Medicinal Products for Rare and Severe Diseases
Rare diseases are defined as life threatening disease or diseases that may lead to chronic weaknesses, affecting maximally five out of 10 000 people in the EU. In other words, between 27 and 36 million people in the EU is affected by a rare disease.
Symptoms of rare diseases may exist from the birth or childhood as in the case of spinal amyotrophic disease, lysosomal storage disorders, open ductus arteriosus disease, familiar adenomatous polyposis and cystic fibrosis. More than half of rare diseases develop in adult age, as in the case of kidney cell carcinoma, glioma and myeloid leukaemia.
Approximately 80% of all rare diseases are inherited genetically and develop between 3 and 4% of births, while others develop as a consequence of degenerative or proliferative changes.
Taking account that under usual market conditions, pharmaceutical industry has a very small interest in developing and placing on the market of medicinal products intended to a small number of patients, the EU has created a number of incentives for development of these medicines. These incentives are set out in Regulation (EC) 141/2000 (so called "Orphan" Regulation) and Regulation (EC) 847/2000. The procedure for designating the orphan status to a certain medicine as well as incentives for development and placing on the market of designated medicines are defined by these regulations.
The "Orphan" status may be granted to sponsors who have proven that:
- the medicine is intended for the diagnosis or treatment of conditions or diseases that lead to chronical weaknesses
- the disease prevalence in the EU is not more than five out of ten thousand persons in the EU, or marketing of the product cannot generate the revenue invested in its development
- no satisfying diagnostic, prevention and treatment method of these conditions exists, or if they exist the medicine will have a proven benefit for the persons affected with this condition.
HALMED has made publically available a list of all orphan medicinal products authorised in the EU via centralised procedure. Marketing authorisations of these medicines are valid in the Republic of Croatia, and access to information of authorised "orphan" medicinal products in Croatia is provided by accessing the list on the HALMED website as well as the access to information on certain authorised medicinal product (Summary of Product Characteristics and Package Leaflet).
The list is reviewed once a month.
Last update of the table: 01.03.2021
Medicinal Products for Rare and Severe Diseases
Name | Active Substance | ATC Code |
---|---|---|
Adakveo | crizanlizumab | B06AX01 |
Adcetris | brentuximab vedotin | L01XC12 |
Adempas | riociguat | C02KX05 |
Alofisel | darvadstrocel | L04 |
Alprolix | eftrenonacog alfa | B02BD04 |
Amglidia | glibenclamide | A10BB01 |
Arikayce liposomal | amikacin | J01GB06 |
Ayvakyt | avapritinib | L01EX18 |
Besponsa | inotuzumab ozogamicin | L01XC26 |
Blenrep | belantamab mafodotin | L01XC39 |
Blincyto | blinatumomab | L01XC19 |
Brineura | cerliponase alfa | A16AB |
Bronchitol | mannitol | R05CB16 |
Cablivi | caplacizumab | B01AX07 |
Carbaglu | carglumic acid | A16AA05 |
Cerdelga | eliglustat | A16AX10 |
Chenodeoxycholic acid Leadiant | chenodeoxycholic acid | A05AA01 |
Coagadex | human coagulation factor X | B02BD13 |
Cometriq | cabozantinib | L01XE |
Cresemba | isavuconazole | J02AC05 |
Crysvita | burosumab | M05BX05 |
Cystadrops | mercaptamine | S01XA21 |
Dacogen | decitabine | L01BC08 |
Darzalex | daratumumab | L01XC24 |
Daurismo | glasdegib | L01XX63 |
Defitelio | defibrotide | B01AX01 |
Deltyba | delamanid | J04AK06 |
Elzonris | tagraxofusp | L01XX67 |
Epidyolex | cannabidiol | N03AX24 |
Esbriet | pirfenidone | L04AX05 |
Farydak | panobinostat | L01XX42 |
Fintepla | fenfluramine | N03AX26 |
Galafold | migalastat | A16AX14 |
Gazyvaro | obinutuzumab | L01XC15 |
Givlaari | givosiran | |
Granupas | para-aminosalicylic acid | J04AA01 |
Hepcludex | bulevirtide | J05AX28 |
Hetlioz | tasimelteon | N05CH03 |
Holoclar | Ex vivo expanded autologous human corneal epithelial cells containing stem cells | S01XA19 |
Iclusig | ponatinib | L01XE24 |
Idefirix | imlifidase | L04AA |
Idelvion | albutrepenonacog alfa | B02BD04 |
Imbruvica | ibrutinib | L01XE27 |
Imnovid | pomalidomide | L04AX06 |
Isturisa | osilodrostat | H02CA02 |
Jorveza | budesonide | A07EA06 |
Kaftrio | ivacaftor / tezacaftor / elexacaftor | R07AX32 |
Kalydeco | ivacaftor | R07AX02 |
Kanuma | sebelipase alfa | A16 |
Ketoconazole HRA | ketoconazole | J02AB02 |
Kuvan | sapropterin dihydrochloride | A16AX07 |
Kymriah | tisagenlecleucel | L01 |
Kyprolis | carfilzomib | L01XX45 |
Lamzede | velmanase alfa | A16AB15 |
Ledaga | chlormethine | L01AA05 |
Libmeldy | autologous CD34+ cell enriched population that contains hematopoietic stem and progenitor cells transduced ex vivo using a lentiviral vector encoding the human arylsulfatase A gene | |
Lutathera | lutetium (177Lu) oxodotreotide | V10XX04 |
Luxturna | voretigene neparvovec | |
Mepsevii | vestronidase alfa | A16AB18 |
Mozobil | plerixafor | L03AX16 |
Myalepta | metreleptin | A16AA07 |
Mylotarg | gemtuzumab ozogamicin | L01XC05 |
Namuscla | mexiletine | C01BB02 |
Natpar | parathyroid hormone | H05AA03 |
NexoBrid | concentrate of proteolytic enzymes enriched in bromelain | D03BA03 |
Ninlaro | ixazomib | L01XX50 |
Normosang 25 mg/ml koncentrat za otopinu za infuziju | humani hemin | B06AB |
Obiltoxaximab SFL | obiltoxaximab | J06BB22 |
Ocaliva | obeticholic acid | A05AA04 |
Onivyde pegylated liposomal | irinotecan | L01XX19 |
Onpattro | patisiran | N07 |
Opsumit | macitentan | C02KX04 |
Orphacol | cholic acid | A05AA03 |
Oxervate | cenegermin | S01 |
Oxlumo | lumasiran | A16AX |
Palynziq | pegvaliase | A16AB19 |
Plenadren | hydrocortisone | H02AB09 |
Polivy | polatuzumab vedotin | L01XC |
Poteligeo | mogamulizumab | L01XC25 |
Pretomanid FGK | pretomanid | J04 |
Prevymis | letermovir | J05AX18 |
Procysbi | mercaptamine bitartrate | A16AA04 |
Qarziba | dinutuximab beta | L01XC |
Ravicti | glycerol phenylbutyrate | A16AX09 |
Raxone | idebenone | N07 |
Reblozyl | luspatercept | B03XA06 |
Revestive | teduglutide | A16AX08 |
Rydapt | midostaurin | L01XE39 |
Scenesse | afamelanotide | D02BB02 |
Signifor | pasireotide diaspartate | H01CB05 |
Sirturo | bedaquiline | J04A |
Soliris | eculizumab | L04AA25 |
SomaKit TOC | edotreotide | V09IX09 |
Spinraza | nusinersen | M09AX07 |
Strensiq | asfotase alfa | A16AB |
Strimvelis | autologous CD34+ enriched cell fraction that contains CD34+ cells transduced with retroviral vector that encodes for the human adenosine deaminase (ADA) cDNA sequence from human haematopoietic stem/progenitor (CD34+) cells | L03 |
Sylvant | siltuximab | L04AC11 |
Symkevi | tezacaftor / ivacaftor | R07AX31 |
Takhzyro | lanadelumab | B06AC05 |
Tecartus | autologous peripheral blood T cells CD4 and CD8 selected and CD3 and CD28 activated transduced with retroviral vector expressing anti-CD19 CD28/CD3-zeta chimeric antigen receptor and cultured | |
Tegsedi | inotersen | N07 |
Tobi Podhaler | tobramycin | J01GB01 |
Translarna | ataluren | M09AX03 |
Trecondi | treosulfan | L01AB02 |
Trepulmix | treprostinil sodium | B01AC21 |
Verkazia | ciclosporin | S01XA18 |
Vimizim | elosulfase alfa | A16AB12 |
Votubia | everolimus | L01XE10 |
Vpriv | velaglucerase alfa | A16AB10 |
Vyndaqel | tafamidis | N07XX08 |
Vyxeos | daunorubicin / cytarabine | L01XY01 |
Wakix | pitolisant | N07XX11 |
Waylivra | volanesorsen | |
Xaluprine | 6-mercaptopurine monohydrate | L01BB02 |
Xermelo | telotristat | A16AX |
Xospata | gilteritinib | L01XE54 |
Yescarta | axicabtagene ciloleucel | L01X |
Zejula | niraparib | L01XX54 |
Zolgensma | onasemnogene abeparvovec | M09AX09 |
Zynteglo | autologous CD34+ cells encoding βA-T87Q-globin gene | B06A |