Drug catalog

198 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 vicleucel Agamree — vamorolone steroid alternative Akeega — abiraterone + niraparib Alhemo — efanesoctocog alfa, factor VIII Alyftrek — pegvaliase Amtagvi — lifileucel Amvuttra — vutrisiran Andembry — garadacimab Anktiva — N-803 Anzupgo — delgocitinib Aqneursa — lusutrombopag Attruby — acoramidis Aucatzyl — obecabtagene autoleucel Augtyro — repotrectinib Avmapki Fakzynja Co‑Pack — vemurafenib + cobimetinib Awiqli — insulin icodec

B

Beqvez — fidanacogene elaparvovec Bimzelx — bimekizumab Bizengri — roxadustat class Breyanzi — lisocabtagene maraleucel Brinsupri — darolutamide class Brixadi — buprenorphine ER Bylvay — odevixibat

C

Camzyos — mavacamten Capvaxive — PCV21 Carvykti — ciltacabtagene autoleucel Casgevy — exagamglogene autotemcel Cobenfy — isavuconazonium class Columvi — glofitamab Crenessity — crinecerfont Crexont — carbidopa/levodopa ER

D

Datroway — unclear; possibly tirzepatide/other Dawnzera — donidalorsen Daybue — trofinetide Duvyzat — DMD

E

Ebglyss — lebrikizumab Ebvallo — tabelecleucel Ekterly — RA, psoriasis Ekterly — sebetralstat Elevidys — delandistrogene moxeparvovec Elfabrio — pegunigalsidase alfa Elrexfio — elranatamab Emrelis — empagliflozin class Emrelis — telisotuzumab vedotin-tllv Emrosi — dapagliflozin 40 mg Encelto — revakinagene taroretcel Enflonsia — fentanyl transdermal class Enhertu — trastuzumab deruxtecan Ensacove — ensitrelvir Eohilia — budesonide oral suspension Epkinly — epcoritamab Evrysdi — risdiplam

F

Fabhalta — iptacopan Filspari — sparsentan Forzinity — elamipretide Fruzaqla — fruquintinib

G

Gattex — teduglutide Gomekli — goserelin class Grafapex — antibody candidate

H

Hemgenix — etranacogene dezaparvovec Hercessi — trastuzumab-pkrb Hernexeos — daridorexant class Hernexeos-as-zongertinib — zongertinib Hyrnuo — sevabertinib

I

Ibtrozi — BTK inhibitor class Imcivree — POMC/LEPR/PCSK1 deficiency Imdelltra — tarlatamab Imkeldi — imatinib oral solution Impavido — miltefosine Inlexo Kit — methotrexate autoinjector Inluriyo — IL‑23 mAb class Iqirvo — resmetirom Itovebi — ixazomib class Iwilfin — eflornithine tablets Izervay — avacincaptad pegol

J

Jascayd — nerandomilast Jaypirca — pirtobrutinib Joenja — leniolisib Journavx — suzetrigine Jylamvo — methotrexate oral solution

K

Kalbitor — ecallantide Keveyis — dichlorphenamide Keytruda — PD‑1 inhibitor Kisunla — donanemab Komzifti — ziftomenib Krazati — adagrasib Kresladi — marnetegragene autotemcel Krystexxa — pegloticase

L

Leclaza / Lazcluze — lazertinib Lenmeldy — atidarsagene autotemcel Leqembi — lecanemab Libervant — diazepam buccal film Litfulo — ritlecitinib Livdelzi — seladelpar Livmarli — maralixibat Loqtorzi — PD‑1 inhibitor Lunsumio — CD20xCD3 bispecific Luxturna — voretigene neparvovec Lyfgenia — lovotibeglogene autotemcel Lymphir — denileukin diftitox-cxdl Lynozyfic — linvoseltamab-gcpt

M

Miebo — perfluorohexyloctane Miplyffa — pemafibrate Miplyffa — arimoclomol Modeyso — danuglipron class Modeyso — dordaviprone Myhibbin — nintedanib class Myobloc — botulinum toxin B

N

Neffy — intranasal epinephrine alternative – nalmefene listing provided; verify Nemluvio — nemolizumab Nexviazyme — avalglucosidase alfa Ngenla — somatrogon Niktimvo — niraparib + bevacizumab class Nipocalimab (Imaavy) — nipocalimab

O

Ogsiveo — seclidemstat Ohtuvayre — ensifentrine Ojemda — tovorafenib Onyda XR — amphetamine class Opdivo Qvantig — nivolumab Opzelura — ruxolitinib cream Orserdu — elacestrant Oziltus — denosumab-mobz

P

Pemrydi RTU — pemetrexed RTU Penpulimab-kcqx — penpulimab Piasky / Crovalimab — crovalimab Poherdy — pertuzumab-dpzb Pombiliti — cipaglucosidase alfa Pyrukynd — mitapivat

Q

Qalsody — SOD1 mutation Qfitlia — fitusiran Quzyttir — IV cetirizine

R

Ravicti — glycerol phenylbutyrate Revuforj — EPO/roxadustat class Revuforj — revumenib Rezdiffra — resmetirom Rezlidhia — olutasidenib Rezurock — belumosudil Rhapsido — vemurafenib class Roctavian — valoctocogene roxaparvovec Romvimza — TPO receptor agonist class Rybrevant — amivantamab Ryoncil — remestemcel-L Rystiggo — rozanolixizumab Rytelo — imetelstat

S

Selarsdi — seladelpar Simlandi — adalimumab‑ryvk biosimilar Skyclarys — omaveloxolone Skysona — elivaldogene autotemcel Sohonos — palovarotene Spinraza — nusinersen Spravato — esketamine nasal Syfovre — pegcetacoplan

T

Talvey — talquetamab Tecartus — brexucabtagene autoleucel Tecelra — afamitresgene autoleucel Tecvayli — BCMAxCD3 bispecific Tepkinly — epcoritamab Terlivaz — terlipressin Tevimbra — PD‑1 inhibitor; tislelizumab Tibsovo — ivosidenib Tivdak — tisotumab vedotin-tftv Truqap — capivasertib Tryngolza — olezarsen Tryptyr — amitriptyline ER class Tryvio — tebipenem pivoxil hydrobromide

V

Vanrafia — atrasentan Veozah — fezolinetant Vistogard — uridine triacetate Vizz Opth Sol 1.44% — unverified Vonvendi — recombinant vWF Voranigo — vorasidenib Voxzogo — vosoritide Voyxact — sibeprenlimab-szsi Vyjuvek — beremagene geperpavec Vykat XR — diazoxide choline Vyloy — zolbetuximab Vyondys 53 — golodirsen Vyvgart — efgartigimod

W

Wainua — eplontersen Wayrilz — elagolix/linzagolix class Welireg — belzutifan Winrevair — sotatercept

X

Xdemvy — lotilaner ophthalmic 0.25%Xolremdi — bexagliflozin class

Y

Ycanth — cantharidin Yescarta — axicabtagene ciloleucel Yeztugo — lenacapavir

Z

Zavzpret — zavegepant nasal spray Zegalogue (flagged — monitoring) — dasiglucagon Zegfrovy — sunvozertinib Zevaskyn — prademagene zamikeracel Ziihera — TKI class Zilbrysq — zilucoplan Zolgensma — onasemnogene abeparvovec Ztalmy — ganaxolone Zycubo — copper histidinate Zynteglo — betibeglogene autotemcel Zynyz — retifanlimab

AI disclosure & medical review. Content on this page is reviewed by Reserve Meds's AI Pharmacist and AI Regulatory Counsel. A US-licensed human pharmacist (dispensed by Altima Care, license: {{PHARMACY_LICENSE_NO}}) reviews every prescription before dispensing. Regulatory posture is AI-summarized and not legal advice; case-specific questions route to retained outside counsel. Last medically reviewed: {{REVIEWED_DATE}}.