Orphan Designations by geographical area
- C1-esterase inhibitor
- Cabiralizumab
- Cabozantinib
- Caffeine citrate
- Camsirubicin
- Canakinumab
- Cannabidiol
- Cannabidivarin
- Caprine hyperimmune serum against HIV lysate
- Carbamazepine (intravenous)
- Carbenoxolone
- Carbetocin
- Carboxypeptidase G2
- Cardiotrophin-1
- Carfilzomib
- Carglumic acid
- Carmustine
- Catridecagog
- Catumaxomab
- CD34+ haematopoietic stem and progenitor cells with CD3+ T-cells
- Cediranib
- Ceftriaxone
- Celecoxib
- Cenersen
- Cetuximab
- Chemically modified human recombinant sulfamidase
- Chenodeoxycholic acid
- Chimeric fibril-reactive IgG1k monoclonal antibody 11-1F4
- Chimeric fusion protein of recombinant human alpha-N-acetylglucosaminidase and human insulin-like growth factor 2
- Chimeric locked nucleic acid-deoxynucleoside phosphorothioate-linked oligonucleotide directed against microRNA-451
- Chimeric monoclonal antibody against claudin-18 splice variant 2
- Chimeric monoclonal antibody against claudin 6
- Chimeric monoclonal antibody to O-acetyl-GD2 antigen
- Chimeric monoclonal antibody to shiga-toxin 1 and 2
- Chlormethine
- Cholic acid
- Choline tetrathiomolybdate
- Ciclopirox
- Ciclosporin
- Ciclosporin (eye drops, solution)
- Ciclosporin (inhalation use)
- Cilengitide
- Cilofexor
- Cinacalcet
- Ciprofloxacin (liposomal)
- Cisplatin
- Cisplatin (liposomal)
- Cladribine
- Clenbuterol
- Clofarabine
- Clofazimine
- Clonidine hydrochloride
- Cobicistat
- codon-optimised human cystic fibrosis transmembrane conductance regulator messenger ribonucleic acid complexed with lipid-based nanoparticles
- Codon -optimised human ornithine transcarbamylase mRNA complexed with lipid -based nanoparticle
- Colchicine
- Collagenase clostridium histolyticum
- Combination of 4-hydroxyandrostenedione, Serenoa serrulata fruit extract and alpha lipoic acid
- Combination of carboplatin and sodium valproate
- Combination of two adeno-associated viral vectors of serotype 8 containing the 5'- and the 3'- half coding sequences of human ABCA4 fused to inteins
- Combretastatin A1 diphosphate-
- Combretastatin A4 phosphate (CA4P)
- Concentrate of proteolytic enzymes enriched in bromelain
- conestat alfa
- Copanlisib
- Copper meso-5,15-bis[3-[(1,2-dicarba-closo-dodecaboranyl)methoxy]phenyl]-meso-12,20-dinitroporphyrin
- Cosyntropin
- Coversin
- Crenolanib besylate
- Crinecerfont
- Crisantaspase
- Cultured allogeneic corneal limbal stem cells
- Cyclic pyranopterin monophosphate
- Cyclo-Cys-Gly-Gln-Arg-Glu-Thr-Pro-Glu-Gly-Ala-Glu-Ala-Lys-Pro-Trp-Tyr-Cys
- Cyclo(-gamma-aminobutyryl-L-phenylalanyl-L-tryptophanyl-D-tryptophanyl-L-lysyl-L-threonyl-L phenylalanyl-N-3-carboxypropyl)-glycine amide, acetate salt
- Cyclo[L-alanyl-L-seryl-L-isoleucyl-L-prolyl-L-prolyl-L-glutaminyl-L-lysyl-L-tyrosyl-D-prolyl-L-prolyl-(2S)-2-aminodecanoyl-L-alpha-glutamyl-L-threonyl] acetate salt
- Cyclopropane-1,1-dicarboxylic acid [4-(6,7-dimethoxy-quinolin-4-yloxy)-phenyl]-amide (4-fluoro-phenyl)-amide, (L)-malate salt
- Cysteamine
- Cysteamine bitartrate
- Cysteamine hydrochloride
- Cystic fibrosis transmembrane conductance regulator (CFTR) amplifier
- Cytochrome P450 isoform 2B1 gene transfected human embryonic kidney 293 cells encapsulated in polymeric cellulose sulphate