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: 30.11.2020

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
Epidyolex cannabidiol N03AX24
Esbriet pirfenidone L04AX05
Farydak panobinostat L01XX42
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
Kolbam cholic acid A05AA03
Kuvan sapropterin dihydrochloride A16AX07
Kymriah tisagenlecleucel L01
Kyprolis carfilzomib L01XX45
Lamzede velmanase alfa A16AB15
Ledaga chlormethine L01AA05
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
Ocaliva obeticholic acid A05AA04
Onivyde pegylated liposomal irinotecan L01XX19
Onpattro patisiran N07
Opsumit macitentan C02KX04
Orphacol cholic acid A05AA03
Oxervate cenegermin S01
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
Tegsedi inotersen N07
Tobi Podhaler tobramycin J01GB01
Translarna ataluren M09AX03
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
Zalmoxis allogeneic T cells genetically modified with a retroviral vector encoding for a truncated form of the human low affinity nerve growth factor receptor (ΔLNGFR) and the herpes simplex I virus thymidine kinase (HSV-TK Mut2) L01
Zejula niraparib L01XX54
Zolgensma onasemnogene abeparvovec M09AX09
Zynteglo autologous CD34+ cells encoding βA-T87Q-globin gene B06A