1
Orphan Designations
0
FDA Approvals
3
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| Alstrom syndrome | Immunotherapeutic vaccine consisting of pAc/emm55, a plasmid DNA (pDNA), which codes for a modified immunogenic, streptococcal Emm55 protein, complexed with in vivo-jetPEI® polymer-based reagent linear polyethylenimine) | Orphan Designation | - |
| Finnish type amyloidosis | Immunotherapeutic vaccine consisting of pAc/emm55, a plasmid DNA (pDNA), which codes for a modified immunogenic, streptococcal Emm55 protein, complexed with in vivo-jetPEI® polymer-based reagent linear polyethylenimine) | Orphan Designation | - |
| lysinuric protein intolerance | Immunotherapeutic vaccine consisting of pAc/emm55, a plasmid DNA (pDNA), which codes for a modified immunogenic, streptococcal Emm55 protein, complexed with in vivo-jetPEI® polymer-based reagent linear polyethylenimine) | Orphan Designation | - |