| NAME |
ORIGINATOR |
INDICATION |
DEVELOPMENT STATUS |
| Carbaglu® |
Orphan Europe
(Recordati) |
NAGS deficiency |
Approved in the EU
Approved in the USA |
| Carbaglu® |
Orphan Europe
(Recordati) |
Organic acidaemias |
Pre-registration in the EU |
| Cystagon® |
Mylan |
Other indication unrelated to nephropathic
cystinosis |
Phase II / III |
| Cystadrops® |
Orphan Europe
(Recordati) |
Ocular cystinosis |
Phase II |
| Normosang® |
Orphan Europe
(Recordati) |
Hepatic porphyria |
Approved in the EU
Pre-registration in the USA |
