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Exondys 51, eteplirsen (AVI-4658)

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Company Sarepta Therapeutics Inc.
DescriptionPhosphorodiamidate morpholino oligomer (PMO) that induces skipping of exon 51 in dystrophin mRNA
Molecular Target DNA
Mechanism of Action 
Therapeutic ModalityNucleic acid: Linear RNA
Latest Stage of DevelopmentMarketed
Standard IndicationMuscular dystrophy
Indication DetailsTreat Duchenne muscular dystrophy (DMD); Treat Duchenne muscular dystrophy (DMD) amendable to exon 51 skipping
Regulatory Designation U.S. - Accelerated Approval (Treat Duchenne muscular dystrophy (DMD));
U.S. - Fast Track (Treat Duchenne muscular dystrophy (DMD));
U.S. - Orphan Drug (Treat Duchenne muscular dystrophy (DMD));
U.S. - Priority Review (Treat Duchenne muscular dystrophy (DMD));
U.S. - Rare Pediatric Disease (Treat Duchenne muscular dystrophy (DMD));
EU - Orphan Drug (Treat Duchenne muscular dystrophy (DMD))
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Total Number of DealsTotal Deal ValueTotal Upfront CashTotal Milestone Payments

1

0

0

0


 Deals Details
DateCompanies InvolvedDeal HeadlineTotal Deal ValueTotal Upfront CashTotal Milestone

09/29/2016

Undisclosed

Undisclosed

Undisclosed

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