drug · oligonucleotide

AMONDYS 45

updated 3mo ago

by $SRPT Sarepta Therapeutics, Inc.🔗 ChEMBL

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Oligonucleotide drug with a maximum clinical stage of Approval (across all indications), with an approval for duchenne muscular dystrophy and muscular dystrophy.

targets

Dystrophin pre-mRNA

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nothing on the calendar yet

Trials studying AMONDYS 45

—A Long-term Observational Study Evaluating Eteplirsen, Golodirsen, or Casimersen in Routine Clinical Practiceenrolling by invitationn=300
phase3An Extension Study to Evaluate Casimersen or Golodirsen in Patients With Duchenne Muscular Dystrophyterminatedn=171
phase2A 48-Week, Open Label, Study to Evaluate the Efficacy and Safety of AMONDYS 45, EXONDYS 51, VYONDYS 53 in Subjects With DuchenneMuscular Dystrophy Carrying Eligible DMD Duplications.completedn=3

News(5)

Capricor CEO won’t rule out legal action against FDA after negative adcomm
After an FDA advisory committee voted 9-3 on Wednesday against recommending Capricor Therapeutics’ Duchenne muscular dystrophy cardiomyopathy drug for approval, stakeholders allege an “ulterior mot…
biospace · 14d ago
Capricor and Replimune prep for FDA adcomms, Q2 earnings roll on, Sarepta’s new leader, more
The FDA published briefing documents ahead of advisory committee meetings for therapies from Capricor Therapeutics and Replimune indicate an uphill battle for the biotechs; GSK and AstraZeneca outl…
biospace · 16d ago
Sarepta names former AbbVie R&D exec Michael Severino as next CEO
After leaving Tessera Therapeutics earlier this month, Michael Severino will take over Sarepta Therapeutics as the biotech awaits two key regulatory decisions in Duchenne muscular dystrophy.
biospace · 18d ago
Sarepta, in search of a turnaround, taps ex-AbbVie exec Severino as CEO
Most recently the leader of buzzy startup Tessera Therapeutics, Severino will look to improve the fortunes of a company facing emerging competition and declining sales for its prized gene therapy E…
biopharma_dive · 18d ago
Dyne gets early 2027 decision date for Duchenne therapy, analysts expect ‘smooth’ review
If approved, Dyne Therapeutics’ zeleciment rostudirsen could “capture the majority” of the exon-51 Duchenne muscular dystrophy market, given its better efficacy and dosing profile versus Sarepta Th…
biospace · 24d ago

brand

AMONDYS 45

trade names

Amondys 45

generic

CASIMERSEN

MOA

12.1 Mechanism of Action Casimersen is designed to bind to exon 45 of dystrophin pre-mRNA resulting in exclusion of this exon during mRNA processing in patients with genetic mutations that are amenable to exon 45 skipping. Exon 45 skipping is intended to allow for production of an internally truncated dystrophin protein in patients with genetic mutations that are amenable to exon 45 skipping [see Clinical Studies ( 14 )] .

indication

1 INDICATIONS AND USAGE AMONDYS 45 is indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene that is amenable to exon 45 skipping. This indication is approved under accelerated approval based on an increase in dystrophin production in skeletal muscle observed in patients treated with AMONDYS 45 [see Clinical Studies ( 14 )] . Continued approval for this indication may be contingent upon verification of a clinical benefit in confirmatory trials. AMONDYS 45 is an antisense oligonucleotide indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene that is amenable to exon 45 skipping. This indication is approved under accelerated approval based on an increase in dystrophin production in skeletal muscle observed in patients treated with AMONDYS 45 [see Clinical Studies ( 14 )] . Continued approval for this indication may be contingent upon verification of a clinical benefit in confirmatory trials. ( 1 )

Data sources

ClinicalTrials.gov · OpenTargets

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News(5)

Capricor CEO won’t rule out legal action against FDA after negative adcomm

After an FDA advisory committee voted 9-3 on Wednesday against recommending Capricor Therapeutics’ Duchenne muscular dystrophy cardiomyopathy drug for approval, stakeholders allege an “ulterior mot…

biospace · 14d ago

Capricor and Replimune prep for FDA adcomms, Q2 earnings roll on, Sarepta’s new leader, more

The FDA published briefing documents ahead of advisory committee meetings for therapies from Capricor Therapeutics and Replimune indicate an uphill battle for the biotechs; GSK and AstraZeneca outl…

biospace · 16d ago

Sarepta names former AbbVie R&D exec Michael Severino as next CEO

After leaving Tessera Therapeutics earlier this month, Michael Severino will take over Sarepta Therapeutics as the biotech awaits two key regulatory decisions in Duchenne muscular dystrophy.

biospace · 18d ago

Sarepta, in search of a turnaround, taps ex-AbbVie exec Severino as CEO

Most recently the leader of buzzy startup Tessera Therapeutics, Severino will look to improve the fortunes of a company facing emerging competition and declining sales for its prized gene therapy E…

biopharma_dive · 18d ago

Dyne gets early 2027 decision date for Duchenne therapy, analysts expect ‘smooth’ review

If approved, Dyne Therapeutics’ zeleciment rostudirsen could “capture the majority” of the exon-51 Duchenne muscular dystrophy market, given its better efficacy and dosing profile versus Sarepta Th…

biospace · 24d ago

AMONDYS 45

🐶 often sniffed alongside

targets

all catalysts(0)

Trials studying AMONDYS 45

News(5)

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AMONDYS 45

🐶 often sniffed alongside

targets

all catalysts(0)

Trials studying AMONDYS 45

News(5)

Report a data issue

comments(0)