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Table 1 Orphan drugs available in Latvia

From: Rare diseases and orphan drugs: Latvian story

Trade name Active substance Indication Reimbursement
Afinitor* Everolimus Neuroendocrine tumours of pancreatic origin (pNET)  
Renal cell carcinoma (RCC)
Aldurazyme* Laronidase Mucopolysaccharidosis I (MPS I) CCUH
Arzerra Ofatumumab CLL Individual
Atriance Nelarabine T-cell ALL Individual
T-cell lymphoblastic lymphoma
Carbaglu Carglumic acid Hyperammonaemia  
Cystadane Betaine anhydrous Homocystinuria CCUH/Individual
Diacomit Stiripentol Severe myoclonic epilepsy in infancy (Dravet’s syndrome) Individual
Elaprase Idursulfase MPS II (Hunter syndrome) CCUH
Evoltra Clofarabine ALL  
Exjade Deferasirox Beta thalassaemia major with iron overload due to blood transfusion Individual
Gliolan 5-aminolevulinic acid hydrochloride Malignant glioma  
Glivec* Imatinib Ph + CML List A
GIST (previously list C)
Dermatofibrosarcoma protuberans Individual
Ph + ALL
Hypereosinophilic syndrome
Myelodysplastic/ myeloproliferative diseases
Increlex Mecasermin Primary insulin-like growth factor 1 deficiency CCUH
Jakavi Ruxolitinib Myelofibrosis  
Kuvan Sapropterin Phenylketonuria CCUH
Tetrahydrobiopterin deficiency
Litak Cladribine Hairy cell leukaemia  
Mozobil Plerixafor Haematopoietic stem cell transplantation in lymphoma or multiple myeloma patients Individual
Myozyme Alglucosidase alpha Pompe disease  
Nexavar Sorafenib Hepatocellular carcinoma Individual
RCC
Nplate Romiplostim ITP Individual
Orfadin Nitisinone Hereditary tyrosinaemia type 1  
Pedea Ibuprofen Patent ductus arteriosus in preterm newborns  
Peyona Caffeine citrate Primary apnea of premature newborns  
Revatio Sildenafil PAH Individual
Revolade* Eltrombopag ITP Individual
Sprycel Dasatinib Ph + CML List C
Ph + ALL Individual
Sutent* Sunitinib GIST Individual
RCC
pNET
Tasigna Nilotinib Ph + CML List C
Tobi Podhaler Tobramycin Pseudomonas aeruginosa infection in cystic fibrosis patients  
Ventavis* Iloprost PAH  
Volibris Ambrisentan PAH Individual
Votubia Everolimus Renal angiomyolipoma or subependymal giant cell astrocytoma associated with tuberous sclerosis complex  
Wilzin Zinc acetate dihydrate Wilson’s disease Individual
Yondelis Trabectedin Soft tissue sarcoma  
Ovarian cancer
  1. *Afinitor, Aldurazyme, Glivec, Revolade, Sutent and Ventavis are no longer considered to be orphan medicines in the EU.