.Pharmacolibrary.Drugs.M_MusculoSkeletalSystem.M09A_OtherDrugsForDisordersOfTheMusculoSkeletalSystem.M09AX15_DelandistrogeneMoxeparvo.DelandistrogeneMoxeparvo .Pharmacolibrary.Drugs.M_MusculoSkeletalSystem.M09A_OtherDrugsForDisordersOfTheMusculoSkeletalSystem.M09AX15_DelandistrogeneMoxeparvo.DelandistrogeneMoxeparvo| name: | DelandistrogeneMoxeparvovec |
| ATC code: | M09AX15 | route: | intravenous |
| n-compartments | 1 |
Delandistrogene moxeparvovec is a gene therapy consisting of a recombinant adeno-associated virus vector (AAVrh74) encoding a micro-dystrophin gene, designed as a disease-modifying treatment for Duchenne Muscular Dystrophy (DMD) in pediatric patients. As of 2024, it is approved by the US FDA for certain ambulatory children with DMD aged 4-5 years.
No published pharmacokinetic data describing PK parameters (such as volume of distribution, clearance, or bioavailability) are available in peer-reviewed literature or regulatory documents for delandistrogene moxeparvovec (SRP-9001). The product is an intravenous gene therapy, and typical small-molecule PK concepts are not directly applicable.