::Definition
The Orphan Designation is a legal procedure that allows for the designation of a medicinal substance with therapeutic potential for a rare disease, before its first administration in humans or during its clinical development.
The exact therapeutic indication is then defined at the time of marketing authorisation.
This procedure has been established in Europe by the Regulation (EC) No 141/2000
(http://eur-lex.europa.eu/LexUriServ/LexUriServ.do?uri=OJ:L:2000:018:0001:0005:EN:PDF)
Similar procedures exist in the US, Australia, Japan and Singapore.
::Help
Choose a letter from the alphabetical list and click on it in order to obtain substances with at least an orphan designation. The concerned geographical area(s) is(are) indicated.
Then click on the substance name to access to the “Substance” sheet or click on the geographical area in order to obtain detailed information on the designation(s) in that particular area.
The list of Orphan Drugs in the Orphanet database includes all the substances which have been granted an orphan designation for disease(s) considered as rare in Europe, whether they were further developed to become drugs with marketing authorisation (MA) or not.
The data is extracted from official sources.
You can also access European or American designation data, respectively, through these two links :
http://ec.europa.eu/health/documents/community-register/html/alforphreg.htm(Europe)
http://www.fda.gov/ForIndustry/DevelopingProductsforRareDiseasesConditions/HowtoapplyforOrphanProductDesignation/default.htm (USA)
Orphanet acknowledges the contribution of the European Medicines Agency (EMA) to the creation of the database on orphan medicinal products in Europe. This project was initiated by the Committee for Orphan Medicinal Products (COMP) at the EMA.
Several members of the COMP have individually contributed to the development of the database, which was welcomed by the COMP as a significant tool for all stakeholders.
The database has been developed entirely and autonomously by Orphanet, using public and private funding and publicly available data; as such, neither the EMA nor the COMP are responsible for the content and management of the database.
The Orphanet database of orphan drugs was developed with co-funding from the following institutions and companies:







Orphan Designations by geographical area
- AASSGVSTPGSAGHDIITEQPRS (P42 peptide)
- Abagovomab
- Acadesine
- Acalabrutinib
- Acamprosate calcium
- Acebutolol
- Acetylleucine
- Acetylsalicylic acid
- Ad5/3-D24-granulocyte-macrophage colony stimulating factor (GMCSF)-encoding oncolytic adenovirus
- Adalimumab
- Adeno-associated viral (AAV) vector expressing human retinoschisin-1 gene
- Adeno-associated viral vector containing a modified U7-snRNA gene
- Adeno-associated viral vector containing DNA encoding an RNAi targeting rhodopsin / adeno-associated viral vector containing a rhodopsin gene
- Adeno-associated viral vector containing modified U1 snRNA
- Adeno-associated viral vector containing porphobilinogen deaminase gene
- Adeno-associated viral vector containing the human alpha-N-acetylglucosaminidase gene
- Adeno-associated viral vector containing the human ARSB gene
- Adeno-associated viral vector containing the human factor IX gene
- Adeno-associated viral vector containing the human NADH dehydrogenase 4 gene (rAAV2/2-ND4)
- Adeno-associated viral vector encoding an inducible short hairpin RNA targeting claudin-5 (prior to administration of 17-dimethylaminoethylamino-17-demethocygeldanamycin)
- Adeno-associated viral vector of serotype 5 containing the human alanine-glyoxylate aminotransferase gene
- Adeno-associated viral vector serotype 10 carrying the human N-sulfoglucosamine sulfohydrolase cDNA
- Adeno-associated viral vector serotype 2.7m8 containing the ChrimsonR-tdTomato gene
- Adeno-associated viral vector serotype 2 containing the human CHM gene encoding human Rab escort protein 1
- Adeno-associated viral vector serotype 2 containing the human Rab escort protein 1 gene
- Adeno-associated viral vector serotype 2 containing the human REP1 gene
- Adeno-associated viral vector serotype 5 containing a B-domain deleted variant of human coagulation factor VIII gene
- Adeno-associated viral vector serotype 5 containing the human CHM gene
- Adeno-associated viral vector serotype 5 containing the human RLBP1 gene
- Adeno-associated viral vector serotype 8 containing the human AIPL1 gene
- Adeno-associated viral vector serotype 8 containing the human alpha-galactosidase A gene
- Adeno-associated viral vector serotype 8 containing the human CNGA3 gene under the control of a cone arrestin promoter
- Adeno-associated viral vector serotype 8 containing the human factor-VII gene
- adeno-associated viral vector serotype 8 containing the human glucose-6-phosphatase gene
- Adeno-associated viral vector serotype 8 containing the human GUCY2D gene
- Adeno-associated viral vector serotype 8 containing the human MTM1 gene
- Adeno-associated viral vector serotype 8 containing the human UGT1A1 gene
- Adeno-associated viral vector serotype 8 encoding engineered rhodopsin DNA-binding repressor and human rhodopsin expression cassettes
- Adeno-associated viral vector serotype 8 encoding human ornithine transcarbamylase
- adeno-associated viral vector serotype 8 encoding the human ATP7B gene under the control of the human alpha-1 antitrypsin promoter
- Adeno-associated viral vector serotype 9 containing the human cardiac calsequestrin gene
- adeno-associated viral vector serotype 9 containing the human glucocerebrosidase gene
- adeno-associated viral vector serotype 9 containing the human HGSNAT gene
- Adeno-associated viral vector serotype 9 containing the human iduronate-2-sulfatase gene
- Adeno-associated viral vector serotype 9 containing the human mini-dystrophin gene
- Adeno-associated viral vector serotype 9 containing the human N-acetylglucosaminidase alpha gene
- Adeno-associated viral vector serotype 9 containing the human sulfamidase gene
- Adeno-associated viral vector serotype Anc80 containing the truncated human ATP7B gene under the control of the human alpha-1 antitrypsin promoter
- Adeno-associated viral vector serotype LK03 encoding human ornithine transcarbamylase
- Adeno-associated viral vector serotype rh.10 expressing beta-galactosidase
- Adeno-associated virus serotype 9 expressing the human Survival Motor Neuron gene
- Adenoassociated virus vector (AAV) carrying a modified AAV serotype 2 backbone and coding sequence of human thymidine phosphorylase preceded by a human thyroxin-binding globulin promoter
- Adeno associated virus with modified transthyretin and sequence encoding factor IX variant gene
- Adenoviral vector containing human p53 gene
- Adenoviral vector of serotype 5 modified to contain a chimeric sequence consisting of a minimal urokinase-type plasminogen activator receptor promoter preceded by three Notch-responsive elements, and coated with oligopeptide end-modified poly (beta-amino) esters
- Adenoviral vector serotype 5 containing the vascular endothelial growth factor D isoform (preprocessed short form) from a CMV promoter
- Adenovirus-associated vector containing human Fas-c gene
- Adenovirus-associated viral vector serotype 10 carrying the human N-sulfoglucosamine sulfohydrolase and sulfatase modifying factor 1 cDNAs
- Adenovirus-associated viral vector serotype 2 containing the human RPE65 gene (AAV2-hRPE65v2)
- Adenovirus associated viral vector serotype 4 containing the human RPE65 gene
- Adenovirus associated viral vector serotype 5 containing the human pde6 beta gene
- Adenovirus associated viral vector serotype 5 containing the human RPE65 gene
- adenovirus associated viral vector serotype 5 containing the RPGR gene
- Adenovirus associated viral vector serotype 8 containing the human CNGB3 gene
- Adenovirus delta 24-RGD
- Adenovirus-mediated Herpes Simplex Virus-thymidine kinase (HKSV-tk) gene
- Adenovirus serotype 5 containing partial E1A deletion and an integrin-binding domain
- Adenovirus-specific T-cells derived from allogeneic donor leukocytes, expanded ex vivo
- A-dmDT390-bisFv(UCHT1)
- Adrenomedullin
- Adult human bone-marrow-derived, ex-vivo-expanded, pooled allogeneic mesenchymal stromal cells
- Afamelanotide
- Agalsidase alfa
- Agalsidase beta
- A highly purified formulation of Staphylococcus aureus protein A
- Albendazole
- Albutrepenonacog alfa
- Aldesleukin
- Aldoxorubicin
- Alemtuzumab
- A lentiviral vector pseudotyped by the Indiana serotype of the vesicular stomatitis virus G protein encoding an antigen derived from the Tax, HBZ, p12I and p30II HTLV-1 proteins
- A lentiviral vector pseudotyped by the New-Jersey serotype of the vesicular stomatitis virus G protein encoding an antigen derived from the Tax, HBZ, p12I and p30II HTLV-1 proteins
- Alginate oligosaccharide (G-block) fragment
- Alglucerase
- Alglucosidase alfa
- Alicaforsen
- Alipogene tiparvovec
- Alisertib
- Alitretinoin
- Allantoin
- All-cis-docosa-4,7,10,13,16,19-hexaenoic acid
- Allogeneic adipose-derived adult mesenchymal stem cells contained in a fibrin-based bioengineered dermis
- Allogeneic and autologous haptenised and irradiated cells and cell lysates derived from glioma
- Allogeneic aortic endothelial cells cultured in a porcine gelatin matrix
- Allogeneic bone-marrow derived adherent ex-vivo expanded multipotent adult
- Allogeneic bone marrow derived mesenchymal cells expanded ex vivo in synthetic media
- Allogeneic bone marrow stem cells treated ex vivo with 16,16-dimethyl prostaglandin E2
- Allogeneic CD34+ cells expanded ex vivo with an aryl hydrocarbon receptor antagonist
- Allogeneic donor-derived ex-vivo expanded T lymphocytes transduced with a retroviral vector containing inducible caspase 9 and truncated CD19
- Allogeneic Epstein-Barr virus specific cytotoxic T lymphocytes
- Allogeneic ex-vivo-expanded human umbilical cord blood-derived mesenchymal stem cells
- Allogeneic ex vivo expanded umbilical cord blood cells
- Allogeneic ex-vivo-expanded umbilical cord blood-derived haematopoietic CD34+ progenitor cells and allogeneic non-expanded umbilical cord blood-derived haematopoietic mature myeloid and lymphoid cells
- Allogeneic ex vivo-generated natural killer cells from CD34+ umbilical cord blood progenitor cells
- Allogeneic fetal human retinal progenitor cells expanded ex vivo
- Allogeneic human dendritic cells derived from a CD34+ progenitor cell line
- Allogeneic human dermal fibroblasts
- Allogeneic motor neuron progenitor cells derived from human embryonic stem cells
- Allogeneic peripheral blood mononuclear cells incubated ex-vivo with 16, 16-dimethyl prostaglandin E2 and dexamethasone
- Allogeneic peripheral blood mononuclear cells induced to an early apoptotic state
- Allogeneic retinal epithelial cells transfected with plasmid vector expressing ciliary neurotrophic growth
- Allogeneic T cells encoding an exogenous TK gene
- Allogeneic umbilical cord blood cells treated ex vivo with 16,16-dimethyl prostaglandin E2
- Allogenic cytomegalovirus-specific cytotoxic T lymphocytes
- Allopregnanolone
- Allopurinol sodium
- Alpha-1 antitrypsin (inhalation use)
- Alpha-1 proteinase inhibitor
- Alpha-1 proteinase inhibitor (inhalation use)
- Alpha-tocopherol
- Alpha-tocopherol and ascorbic acid
- Alpha-tocotrienol quinone
- Altiratinib
- Alvocidib
- Amatuximab
- Ambrisentan
- Amikacin sulfate
- Amikacin sulfate (liposomal)
- Aminosalicylic acid
- A mixture of anti-CD3 mAb (SPV-T3a)-ricin A chain fusion protein and anti-CD7 mAb (WT1)-ricin A chain fusion protein
- Ammonium tetrathiomolybdate
- Amphotericin B (for inhalation use)
- Amphotericin B lipid complex
- Amrubicin hydrochloride
- [a-N-(2'succinyl-paclitaxel)Thr]-Phe-Phe-Tyr-Gly-Gly-Ser-Arg-Gly-[epsilon-N-(2'succinyl-paclitaxel)Lys]-Arg-Asn-Asn-Phe-[epsilon-N-(2'succinyl-paclitaxel)Lys]-Thr-Glu-Glu-Tyr
- Anagrelide hydrochloride
- Anakinra
- Angiotensin (1-7)
- Antagonist of the complement 5a receptor
- Anti-beta1 integrin monoclonal antibody
- Anti-EphA2 monoclonal antibody conjugated to maleimidocaproyl monomethylauristatin phenylalanine
- Anti-epithelial cell adhesion molecule / anti-CD3 monoclonal antibody
- Antihemophilic factor / Von Willebrand factor complex (human)
- Anti-inhibitor coagulant complex
- Anti RhO (D) immune globulin intravenous (human)
- Antisense NF-kappaB p65 Oligonucleotide
- Antisense oligonucleotide 5'-d[P-Thio} (CCCTG CTCCC CCCTG GCTCC)-3'
- Antisense oligonucleotide complementary to the exonic splicer enhancer sequence at intron 26 of the centrosomal protein 290 pre-mRNA
- Antisense oligonucleotide targeted to the SMN2 gene
- Antisense oligonucleotide targeting exon 13 in the USH2A gene
- Antisense oligonucleotide targeting exon 73 in the COL7A1 gene
- Antisense oligonucleotide targeting the F508delta mutation of CFTR
- Antisense oligonucleotide targeting the USH2A gene
- Antisense Oligonucleotide (TATCCGGAGGGCTCGCCATGCTGCT)
- Antithrombin alfa
- Antithrombin III (human)
- Anti-tumor necrosis factor polyclonal antibody (bovine)
- Apatinib mesylate
- Aplidine
- Apolipotrotein C-III antisense oligonucleotide
- Apomorphine hydrochloride
- Apomorphine hydrochloride (inhalation use)
- Apremilast
- Arimoclomol
- Arimoclomol citrate
- Arsenic trioxide
- Artemether / Lumefantrine
- Artesunate
- Ascorbic acid
- Asfotase alfa
- Asp-Arg-Val-Try-Ile-His-Pro
- Asunercept
- Ataluren
- Atovaquone
- Autologous adipose derived mesenchymal stromal cells
- Autologous adipose tissue-derived mesenchymal stem cells
- autologous adipose tissue-derived stromal vascular fraction cells
- autologous adult bone marrow-derived non-expanded CD133+ haematopoietic stem cells for the treatment of Asherman's syndrome
- Autologous bone marrow CD34+ cells transduced ex vivo with a self activating HIV-1 -based lentiviral vector, EFS-ADA
- Autologous bone marrow-derived mesenchymal stromal cells secreting neurotrophic factors
- Autologous bone marrow-derived mononuclear cell fraction
- Autologous CD34+ cells transduced with a lentiviral vector containing the human ADA gene
- Autologous CD34+ cells transduced with a lentiviral vector containing the human RAG1 gene
- Autologous CD34+ cells transduced with a lentiviral vector containing the human SGSH gene
- Autologous CD34+ cells transduced with lentiviral vector encoding the human beta globin gene
- Autologous CD34+ cells transduced with the W1.6_hWASP_WPRE lentiviral vector
- Autologous CD34+ cells transfected with lentiviral vector containing the human arylsulfatase AcDNA
- Autologous CD34+ cells transfected with lentiviral vector containing the Wiskott-Aldrich syndrome protein gene
- Autologous CD34+ cells transfected with retroviral vector containing adenosine deaminase gene
- Autologous CD34+ haematopoietic stem cells transduced with lentiviral vector encoding the human betaA-T87Q-globin gene
- Autologous CD34+ hematopoietic stem cells transduced with LentiGlobin BB305 lentiviral vector encoding the human BA-T87Q-globin gene
- Autologous CD3+ T cells transduced with retroviral vector containing a chimeric antigen receptor directed against CD19 (Autologous CD3+ T cells containing CD19 chimeric antigen receptor)
- Autologous CD4+ and CD8+ T cells expressing a CD19-specific chimeric antigen receptor
- Autologous CD4+ and CD8+ T cells transduced with lentiviral vector containing an affinity-enhanced T-cell receptor targeting the New York esophageal antigen-1
- Autologous collagen type II-specific regulatory T cells
- Autologous cultured endothelial cells on a donor human corneal disk
- Autologous dendritic cells pulsed with allogeneic tumor cell lysate
- Autologous dendritic cells pulsed with allogeneic tumour cell lysate
- Autologous dendritic cells pulsed with autologous cell lysate
- Autologous dendritic cells pulsed with recombinant human-fusion protein (mucin 1 - glutathione S transferase) coupled to oxidised polymannose
- Autologous dendritic cells pulsed with RNA from glioma stem cells
- Autologous dendritic cells pulsed with tumour antigen-derived synthetic peptides (MAGE-1, HER-2, AIM-2, TRP-2, gp-100, and interleukin-13 receptor alpha)
- Autologous engineered anti -CD19 Chimeric Antigen Receptor (CAR +) T -cells
- Autologous Epstein-Barr virus specific T cells derived from peripheral blood mononuclear cells, expanded ex vivo-
- Autologous ex-vivo-expanded leucocytes treated with 5-aza-2'-deoxycytidine
- Autologous ex-vivo-expanded peripheral polyclonal lymphocytes enriched in activated natural killer cells (CellProtect)
- Autologous genetically modified human dermal fibroblasts
- Autologous haematopoietic cells genetically modified with a lentiviral vector containing the human gp91(phox) gene
- Autologous haematopoietic stem cells transduced with lentiviral vector encoding the human beta-globin gene
- Autologous haematopoietic stem cells transduced with lentiviral vector Lenti-D encoding the human ABCD1 cDNA
- Autologous human peripheral blood Vdelta1+ T lymphocytes activated in vitro by cytokine and monoclonal antibody treatment
- Autologous mesenchymal stromal cells on a decellularised tracheal scaffold from a cadaveric donor
- Autologous mononuclear cells derived from human cord blood
- Autologous olfactory neural progenitors
- Autologous regulatory T cells with an immunophenotype of CD4+CD25hiFoxP3+
- Autologous renal cell tumor vaccine
- Autologous T-cells transduced with lentiviral vector encoding an anti-SLAMF7 CD28/CD3-zeta chimeric antigen receptor
- Autologous T lymphocyte-enriched population of cells transduced with a lentiviral vector encoding a chimeric antigen receptor targeting human B cell maturation antigen with 4-1BB and CD3-zeta intracellular signalling domains
- Autologous Tumor-Derived gp96 Heat Shock Protein-Peptide Complex
- Autologous tumour-derived immunoglobulin idiotype coupled to keyhole limpet haemocyanin
- Avacopan
- Avelumab
- Avian polyclonal IgY antibody against Pseudomonas aeruginosa
- Aviptadil
- Axalimogene filolisbac
- Axicabtagene ciloleucel
- Azacitidine
- Aztreonam lysinate (inhalation use)