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:
Pages in category "Orphan drugs"
The following 200 pages are in this category, out of approximately 471 total. This list may not reflect recent changes.
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- Acalabrutinib
- Acetohydroxamic acid
- Adagrasib
- Adalimumab
- Afamelanotide
- 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
- 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
- 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