orladeyo
biocryst ireland limited - berotralstat dihydrochloride - angioedemas, nasljedni - other hematological agents - orladeyo is indicated for routine prevention of recurrent attacks of hereditary angioedema (hae) in adult and adolescent patients aged 12 years and older.
evrysdi
roche registration gmbh - risdiplam - mišićna atrofija, kralješnica - ostali lijekovi za poremećaje mišićno-koštanog sustava - evrysdi is indicated for the treatment of 5q spinal muscular atrophy (sma) in patients with a clinical diagnosis of sma type 1, type 2 or type 3 or with one to four smn2 copies.
pemazyre
incyte biosciences distribution b.v. - pemigatinib - cholangiocarcinoma - antineoplastična sredstva - pemazyre monotherapy is indicated for the treatment of adults with locally advanced or metastatic cholangiocarcinoma with a fibroblast growth factor receptor 2 (fgfr2) fusion or rearrangement that have progressed after at least one prior line of systemic therapy.
sogroya
novo nordisk a/s - somapacitan - rast - hipofiza i hipotalamusni hormoni i analozi - sogroya is indicated for the replacement of endogenous growth hormone (gh) in children aged 3 years and above, and adolescents with growth failure due to growth hormone deficiency (paediatric ghd), and in adults with growth hormone deficiency (adult ghd).
skysona
bluebird bio (netherlands) b.v. - elivaldogene autotemcel - adrenoleukodystrophy - ostali lijekovi protiv živčanog sustava - treatment of early cerebral adrenoleukodystrophy in patients less than 18 years of age, with an abcd1 genetic mutation, and for whom a human leukocyte antigen (hla) matched sibling haematopoietic stem cell donor is not available.
efmody
diurnal europe b.v. - hidrokortizon - adrenal hyperplasia, congenital - kortikosteroidi za sistemsku upotrebu - treatment of congenital adrenal hyperplasia (cah) in adolescents aged 12 years and over and adults.
enspryng
roche registration gmbh - satralizumab - neuromyelitis optica - imunosupresivi - satralizumab (enspryng) is indicated as a monotherapy or in combination with immunosuppressive therapy (ist) for the treatment of neuromyelitis optica spectrum disorders (nmosd) in adult and adolescent patients from 12 years of age who are anti-aquaporin-4 igg (aqp4-igg) seropositive.
voxzogo
biomarin international limited - vosoritide - achondroplasia - lijekovi za liječenje bolesti kostiju - voxzogo is indicated for the treatment of achondroplasia in patients 4 months of age and older whose epiphyses are not closed. the diagnosis of achondroplasia should be confirmed by appropriate genetic testing.
minjuvi
incyte biosciences distribution b.v. - tafasitamab - lymphoma, large b-cell, diffuse - antineoplastična sredstva - minjuvi is indicated in combination with lenalidomide followed by minjuvi monotherapy for the treatment of adult patients with relapsed or refractory diffuse large b-cell lymphoma (dlbcl) who are not eligible for autologous stem cell transplant (asct).
abecma
bristol-myers squibb pharma eeig - idecabtagene vicleucel - multiple myeloma; neoplasms; cancer; neoplasms, plasma cell; hemostatic disorders; vascular diseases; cardiovascular diseases; paraproteinemias; blood protein disorders; hematologic diseases; hemic and lymphatic diseases; hemorrhagic disorders; infectious mononucleosis; lymphoproliferative disorders; immunoproliferative disorders; immune system diseases - antineoplastična sredstva - abecma is indicated for the treatment of adult patients with relapsed and refractory multiple myeloma who have received at least three prior therapies, including an immunomodulatory agent, a proteasome inhibitor and an anti cd38 antibody and have demonstrated disease progression on the last therapy.