Category:Orphan drugs
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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 [dead link]
- European Commission Register of designated Orphan Medicinal Products
Pages in category "Orphan drugs"
The following 200 pages are in this category, out of approximately 489 total. This list may not reflect recent changes.
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- Acalabrutinib
- Acetohydroxamic acid
- Acoramidis
- Adagrasib
- Adalimumab
- Afamelanotide
- Afamitresgene autoleucel
- Afatinib
- Afegostat
- Alectinib
- Alglucosidase alfa
- Alisporivir
- Allogeneic cultured keratinocytes and dermal fibroblasts in murine collagen
- Allogeneic processed thymus tissue
- Alpha-1 antitrypsin
- Altretamine
- Alvocidib
- Amatuximab
- Ambrisentan
- Amifampridine
- Amikacin
- 4-Aminopyridine
- Amitriptyline
- Anagrelide
- Ansuvimab
- Anti-CD22 immunotoxin
- Apadamtase alfa
- Artesunate
- Arylsulfatase B
- Asciminib
- Asfotase alfa
- Asparaginase
- Ataluren
- Atezolizumab
- Atidarsagene autotemcel
- Atoltivimab/maftivimab/odesivimab
- Avacopan
- Avalglucosidase alfa
- Avapritinib
- Avatrombopag
- Avelumab
- Axatilimab
- Axicabtagene ciloleucel
- Azacitidine
B
- 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
- Cenegermin
- 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
- Concizumab
- 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