Drug catalog
268 specialty and rare-disease drugs accessible through Named Patient Program pathways. Alphabetical index.
Each page explains the drug, approval status, typical access pathway, and country-specific guidance.
A
Abecma, idecabtagene vicleucelAdbry, tralokinumab-ldrmAgamree, vamorolone steroid alternativeAkeega, abiraterone + niraparibAlecensaAlhemo, efanesoctocog alfa, factor VIIIAltuviiio, efanesoctocog alfaAlunbrigAlyftrek, pegvaliaseAmondys 45, casimersenAmtagvi, lifileucelAmvuttra, vutrisiranAndembry, garadacimabAnktiva, N-803Anzupgo, delgocitinibAptiom, eslicarbazepine acetateAqneursa, lusutrombopagAttruby, acoramidisAucatzyl, obecabtagene autoleucelAugtyro, repotrectinibAustedoAvmapki Fakzynja Co-Pack, avutometinib and defactinibAwiqli, insulin icodecB
Beqvez, fidanacogene elaparvovecBimzelx, bimekizumab-bkzxBizengri, zenocutuzumab-zbcoBraftoviBreyanzi, lisocabtagene maraleucelBrinsupri, brensocatibBylvay, odevixibatC
Camzyos, mavacamtenCapvaxive, 21-valent pneumococcal conjugate vaccine (V116)Carvykti, ciltacabtagene autoleucelCasgevy, exagamglogene autotemcelCerdelgaCerezymeCobenfy, xanomeline and trospium chlorideColumvi, glofitamabCosentyxCrenessity, crinecerfontCrexont, carbidopa and levodopa extended-releaseD
Datroway, unclear; possibly tirzepatide/otherDawnzera, donidalorsenDaybue, trofinetideDiacomit, stiripentolDopteletDupixentDuvyzat, DMDE
Ebglyss, lebrikizumabEbvallo, tabelecleucelEkterly, sebetralstatElapraseElevidys, delandistrogene moxeparvovecElfabrio, pegunigalsidase alfaElrexfio, elranatamabEmpaveliEmrelis, telisotuzumab vedotin-tllvEmrosi, minocycline extended-release capsulesEncelto, revakinagene taroretcelEndari, l-glutamineEnflonsia, clesrovimab-cforEnhertu, trastuzumab deruxtecanEnjaymoEnsacove, ensartinibEnspryng, satralizumab-mwgeEntrestoEntyvioEohilia, budesonide oral suspensionEpkinly, epcoritamabEvrysdi, risdiplamExondys 51, eteplirsenF
Fabhalta, iptacopanFabrazymeFarxigaFilspari, sparsentanFintepla, fenfluramineForzinity, elamipretideFruzaqla, fruquintinibG
Gattex, teduglutideGavretoGocovriGomekli, mirdametinibGrafapex, treosulfanH
Hemgenix, etranacogene dezaparvovecHemlibra, emicizumab-kxwhHercessi, trastuzumab-strfHernexeos, zongertinibHernexeos-as-zongertinib, zongertinibHyrnuo, sevabertinibI
Ibtrozi, taletrectinibImcivree, setmelanotideImdelltra, tarlatamabImkeldi, imatinib oral solution (80 mg/mL)Impavido, miltefosineIngrezzaInlexzo, gemcitabine intravesical systemInluriyo, imlunestrantIqirvo, elafibranorItovebi, ixazomib classIwilfin, eflornithine tabletsIzervay, avacincaptad pegolJ
JardianceJascayd, nerandomilastJaypirca, pirtobrutinibJoenja, leniolisibJournavx, suzetrigineJylamvo, methotrexate oral solutionK
Kalbitor, ecallantideKerendiaKeveyis, dichlorphenamideKeytruda, pembrolizumabKisunla, donanemabKomzifti, trofinetideKrazati, adagrasibKresladi, marnetegragene autotemcelKrystexxa, pegloticaseL
Leclaza / Lazcluze, lazertinibLenmeldy, atidarsagene autotemcelLeqembi, lecanemabLeqvioLibervant, diazepam buccal filmLitfulo, ritlecitinibLivdelzi, seladelparLivmarli, maralixibatLoqtorzi, PD‑1 inhibitorLorbrenaLunsumio, mosunetuzumab-axgbLuxturna, voretigene neparvovecLyfgenia, lovotibeglogene autotemcelLymphir, denileukin diftitox-cxdlLynozyfic, linvoseltamab-gcptLynparzaM
MayzentMekinistMektoviMepseviiMiebo, perfluorohexyloctane ophthalmic solutionMiplyffa, arimoclomolModeyso, dordaviproneMyhibbin, mycophenolate mofetil oral suspensionMyobloc, rimabotulinumtoxinBN
Neffy, epinephrine nasal sprayNemluvio, nemolizumabNexviazyme, avalglucosidase alfaNgenla, somatrogon-ghlaNiktimvo, axatilimab-csfrNipocalimab (Imaavy), nipocalimabNucalaNuplazidNurtec ODT, rimegepantO
Ogsiveo, seclidemstatOhtuvayre, ensifentrineOjemda, tovorafenibOlumiantOngentysOnpattroOnyda XR, clonidine hydrochloride extended-release oral suspensionOpdivo Qvantig, nivolumab and hyaluronidase-nvhyOpzelura, ruxolitinib creamOrserdu, elacestrantOziltus, denosumab-mobzP
Pemrydi RTU, pemetrexed (ready-to-use injection)Penpulimab-kcqx, penpulimabPiasky / Crovalimab, crovalimabPluvicto, lutetium Lu 177 vipivotide tetraxetanPoherdy, pertuzumab-dpzbPombiliti, cipaglucosidase alfaPraluentPyrukynd, mitapivatQ
Qalsody, SOD1 mutationQfitlia, fitusiranQulipta, atogepantQuzyttir, cetirizine hydrochloride (IV injection)R
Ravicti, glycerol phenylbutyrateReblozylRepathaRetevmoRevuforj, revumenibRezdiffra, resmetiromRezlidhia, olutasidenibRezurock, belumosudilRhapsido, remibrutinibRinvoqRoctavian, valoctocogene roxaparvovecRomvimza, vimseltinibRybrevant, amivantamabRyoncil, remestemcel-LRystiggo, rozanolixizumabRytelo, imetelstatS
Selarsdi, ustekinumab-aeknSiliqSimlandi, adalimumab-ryvkSkyclarys, omaveloxoloneSkyriziSkysona, elivaldogene autotemcelSohonos, palovaroteneSolirisSpinraza, nusinersenSpravato, esketamine (CIII controlled substance)StelaraSyfovre, pegcetacoplanT
TafinlarTagrissoTaltzTalvey, talquetamabTalzennaTavalisseTecartus, brexucabtagene autoleucelTecelra, afamitresgene autoleucelTecvayli, BCMAxCD3 bispecificTegsediTepkinly, epcoritamab-byspTerlivaz, terlipressinTevimbra, PD‑1 inhibitor; tislelizumabTezspireTibsovo, ivosidenibTivdak, tisotumab vedotin-tftvTremfyaTrikaftaTruqap, capivasertibTryngolza, olezarsenTryptyr, acoltremon ophthalmic solutionTryvio, aprocitentanU
Ubrelvy, ubrogepantUltomirisUplizna, inebilizumab-cdonV
Vanrafia, atrasentanVeozah, fezolinetantViltepso, viltolarsenVimizimVistogard, uridine triacetateVizz, aceclidine ophthalmic solution 1.44%Vonvendi, von Willebrand factor (recombinant)Voranigo, vorasidenibVoxzogo, vosoritideVoydeya, danicopanVoyxact, sibeprenlimab-szsiVPRIVVumerityVyjuvek, beremagene geperpavecVykat XR, diazoxide cholineVyloy, zolbetuximabVyndaqelVyondys 53, golodirsenVyvgart, efgartigimodW
Wainua, eplontersenWainzuaWayrilz, rilzabrutinibWelireg, belzutifanWinrevair, sotaterceptX
XadagoXcopri, cenobamateXdemvy, lotilaner ophthalmic solution, 0.25%Xolremdi, bexagliflozin classY
Ycanth, cantharidinYescarta, axicabtagene ciloleucelYeztugo, lenacapavirZ
Zavzpret, zavegepantZegalogue (flagged, monitoring), dasiglucagonZegfrovy, sunvozertinibZevaskyn, prademagene zamikeracelZiihera, zanidatamab-hriiZilbrysq, zilucoplanZolgensma, onasemnogene abeparvovecZtalmy, ganaxoloneZycubo, copper histidinateZynteglo, betibeglogene autotemcelZynyz, retifanlimabReserved for you.