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: 26.03.2024

Medicinal Products for Rare and Severe Diseases

Name Active Substance ATC Code
Abecma idekaptagen vikleucel L01XL07
Adcetris brentuksimab vedotin L01FX05
Agamree vamorolon
Alofisel darvadstrocel L04AX08
Alprolix eftrenonacog alfa (fuzijski protein rekombinantnog faktora zgrušavanja krvi IX i Fc fragmenta) B02BD04
Amglidia glibenklamid A10BB01
Amvuttra vutrisiran N07XX18
Arikayce liposomal amikacinsulfat J01GB06
Artesunate Amivas artezunat P01BE03
Aspaveli pegcetakoplan L04AA54
Ayvakyt avapritinib L01EX18
Besponsa inotuzumab ozogamicin L01FB01
Blenrep belantamab mafodotin L01FX15
Blincyto blinatumomab L01FX07
Brineura cerliponaza alfa A16AB17
Bylvay odeviksibat seskvihidrat A05AX05
Cablivi kaplacizumab B01AX07
Carvykti ciltakaptagen autoleucel L01XL05
Casgevy eksagamglogen autotemcel B06AX05
Cerdelga eliglustattartarat A16AX10
Chenodeoxycholic acid Leadiant kenodeoksikolatna kiselina A05AA01
Coagadex koagulacijski faktor X, ljudski B02BD13
Columvi glofitamab L01FX28
Cometriq kabozantinib (S)-malat L01EX07
Cresemba izavukonazonijev sulfat J02AC05
Crysvita burosumab M05BX05
Cystadrops merkaptaminklorid S01XA21
Dacogen decitabin L01BC08
Darzalex daratumumab L01FC01
Daurismo glasdegibmaleat L01XJ03
Defitelio defibrotid B01AX01
Deltyba delamanid J04AK06
Dovprela pretomanid J04AK08
Ebvallo tabelekleucel
Elzonris tagraksofusp L01XX67
Enspryng satralizumab L04AC19
Enjaymo sutimlimab L04AA55
Epidyolex kanabidiol N03AX24
Evrysdi risdiplam M09AX10
Farydak bezvodni panobinostatlaktat L01XH03
Filsuvez suhi ekstrakt brezove kore (5-10:1), ekstrakcijsko otapalo n-heptan 95% D03AX13
Finlee dabrafenibmesilat L01EC02
Fintepla fenfluraminklorid N03AX26
Galafold migalastatklorid A16AX14
Gazyvaro obinutuzumab L01XC15
Givlaari givosirannatrij A16AX16
Granupas paraaminosalicilatna kiselina J04AA01
Hemgenix etranakogen dezaparvovek B06
Hepcludex bulevirtidacetat J05AX28
Hetlioz tazimelteon N05CH03
Holoclar Ex vivo umnožene autologne epitelne stanice ljudske rožnice koje sadrže matične stanice S01XA19
Hyftor sirolimus L04AA10
Iclusig ponatinibklorid L01EA05
Idefirix imlifidaza L04AA41
Idelvion albutrepenonakog alfa (rekombinantni koagulacijski faktor IX) B02BD04
Imcivree setmelanotid A08AA12
Imnovid pomalidomid L04AX06
Inrebic fedratinibklorid hidrat L01EJ02
Isturisa osilodrostatfosfat H02CA02
Jorveza budezonid A07EA06
Kaftrio ivakaftor; tezakaftor; eleksakaftor R07AX32
Kanuma sebelipaza alfa A16AB14
Ketoconazole HRA ketokonazol J02AB02
Kimmtrak tebentafusp L01
Kinpeygo budezonid A07EA06
Koselugo selumetinib sulfat L01EE04
Kymriah tisagenlekleucel L01XL04
Kyprolis karfilzomib L01XG02
Lamzede velmanaza alfa A16AB15
Ledaga klormetin L01AA05
Libmeldy atidarsagen autotemcel (obogaćena populacija autolognih stanica CD34+ koja sadrži hematopoetske matične i progenitorne stanice transducirane ex vivo lentivirusnim vektorom koji kodira gen ljudske arilsulfataze A (ARSA)) A16AB21
Livtencity maribavir J05AX10
Loargys pegzilarginaza A16AB24
Lunsumio mosunetuzumab L01FX25
Lutathera lutecijev[177Lu] oksodotreotid V10XX04
Luxturna voretigen neparvovek S01XA27
Mepsevii vestronidaza alfa A16AB18
Minjuvi tafasitamab L01FX12
Myalepta metreleptin A16AA07
Mycapssa oktreotidacetat H01CB02
Mylotarg gemtuzumab ozogamicin L01FX02
Namuscla meksiletinklorid C01BB02
Natpar paratiroidni hormon H05AA03
NexoBrid koncentrat proteolitičkih enzima obogaćenih bromelainom D03BA03
Ngenla somatrogon H01AC08
Ninlaro iksazomibcitrat L01XG03
Nulibry fosdenopterinbromid dihidrat A16AX19
Nyxthracis obiltoksaksimab J06BC04
Ocaliva obetikolatna kiselina A05AA04
Omjjara momelotinibdiklorid hidrat
Onivyde pegylated liposomal irinotekanklorid trihidrat L01CE02
Onpattro patisirannatrij N07XX12
Opsumit macitentan C02KX04
Orphacol kolatna kiselina A05AA03
Oxbryta vokselotor B06AX03
Oxervate cenegermin (rekombinantni ljudski faktor rasta živaca) S01XA24
Oxlumo lumasirannatrij A16AX18
Palynziq pegvaliaza A16AB19
Pemazyre pemigatinib L01EN02
Polivy polatuzumab vedotin L01FX14
Pombiliti cipaglukozidaza alfa A16AB23
Poteligeo mogamulizumab L01FX09
Prevymis letermovir J05AX18
Procysbi merkaptaminhidrogentartarat A16AA04
Pyrukynd mitapivatsulfat B06AX04
Qarziba dinutuksimab beta L01FX06
Qinlock ripretinib L01EX19
Ravicti glicerolfenilbutirat A16AX09
Raxone idebenon N06BX13
Reblozyl luspatercept B03XA06
Revestive teduglutid A16AX08
Rezzayo rezafunginacetat J02AX08
Roctavian valoktokogen roksaparvovek
Rydapt midostaurin L01EX10
Rystiggo rozanoliksizumab L04AG16
Scemblix asciminibklorid L01EA06
Scenesse afamelanotid D02BB02
Signifor pasireotiddiaspartat H01CB05
Sirturo bedakilinfumarat J04AK05
Skyclarys omaveloksolon
Skytrofa lonapegsomatropin H01AC
Sogroya somapacitan H01AC07
Soliris ekulizumab L04AA25
SomaKit TOC edotreotid V09IX09
Spexotras trametinib dimetilsulfoksid L01EE01
Spinraza nusinersennatrij M09AX07
Strensiq asfotaza alfa A16AB13
Strimvelis stanična frakcija obogaćena autolognim CD34+ stanicama koja sadrži CD34+ stanice transducirane retrovirusnim vektorom koji kodira sekvencu cDNA ljudske adenozin deaminaze (ADA) L03
Sylvant siltuksimab L04AC11
Symkevi tezakaftor; ivakaftor R07AX31
Takhzyro lanadelumab B06AC05
Talvey talkvetamab L01FX
Tavneos avakopan L04
Tecartus autologne T-stanice dobivene iz periferne krvi, odabrane pomoću čestica koje prepoznaju CD4 i CD8, aktivirane protutijelima protiv CD3 i CD28, transducirane retrovirusnim vektorom da bi eksprimirale CD28/CD3-zeta kimerični antigenski receptor protiv CD19 i uzgojene u kulturi (breksukaptagen autoleucel) L01XL06
Tegsedi inotersennatrij N07XX15
Tepkinly epkoritamab L01FX27
Tevimbra tislelizumab L01FF09
Tibsovo ivosidenib L01XX62
Tobi Podhaler tobramicin J01GB01
Translarna ataluren M09AX03
Trecondi treosulfan L01AB02
Trepulmix treprostinilnatrij B01AC21
Uplizna inebilizumab L04AA47
Upstaza eladokagen eksuparvovek A16AB26
Verkazia ciklosporin S01XA18
Vimizim elosulfaza alfa A16AB12
Voraxaze glukarpidaza V03AF09
Votubia everolimus L01EG02
Voxzogo vosoritid M05BX07
Vyndaqel tafamidismeglumin N07XX08
Vyvgart efgartigimod alfa L04AA58
Vyxeos liposomal daunorubicinklorid; citarabin L01XY01
Wakix pitolizantklorid N07XX11
Waylivra volanesorsen natrij C10AX18
Xaluprine merkaptopurin hidrat L01BB02
Xenpozyme olipudaza alfa A16AB25
Xermelo telotristatetiprat A16AX15
Xospata gilteritinibfumarat L01EX13
Yescarta aksikaptagen ciloleucel L01XL03
Yorvipath palopegteriparatid H05AA05
Zejula niraparibtosilat hidrat L01XK02
Zokinvy lonafarnib A16AX20
Zolgensma onasemnogen abeparvovek M09AX09
Ztalmy ganaksolon N03AX27