Medicinal Products

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: 18.10.2021

Medicinal Products for Rare and Severe Diseases

Name Active Substance ATC Code
Abecma idekaptagen vikleucel L01
Adakveo krizanlizumab B06AX01
Adcetris brentuksimab vedotin L01XC12
Adempas riocigvat C02KX05
Alofisel darvadstrocel L04AX08
Alprolix eftrenonacog alfa (fuzijski protein rekombinantnog faktora zgrušavanja krvi IX i Fc fragmenta) B02BD04
Amglidia glibenklamid A10BB01
Arikayce liposomal amikacinsulfat J01GB06
Ayvakyt avapritinib L01EX18
Besponsa inotuzumab ozogamicin L01XC26
Blenrep belantamab mafodotina L01XC39
Blincyto blinatumomab L01XC19
Brineura cerliponaza alfa A16AB17
Bronchitol manitol R05CB16
Bylvay odeviksibat seskvihidrat A05AX05
Cablivi kaplacizumab B01AX07
Carbaglu karglumatna kiselina A16AA05
Cerdelga eliglustattartarat A16AX10
Chenodeoxycholic acid Leadiant kenodeoksikolatna kiselina A05AA01
Coagadex ljudski koagulacijski faktor X B02BD13
Cometriq kabozantinib (S)-malat L01EX07
Cresemba izavukonazonijev sulfat J02AC05
Crysvita burosumab M05BX05
Cystadrops mercaptamine S01XA21
Dacogen decitabin L01BC08
Darzalex daratumumab L01XC24
Daurismo glasdegibmaleat L01XJ03
Defitelio defibrotid B01AX01
Deltyba delamanid J04AK06
Dovprela pretomanid J04AK08
Elzonris tagraksofusp L01XX67
Enspryng satralizumab L04AC19
Epidyolex kanabidiol N03AX24
Esbriet pirfenidon L04AX05
Evrysdi risdiplam M09AX10
Farydak bezvodni panobinostatlaktat L01XH03
Fintepla fenfluraminklorid N03AX26
Galafold migalastatklorid A16AX14
Gazyvaro obinutuzumab L01XC15
Givlaari givosirannatrij A16AX16
Granupas paraaminosalicilatna kiselina J04AA01
Hepcludex bulevirtidacetat J05AX28
Hetlioz tazimelteon N05CH03
Holoclar Ex vivo expanded autologous human corneal epithelial cells containing stem cells S01XA19
Iclusig ponatinibklorid L01EA05
Idefirix imlifidaza L04AA41
Idelvion albutrepenonakog alfa (rekombinantni koagulacijski faktor IX) B02BD04
Imbruvica ibrutinib L01EL01
Imcivree setmelanotid A08AA12
Imnovid pomalidomid L04AX06
Inrebic fedratinibklorid hidrat L01EJ02
Isturisa osilodrostatfosfat H02CA02
Jorveza budezonid A07EA06
Kaftrio ivakaftor; tezakaftor; eleksakaftor R07AX32
Kalydeco ivakaftor R07AX02
Kanuma sebelipaza alfa A16AB14
Ketoconazole HRA ketokonazol J02AB02
Koselugo selumetinib sulfat L01EE04
Kuvan sapropterindiklorid A16AX07
Kymriah tisagenlekleucel L01XX71
Kyprolis karfilzomib L01XG02
Lamzede velmanaz alfa A16AB15
Ledaga klormetin 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 N07
Lumoxiti moksetumomab pasudotoks L01XC34
Lutathera lutecijev (177Lu) oksodotreotid V10XX04
Luxturna voretigen neparvovek S01XA27
Mepsevii vestronidaz alfa A16AB18
Minjuvi tafasitamab L01XC35
Mozobil pleriksafor L03AX16
Myalepta metreleptin A16AA07
Mylotarg gemtuzumab ozogamicin L01XC05
Namuscla meksiletinklorid C01BB02
Natpar paratiroidni hormon H05AA03
NexoBrid koncentrat proteolitičkih enzima obogaćenih bromelainom D03BA03
Nexpovio selineksor L01XX66
Ninlaro iksazomibcitrat L01XG03
Normosang 25 mg/ml koncentrat za otopinu za infuziju humani hemin B06AB
Obiltoxaximab SFL obiltoksaksimab J06BB22
Ocaliva obetikolatna kiselina A05AA04
Onivyde pegylated liposomal irinotecan L01CE02
Onpattro patisirannatrij N07XX12
Opsumit macitentan C02KX04
Orphacol kolatna kiselina A05AA03
Oxervate cenegermin S01XA24
Oxlumo lumasirannatrij A16AX18
Palynziq pegvaliaz A16AB19
Pemazyre pemigatinib L01EX20
Plenadren hidrokortizon H02AB09
Polivy polatuzumab vedotin L01XC37
Poteligeo mogamulizumab L01XC25
Prevymis letermovir J05AX18
Procysbi mercaptamine bitartrate A16AA04
Qarziba dinutuksimab beta L01XC16
Ravicti glicerolfenilbutirat A16AX09
Raxone idebenon N06BX13
Reblozyl luspatercept B03XA06
Revestive teduglutid A16AX08
Rydapt midostaurin L01EX10
Scenesse afamelanotid D02BB02
Signifor pasireotiddiaspartat H01CB05
Sirturo bedakilinfumarat J04AK05
Skysona elivaldogen autotemcel N07
Sogroya somapacitan H01AC07
Soliris ekulizumab L04AA25
SomaKit TOC edotreotida V09IX09
Spinraza nusinersennatrij M09AX07
Strensiq asfotaza alfa A16AB13
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 siltuksimab L04AC11
Symkevi tezakaftor, ivakaftor 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 L01X
Tegsedi inotersennatrij N07XX15
Tobi Podhaler tobramicin J01GB01
Translarna ataluren M09AX03
Trecondi treosulfan L01AB02
Trepulmix treprostinilnatrij B01AC21
Verkazia ciklosporin S01XA18
Vimizim elosulfaza alfa A16AB12
Votubia everolimus L01EG02
Voxzogo vosoritid M05BX07
Vpriv velagluceraza alfa A16AB10
Vyndaqel tafamidismeglumin N07XX08
Vyxeos liposomal daunorubicin, citarabin L01XY01
Wakix pitolizantklorid N07XX11
Waylivra volanesorsen natrij C10AX18
Xaluprine merkaptopurin hidrat L01BB02
Xermelo telotristatetiprat A16AX15
Xospata gilteritinib fumarat L01EX13
Yescarta axicabtagene ciloleucel L01XX70
Zejula niraparibtosilat hidrat L01XK02
Zolgensma onasemnogen abeparvovek M09AX09
Zynteglo autologous CD34+ cells encoding βA-T87Q-globin gene B06AX02