Drug Profile
Delandistrogene moxeparvovec - Roche/Sarepta Therapeutics
Alternative Names: Delandistrogene moxeparvovec-rokl; Delandistrogenum moxeparvovecum - Roche/Sarepta Therapeutics; ELEVIDYS; rAAVrh74.MHCK7.micro-dystrophin; RG 6356; RO-7494222; SRP-9001Latest Information Update: 14 Mar 2024
Price :
$50
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At a glance
- Originator Nationwide Children's Hospital
- Developer Nationwide Children's Hospital; Roche; Sarepta Therapeutics
- Class Gene therapies; Morpholines
- Mechanism of Action Dystrophin replacements; Gene transference
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Orphan Drug Status
Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.
- New Molecular Entity No
Highest Development Phases
- Marketed Duchenne muscular dystrophy
Most Recent Events
- 08 Mar 2024 Sarepta Therapeutics suspends phase I trial in Duchenne muscular dystrophy (In children, In infants) in Spain (NCT06241950, EudraCT2022-003407-15)
- 16 Feb 2024 The US FDA assigns review goal date of (21/06/2024) for the priority review of efficacy supplement of BLA for delandistrogene moxeparvovec for Duchenne muscular dystrophy
- 16 Feb 2024 The US FDA grants a priority review for the efficacy supplement of a BLA for label expansion of delandistrogene moxeparvovec in Duchenne muscular dystrophy and to convert accelerated approval to traditional approval