NAV

Delandistrogene moxeparvovec

Identification

Summary

Delandistrogene moxeparvovec is a gene therapy used to treat Duchenne Muscular Dystrophy in ambulatory children with DMD gene mutation.

Brand Names
Elevidys
Generic Name
Delandistrogene moxeparvovec
DrugBank Accession Number
DB16802
Background

Delandistrogene moxeparvovec is an adeno-associated virus vector-based gene therapy developed by Sarepta Therapeutics. It was granted accelerated approval by the FDA on June 22, 2023, as the first gene therapy to treat Duchenne Muscular Dystrophy (DMD).5 DMD is an X-linked genetic disorder characterized by mutations in the dystrophin gene, leading to a deficiency in functional dystrophin protein. Dystrophin is an essential protein responsible for muscle function; thus, patients with DMD experience a progressive deterioration of muscle mass and function.1 DMD tends to be more prevalent in males.2

Delandistrogene moxeparvovec comprises a non-replicating, recombinant adeno-associated virus serotype rh74 (AAVrh74) based vector containing the microdystrophin transgene under the control of a muscle-specific promoter (MHCK7) to enhance expression in cardiac and skeletal muscles. Microdystrophin is a truncated, functional form of dystrophin.1,4

Type
Biotech
Groups
Approved, Investigational
Biologic Classification
Gene Therapies
Other gene therapies
Synonyms
  • Delandistrogene moxeparvovec
  • rAAVrh74.MHCK7.micro-dystrophin
External IDs
  • RG 6356
  • RG-6356
  • SRP 9001
  • SRP-9001
Poor quality drug data slowing you down?
Unlock 38% more drug discovery time and eliminate decision-making doubts with this one-stop guide to quality drug data.
Download eBook
Poor quality drug data slowing you down?
Download eBook

Pharmacology

Indication

Under accelerated approval, delandistrogene moxeparvovec is indicated for the treatment of ambulatory pediatric patients four to five years old with Duchenne muscular dystrophy (DMD) with a confirmed mutation in the DMD gene.4

This indication is approved under accelerated approval based on the expression of delandistrogene moxeparvovec microdystrophin in skeletal muscle observed in patients treated with this therapy. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s).4

Reduce drug development failure rates
Build, train, & validate machine-learning models
with evidence-based and structured datasets.
See how
Build, train, & validate predictive machine-learning models with structured datasets.
See how
Associated Conditions
Indication TypeIndicationCombined Product DetailsApproval LevelAge GroupPatient CharacteristicsDose Form
Treatment ofDuchenne muscular dystrophy (dmd)•••••••••••••••••••••••• •••• ••••••••• ••••••••••
Contraindications & Blackbox Warnings
Prevent Adverse Drug Events Today
Tap into our Clinical API for life-saving information on contraindications & blackbox warnings, population restrictions, harmful risks, & more.
Learn more
Avoid life-threatening adverse drug events with our Clinical API
Learn more
Pharmacodynamics

Delandistrogene moxeparvovec delivers the shortened functional dystrophin protein in skeletal and cardiac muscles, promoting muscle function and delaying disease progression.2 In one study, treatment of male children with DMD with delandistrogene moxeparvovec was associated with robust expression of microdystrophin protein at 12 weeks of treatment, with the mean expression levels ranging from 51.7% to 95.7%.1,4

Mechanism of action

DMD is a progressive, fatal X-linked recessive disease caused by mutations in the DMD gene that encodes dystrophin,3 an essential protein involved in muscle integrity and function. As a component of the dystrophin-glycoprotein complex (DGC), which absorbs shock and maintains muscle integrity during normal muscle contraction,2 dystrophin links the intracellular cytoskeleton network of muscle fibre cells to the sarcolemma.1,2 A lack of functional dystrophin protein results in the failure of DGC assembly, muscle inflammation and damage, impaired muscle fibre regeneration, and progressive and irreversible deterioration of muscle function and mass.1,2

Delandistrogene moxeparvovec carries a transgene encoding microdystrophin. Microdystrophin is a shortened form of dystrophin and contains selected domains of dystrophin expressed in normal muscle cells. Microdystrophin delivered by delandistrogene moxeparvovec has been demonstrated to localize to the sarcolemma.4

Absorption

No information is available.

Volume of distribution

There is limited information. In mice toxicology studies, vector DNA was detected in all major organs with the highest quantities detected in the liver, followed by lower levels in the heart, adrenal glands, skeletal muscle, and aorta following delandistrogene moxeparvovec administration. Low levels of delandistrogene moxeparvovec were also detected in the spinal cord, sciatic nerve and testis.4

Protein expression of microdystrophin was highest in the cardiac tissue, exceeding physiologic dystrophin expression levels in healthy mice, with lower levels in the skeletal muscle and diaphragm. In some studies, microdystrophin was also detected at low levels in the liver.4

Protein binding

No information is available.

Metabolism

No information is available.

Route of elimination

No information is available.

Half-life

No information is available.

Clearance

No information is available.

Adverse Effects
Improve decision support & research outcomes
With structured adverse effects data, including: blackbox warnings, adverse reactions, warning & precautions, & incidence rates. View sample adverse effects data in our new Data Library!
See the data
Improve decision support & research outcomes with our structured adverse effects data.
See a data sample
Toxicity

No information is available regarding the acute toxicity (LD50) and overdosage of delandistrogene moxeparvovec.

Pathways
Not Available
Pharmacogenomic Effects/ADRs
Not Available

Interactions

Drug Interactions
This information should not be interpreted without the help of a healthcare provider. If you believe you are experiencing an interaction, contact a healthcare provider immediately. The absence of an interaction does not necessarily mean no interactions exist.
Not Available
Food Interactions
No interactions found.

Products

Drug product information from 10+ global regions
Our datasets provide approved product information including:
dosage, form, labeller, route of administration, and marketing period.
Access now
Access drug product information from over 10 global regions.
Access now
Mixture Products
NameIngredientsDosageRouteLabellerMarketing StartMarketing EndRegionImage
ElevidysDelandistrogene moxeparvovec (13300000000000 {GC}/1mL) + Isopropyl alcohol (0.75 g/1)KitIntravenousSarepta Therapeutics, Inc.2023-06-22Not applicableUS flag
ElevidysDelandistrogene moxeparvovec (13300000000000 {GC}/1mL) + Isopropyl alcohol (0.75 g/1)KitIntravenousSarepta Therapeutics, Inc.2023-06-22Not applicableUS flag
ElevidysDelandistrogene moxeparvovec (13300000000000 {GC}/1mL) + Isopropyl alcohol (0.75 g/1)KitIntravenousSarepta Therapeutics, Inc.2023-06-22Not applicableUS flag
ElevidysDelandistrogene moxeparvovec (13300000000000 {GC}/1mL) + Isopropyl alcohol (0.75 g/1)KitIntravenousSarepta Therapeutics, Inc.2023-06-22Not applicableUS flag
ElevidysDelandistrogene moxeparvovec (13300000000000 {GC}/1mL) + Isopropyl alcohol (0.75 g/1)KitIntravenousSarepta Therapeutics, Inc.2023-06-22Not applicableUS flag

Categories

Drug Categories
Classification
Not classified
Affected organisms
  • Humans and other mammals

Chemical Identifiers

UNII
2P6QV2ZE52
CAS number
2305040-16-6

References

General References
  1. Deng J, Zhang J, Shi K, Liu Z: Drug development progress in duchenne muscular dystrophy. Front Pharmacol. 2022 Jul 22;13:950651. doi: 10.3389/fphar.2022.950651. eCollection 2022. [Article]
  2. Klimchak AC, Sedita LE, Rodino-Klapac LR, Mendell JR, McDonald CM, Gooch KL, Malone DC: Assessing the value of delandistrogene moxeparvovec (SRP-9001) gene therapy in patients with Duchenne muscular dystrophy in the United States. J Mark Access Health Policy. 2023 May 26;11(1):2216518. doi: 10.1080/20016689.2023.2216518. eCollection 2023. [Article]
  3. Wilton-Clark H, Yokota T: Antisense and Gene Therapy Options for Duchenne Muscular Dystrophy Arising from Mutations in the N-Terminal Hotspot. Genes (Basel). 2022 Jan 28;13(2):257. doi: 10.3390/genes13020257. [Article]
  4. FDA Approved Drug Products: ELEVIDYS (delandistrogene moxeparvovec-rokl) suspension, for intravenous infusion [Link]
  5. Business Wire: Sarepta Therapeutics Announces FDA Approval of ELEVIDYS, the First Gene Therapy to Treat Duchenne Muscular Dystrophy [Link]
RxNav
2641675
Wikipedia
Delandistrogene_moxeparvovec

Clinical Trials

Clinical Trials
PhaseStatusPurposeConditionsCount
1Active Not RecruitingTreatmentDuchenne Muscular Dystrophy (DMD)1

Pharmacoeconomics

Manufacturers
Not Available
Packagers
Not Available
Dosage Forms
FormRouteStrength
KitIntravenous
Prices
Not Available
Patents
Not Available

Properties

State
Liquid
Experimental Properties
Not Available

Drug created at May 10, 2022 21:18 / Updated at July 23, 2023 12:09