6
Orphan Designations
0
FDA Approvals
17
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| Fabry disease | a recombinant adeno-associated virus (AAV) vector that contains a bioengineered capsid (AAV-Spark100) and a codonoptimized expression cassette to drive expression of a secretable form of alpha-galactosidase A (alpha-Gal A) | Orphan Designation | - |
| Leber congenital amaurosis | Recombinant adeno-associated virus retinal pigment epithelium gene vector AAV2-hRPE65v2 | Orphan Designation | - |
| Leber congenital amaurosis 5 | Recombinant adeno-associated virus retinal pigment epithelium gene vector AAV2-hRPE65v2 | Orphan Designation | - |
| Leber congenital amaurosis 8 | Recombinant adeno-associated virus retinal pigment epithelium gene vector AAV2-hRPE65v2 | Orphan Designation | - |
| PMM2-congenital disorder of glycosylation | LUXTURNA | Orphan Designation | - |
| adult neuronal ceroid lipofuscinosis | adeno-associated viral serotype 2 vector under the regulatory control of a CMV promoter encoding the human tripeptidyl peptidase-1 (hTPP1) cDNA | Orphan Designation | - |
| autosomal recessive inherited pseudoxanthoma elasticum | LUXTURNA | Orphan Designation | - |
| carbon monoxide-induced delayed encephalopathy | LUXTURNA | Orphan Designation | - |
| hemophilia A | recombinant adeno-associated viral (AAV) vector that contains a bioengineered capsid (AAV-Spark200) and a codon-optimized expression cassette to drive expression of the SQ form of a B-domain deleted human coagulation factor VIII (hFVIII) | Orphan Designation | - |
| idiopathic interstitial pneumonia | immunomodulatory inhibitor of high mobility group box 1 | Orphan Designation | - |
| idiopathic pulmonary fibrosis | immunomodulatory inhibitor of high mobility group box 1 | Orphan Designation | - |
| inherited retinal dystrophy | LUXTURNA | Orphan Designation | - |
| interstitial lung disease 2 | immunomodulatory inhibitor of high mobility group box 1 | Orphan Designation | - |
| maternally-inherited diabetes and deafness | LUXTURNA | Orphan Designation | - |
| neuronal ceroid lipofuscinosis 1 | adeno-associated viral serotype 2 vector under the regulatory control of a CMV promoter encoding the human tripeptidyl peptidase-1 (hTPP1) cDNA | Orphan Designation | - |
| neuronal ceroid lipofuscinosis 2 | adeno-associated viral serotype 2 vector under the regulatory control of a CMV promoter encoding the human tripeptidyl peptidase-1 (hTPP1) cDNA | Orphan Designation | - |
| retinal dystrophies primarily involving Bruch's membrane | LUXTURNA | Orphan Designation | - |