
Open Targets Platform — Drug–disease associations for Casimersen. EMBL-EBI & Wellcome Sanger Institute.
[4]
OpenFDA — FDA approval records (NDA NDA213026). U.S. Food & Drug Administration.
[5]
FDA Orange Book — Patent and exclusivity data for Amondys 45. U.S. Food & Drug Administration.
[6]
SEC EDGAR — Sarepta Therapeutics company filings. U.S. Securities and Exchange Commission.

Details

Drug Info

Quick Facts

Mechanism of Action: Exon 45 Skipping ASO[2]
Modality: RNA therapy[2]
Highest Phase: Approved[1]
Company: Sarepta Therapeutics

Patent Landscape[5]

Patent Cliff

Feb 16, 2029

Earliest patent expiration — generic entry possible after this date

Patent #Expires

RE48960Feb 16, 2029

9758783Nov 12, 2030

10781450Nov 12, 2030

10287586Nov 12, 2030

9228187Nov 12, 2030

Amondys 45

Status by Indication[3][1]

Competitive Landscape

Trials by Indication[1]

Observational Study1 trial

Duchenne Muscular Dystrophy1 trial

FDA Approval Timeline[4]

References

Drug Info

Quick Facts

Patent Landscape[5]

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