| Gen name | trade name | catogry |
| lonafarnib | Zokinvy | Miscellaneous metabolic agents |
| miglustat | Zavesca | Miscellaneous metabolic agents |
| uridine | Xuriden | Miscellaneous metabolic agents |
| uridine | Vistogard | Miscellaneous metabolic agents |
| inotersen | Tegsedi | Miscellaneous metabolic agents |
| asfotase alfa | Strensiq | Miscellaneous metabolic agents |
| mitapivat | Pyrukynd | Miscellaneous metabolic agents |
| pegvaliase | Palynziq | Miscellaneous metabolic agents |
| lumasiran | Oxlumo | Miscellaneous metabolic agents |
| nitisinone | Orfadin | Miscellaneous metabolic agents |
| patisiran | Onpattro | Miscellaneous metabolic agents |
| fosdenopterin | Nulibry | Miscellaneous metabolic agents |
| nitisinone | Nityr | Miscellaneous metabolic agents |
| metreleptin | Myalept | Miscellaneous metabolic agents |
| sapropterin | Kuvan | Miscellaneous metabolic agents |
| givosiran | Givlaari | Miscellaneous metabolic agents |
| migalastat | Galafold | Miscellaneous metabolic agents |
| burosumab | Crysvita | Miscellaneous metabolic agents |
| eliglustat | Cerdelga | Miscellaneous metabolic agents |