Table 1. Orphan drugs available in Latvia

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
T-cell lymphoblastic lymphoma / Individual
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
GIST
Dermatofibrosarcoma protuberans
Ph+ ALL
Hypereosinophilic syndrome
Myelodysplastic/ myeloproliferative diseases / List A
(previously list C)
Individual
Increlex / Mecasermin / Primary insulin-like growth factor 1 deficiency / CCUH
Jakavi / Ruxolitinib / Myelofibrosis
Kuvan / Sapropterin / Phenylketonuria
Tetrahydrobiopterin deficiency / CCUH
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
RCC / Individual
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
Ph+ ALL / List C
Individual
Sutent* / Sunitinib / GIST
RCC
pNET / Individual
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

*Afinitor, Aldurazyme, Glivec, Revolade, Sutent and Ventavis are no longer considered to be orphan medicines in the EU.