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: 24 Jun 2024
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
- 20 Jun 2024 The US FDA grants traditional approval for delandistrogene moxeparvovec in Duchenne muscular dystrophy (In children, ambulatory individuals with a confirmed mutation in the DMD gene)
- 20 Jun 2024 The US FDA grants accelerated approval for delandistrogene moxeparvovec in Duchenne muscular dystrophy (In children, non-ambulatory with a confirmed mutation in the DMD gene)
- 20 Jun 2024 The US FDA approves delandistrogene moxeparvovec for label extension treatment of Duchenne muscular dystrophy (In children) in USA