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: 21.11.2024
Medicinal Products for Rare and Severe Diseases
Name | Active Substance | ATC Code |
---|---|---|
Abecma | idekaptagen vikleucel | L01XL07 |
Adcetris | brentuksimab vedotin | L01FX05 |
Adzynma | rADAMTS13 | B01AD13 |
Agamree | vamorolon | H02AB18 |
Akantior | poliheksanid | S01AX24 |
Alofisel | darvadstrocel | L04AX08 |
Alprolix | eftrenonacog alfa (fuzijski protein rekombinantnog faktora zgrušavanja krvi IX i Fc fragmenta) | B02BD04 |
Altuvoct | efanesoktokog alfa | B02BD02 |
Amglidia | glibenklamid | A10BB01 |
Amvuttra | vutrisiran | N07XX18 |
Arikayce liposomal | amikacinsulfat | J01GB06 |
Artezunat Amivas | artezunat | P01BE03 |
Aspaveli | pegcetakoplan | L04AJ03 |
Ayvakyt | avapritinib | L01EX18 |
Besponsa | inotuzumab ozogamicin | L01FB01 |
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 | L01XL09 |
Elzonris | tagraksofusp | L01XX67 |
Enspryng | satralizumab | L04AC19 |
Enjaymo | sutimlimab | L04AJ04 |
Epidyolex | kanabidiol | N03AX24 |
Evrysdi | risdiplam | M09AX10 |
Fabhalta | iptakopanklorid hidrat | L04AJ08 |
Farydak | bezvodni panobinostatlaktat | L01XH03 |
Filspari | sparsentan | C09XX01 |
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 | L01FA03 |
Givlaari | givosirannatrij | A16AX16 |
Granupas | paraaminosalicilatna kiselina | J04AA01 |
Hemgenix | etranakogen dezaparvovek | B02BD16 |
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 | fedratinibdiklorid hidrat | L01EJ02 |
Iqirvo | elafibranor | A05AX06 |
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 |
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 |
Qalsody | tofersen | N07XX22 |
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 | B02BD15 |
Rydapt | midostaurin | L01EX10 |
Rystiggo | rozanoliksizumab | L04AG16 |
Scemblix | asciminibklorid | L01EA06 |
Scenesse | afamelanotid | D02BB02 |
Signifor | pasireotid | H01CB05 |
Sirturo | bedakilinfumarat | J04AK05 |
Skyclarys | omaveloksolon | |
Skytrofa | lonapegsomatropin | H01AC09 |
Sogroya | somapacitan | H01AC07 |
Soliris | ekulizumab | L04AJ01 |
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) | L03AX |
Sylvant | siltuksimab | L04AC11 |
Symkevi | tezakaftor; ivakaftor | R07AX31 |
Takhzyro | lanadelumab | B06AC05 |
Talvey | talkvetamab | L01FX29 |
Tavneos | avakopan | L04AJ05 |
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 |
Voydeya | danikopan | L04AJ09 |
Vyloy | zolbetuksimab | L01FX31 |
Vyndaqel | tafamidismeglumin | N07XX08 |
Vyvgart | efgartigimod alfa | L04AA58 |
Vyxeos liposomal | daunorubicinklorid; citarabin | L01XY01 |
Wakix | pitolizantklorid | N07XX11 |
Waylivra | volanesorsen natrij | C10AX18 |
Winrevair | sotatercept | C02KX06 |
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 |
Zynyz | retifanlimab | L01FF10 |