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Kyndrisa, drisapersen (GSK2402968, PRO051)

Also known as: 2402968

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Company BioMarin Pharmaceutical Inc.
DescriptionAntisense oligoribonucleotide that induces exon 51 skipping on the dystrophin gene
Molecular Target
Mechanism of Action 
Therapeutic ModalityNucleic acid: Linear RNA: Antisense
Latest Stage of DevelopmentRegistration
Standard IndicationMuscular dystrophy
Indication DetailsTreat Duchenne muscular dystrophy (DMD)
Regulatory Designation U.S. - Breakthrough Therapy (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. - Rare Pediatric Disease (Treat Duchenne muscular dystrophy (DMD));
EU - Orphan Drug (Treat Duchenne muscular dystrophy (DMD));
Japan - Orphan Drug (Treat Duchenne muscular dystrophy (DMD));
Australia - Orphan Drug (Treat Duchenne muscular dystrophy (DMD))
Partner

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Total Number of DealsTotal Deal ValueTotal Upfront CashTotal Milestone Payments

2

$1,514.2M

$680.0M

$160.0M


 Deals Details
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