Dystrophic Epidermolysis Bullosa Approved Therapies & Global Pipeline

Last Update: Aug 4, 2023

Approved Therapies

Company Candidate MOA Indication Route Approval
Krystal BiotechVyjuvek (B-VEC)Modified replication-incompetent HSV-1 expressing Type VII Collagen (COL7)Treatment of wounds in patients aged 6 mths and older with DEB with confirmed mutation/s in the COL7A1 geneTopicalMay 2023 (FDA)
Chiesi Farmaceutici (Amryt)Filsuvez (episalvan)Birch bark extractTreatment of wounds in patients aged 6 mths and older with EB (DEB/JEB)TopicalJune 2022 (EU)

Global Pipeline

Company Candidate MOA Indication Route Phase
Abeona TherapeuticsEB-101Autologous ex-vivo COL7A1 gene-corrected cell therapy (transplant)RDEB large non-healing chronic woundsSurgical graftBLA
Castle Creek (Fibrocell)FCX-007 (D-Fi)Ex-vivo autologous cell-based lentiviral vector COL7A1 gene therapyRDEBIntradermal injectionIII
InMed PharmaceuticalsINM-755Cannabinol formulationEB, all typesTopicalII
Relief TherapeuticsRLF-TD011 (Nexodyn)Hypochlorous acid formulation/Antibacterial EB, all typesSprayII
Quoin PharmaceuticalsQRX004Read-through of nonsense mutationsRDEBTopicalII
Phoenicis TherapeuticsPTW-002Anti-sense oligonucleotide exon skippingDDEB and RDEBTopicalII
ShionogiRedasemtidePeptide analogue of HMGB1, stem cell modulationDEBIntravenousII
Aegle TherapeuticsAGLE 102Allogeneic extracellular vesicles from MSCsDEBTopicalI/II
Eloxx PharmaceuticalsZKN-013Ribosome modulating agent for non-sense readthroughRDEB, JEBOralIND-C
Amryt PharmaAP103Corrected collagen VII gene delivered using HPAE as non-viral vectorDEBTopicalPreclin
Constant TherapeuticsTXA127Angiotensin-(1-7)EBOralPreclin
Branca BunusBrB-101CRISPR-Cas9 to remove COL7A1 mutations in exon80RDEB exon 80-Preclin
Telaria (Replay Bio)-synHSV (HSV-1 vector)DEB-Disc?