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: 30.11.2017

Medicinal Products for Rare and Severe Diseases

Name Active Substance ATC Code
Adcetris brentuximab vedotin L01XC12
Adempas riociguat C02KX05
Alprolix eftrenonacog alfa B02BD04
Arzerra ofatumumab L01XC10
Atriance nelarabine L01BB07
Bavencio avelumab L01XC
Besponsa inotuzumab ozogamicin L01XC26
Blincyto blinatumomab L01XC19
Bosulif bosutinib (as monohydrate) L01XE14
Brineura cerliponase alfa A16AB
Bronchitol mannitol R05CB16
Carbaglu carglumic acid A16AA05
Cayston aztreonam lysine J01DF01
Ceplene histamine dihydrochloride L03AX14
Cerdelga eliglustat A16AX10
Chenodeoxycholic acid sigma-tau chenodeoxycholic acid A05AA01
Coagadex human coagulation factor X B02BD13
Cometriq cabozantinib L01XE
Cresemba isavuconazole J02AC05
Cystadane betaine anhydrous A16AA06
Cystadrops mercaptamine S01XA21
Dacogen decitabine L01BC08
Darzalex daratumumab L01XC24
Defitelio defibrotide B01AX01
Deltyba delamanid J04AK06
Diacomit stiripentol N03AX17
Dinutuximab beta Apeiron dinutuximab beta L01XC
Elaprase idursulfase A16AB09
Esbriet pirfenidone L04AX05
Farydak panobinostat L01XX42
Firazyr icatibant C01EB19
Firdapse amifampridine N07XX05
Galafold migalastat
Gazyvaro obinutuzumab L01XC15
Gliolan 5-aminolevulinic acid hydrochloride L01XD04
Granupas para-aminosalicylic acid J04AA01
Hetlioz tasimelteon N05CH03
Holoclar Ex vivo expanded autologous human corneal epithelial cells containing stem cells S01XA19
Iclusig ponatinib L01XE24
Idelvion albutrepenonacog alfa B02BD04
Imbruvica ibrutinib L01XE27
Imnovid pomalidomide L04AX06
Increlex mecasermin H01AC03
Inovelon rufinamide N03AF03
Kalydeco ivacaftor R07AX02
Kanuma sebelipase alfa A16
Ketoconazole HRA ketoconazole J02AB02
Kolbam cholic acid A05AA03
Kuvan sapropterin dihydrochloride A16AX07
Kyprolis carfilzomib L01XX45
Lartruvo olaratumab L01XC27
Ledaga chlormethine L01AA05
Lenvima lenvatinib L01XE29
Lutathera lutetium (177Lu) oxodotreotide V10XX04
Lynparza olaparib L01
Mepact mifamurtide L03AX15
Mozobil plerixafor L03AX16
Natpar parathyroid hormone H05AA03
NexoBrid concentrate of proteolytic enzymes enriched in bromelain D03BA03
Ninlaro ixazomib L01XX50
Nplate romiplostim B02BX04
Ocaliva obeticholic acid A05AA04
Ofev nintedanib L01XE31
Onivyde irinotecan L01XX19
Opsumit macitentan C02KX04
Orphacol cholic acid A05AA03
Oxervate cenegermin S01
Peyona caffeine citrate N06BC01
Plenadren hydrocortisone H02AB09
Procysbi mercaptamine bitartrate A16AA04
Ravicti glycerol phenylbutyrate A16AX09
Raxone idebenone N07
Revestive teduglutide A16AX08
Revlimid lenalidomide L04AX04
Rydapt midostaurin L01XE39
Scenesse afamelanotide D02BB02
Signifor pasireotide diaspartate H01CB05
Siklos hydroxycarbamide L01XX05
Sirturo bedaquiline J04A
Soliris eculizumab L04AA25
SomaKit TOC edotreotide V09IX09
Spinraza nusinersen
Sprycel dasatinib L01XE06
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
Tasigna nilotinib L01XE08
Tepadina thiotepa L01AC01
Thalidomide Celgene thalidomide L04AX02
Tobi Podhaler tobramycin J01GB01
Torisel temsirolimus L01XE09
Translarna ataluren
Venclyxto venetoclax L01X
Vidaza azacitidine L01BC07
Vimizim elosulfase alfa A16AB12
Volibris ambrisentan C02KX02
Votubia everolimus L01XE10
Vpriv velaglucerase alfa A16AB10
Vyndaqel tafamidis N07XX08
Wakix pitolisant N07XX11
Xagrid anagrelide L01XX35
Xaluprine 6-mercaptopurine monohydrate L01BB02
Xermelo telotristat A16AX
Yondelis trabectedin L01CX01
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