Category:Orphan drugs
Wikimedia Commons has media related to Orphan drugs.
Lists of all drugs that have received orphan status in the United States and Europe are available from the U.S. Food and Drug Administration and the European Commission, respectively:
- FDA List of Orphan Designations and Approvals
- European Commission Register of designated Orphan Medicinal Products
Pages in category "Orphan drugs"
The following 200 pages are in this category, out of approximately 472 total. This list may not reflect recent changes.
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- Baloxavir marboxil
- Bardoxolone methyl
- Belantamab mafodotin
- Belimumab
- Belumosudil
- Belzutifan
- Benralizumab
- Beremagene geperpavec
- Berotralstat
- Betibeglogene autotemcel
- Bevacizumab
- Binimetinib
- Birch bark
- Birch triterpenes
- Bortezomib
- Bosentan
- Brentuximab vedotin
- Brexucabtagene autoleucel
- Brigatinib
- Brincidofovir
- Bromelain (pharmacology)
- Budesonide
- Bulevirtide
- Buprenorphine/naloxone
- Burosumab
- Busulfan
C
- C-Met inhibitor
- Cabozantinib
- Caffeine citrate
- Calaspargase pegol
- Cannabidiol
- Caplacizumab
- Capmatinib
- Carboprost
- Carglumic acid
- Carmustine
- Casimersen
- Catridecacog
- CER-001
- Cereblon E3 ligase modulator
- Cerliponase alfa
- Chlormethine
- Cholic acid
- Ciclosporin
- Cilengitide
- Ciltacabtagene autoleucel
- Cipaglucosidase alfa
- Cladribine
- Clofazimine
- Cobicistat
- Cobimetinib
- Colchicine
- Collagenase clostridium histolyticum
- Copper (64Cu) oxodotreotide
- Crizanlizumab
- Cromoglicic acid
- Cysteamine
D
- Dabrafenib
- Dacomitinib
- Danicopan
- Dantrolene
- Daratumumab
- Darbepoetin alfa
- Darvadstrocel
- Dasatinib
- Dasiglucagon
- Daunorubicin/cytarabine
- Decitabine/cedazuridine
- Deferasirox
- Defibrotide
- Depatuxizumab mafodotin
- Dexrazoxane
- Diflunisal
- Dimethyl fumarate
- Donislecel
- DOTA-TATE
- Doxorubicin
- Durvalumab
- Duvelisib
E
- Eculizumab
- Edaravone
- Efanesoctocog alfa
- Efgartigimod alfa
- Eflornithine
- Eladocagene exuparvovec
- Elafibranor
- Elapegademase
- Elesclomol
- Elexacaftor
- Elexacaftor/tezacaftor/ivacaftor
- Elivaldogene autotemcel
- Elotuzumab
- Elranatamab
- Eltrombopag
- Emapalumab
- Enasidenib
- Encorafenib
- Entinostat
- Entrectinib
- Enzastaurin
- Epcoritamab
- Eplontersen
- Eribulin
- Erlotinib
- Estetrol
- Etranacogene dezaparvovec
- Everolimus
- Evinacumab
- Exagamglogene autotemcel
F
G
I
- Ibalizumab
- Ibrutinib
- Ibutamoren
- Icatibant
- Idebenone
- Idecabtagene vicleucel
- Iduronidase
- Idursulfase
- Iloprost
- Imatinib
- Imiglucerase
- Imlifidase
- Inclisiran
- Inebilizumab
- Infigratinib
- Inotersen
- Inotuzumab ozogamicin
- Iptacopan
- IRGD
- Irinotecan
- Isatuximab
- Istiratumab
- Itolizumab
- Itraconazole
- Ivacaftor
- Ivosidenib
- Ixazomib
L
- Lanadelumab
- Larotrectinib
- Lecanemab
- Lenacapavir
- Leniolisib
- Lenvatinib
- Letermovir
- Lifileucel
- Lintuzumab
- Lisocabtagene maraleucel
- Lithium carbonate
- Lomitapide
- Lonafarnib
- Lonapegsomatropin
- Loncastuximab tesirine
- Lorlatinib
- Losartan
- Losmapimod
- Lovotibeglogene autotemcel
- Lumacaftor
- Lumacaftor/ivacaftor
- Lumasiran
- Lurbinectedin
- Luspatercept
- Lusutrombopag
- Lutetium (177Lu) chloride
- Lutetium (177Lu) oxodotreotide