Drug Decoder

Look up any rare disease drug by its generic name, brand name, or development code. Side effects, mechanisms, clinical trial results, and pricing context written in plain English.

293
Drugs covered
109
Conditions
220
FDA approved

Browse rare disease drugs by condition

Aceruloplasminemia drugs

DeferasiroxExjadeICL670
Approved
DeferiproneFerriproxL1
Approved

Achondroplasia drugs

NavepegritideYuviwelTransCon CNP
Approved

Acromegaly drugs

OctreotideSandostatinSMS 201-995
Approved
LanreotideSomatuline DepotBIM 23014
Approved
PasireotideSigniforSOM230
Approved
PegvisomantSomavertB2036-PEG
Approved
PaltusotinePalsonifyCRN00808
Approved
Octreotide (oral capsules)Mycapssaoral octreotide capsules
Approved
Octreotide (subcutaneous depot)OclaizCAM2029
Phase 3

Adrenocortical Carcinoma drugs

MitotaneLysodren
Approved
CabozantinibCabometyxXL184
Phase 2
CemiplimabLibtayoREGN2810
Phase 2

Alexander Disease drugs

ZilganersenZanvastroION373
Approved

Alpha-1 Antitrypsin Deficiency drugs

Alpha-1 proteinase inhibitor (human)Prolastin-C
Approved
FazirsiranARO-AAT
Phase 3
BEAM-302BEAM-302
Phase 2
AlvelestatMPH966
Phase 2
Efdoralprin alfaINBRX-101
Phase 2

Amyotrophic Lateral Sclerosis drugs

TofersenQalsodyBIIB067
Approved
RiluzoleRilutek
Approved
EdaravoneRadicavaMCI-186
Approved
JacifusenION363
Phase 3
MecobalaminRozebalamin
Phase 3

Anaplasmosis drugs

DoxycyclineVibramycin
Approved

Aplastic Anemia drugs

EltrombopagPromactaSB-497115
Approved
Antithymocyte globulin (horse)ATGAM
Approved
CyclosporineNeoral
Approved

Aromatic L-Amino Acid Decarboxylase Deficiency drugs

Eladocagene exuparvovec-tneqKebilidiAAV2-hAADC
Approved

Ataxia-Telangiectasia drugs

LevacetylleucineAqneursaIB1001
Approved

ATTR Amyloidosis drugs

PatisiranOnpattroALN-TTR02
Approved
VutrisiranAmvuttraALN-TTRSC02
Approved
TafamidisVyndamaxFx-1006A
Approved
AcoramidisAttrubyAG10
Approved
EplontersenWainuaION-682884
Approved
Nexiguran ziclumeranNTLA-2001
Phase 3

Babesiosis drugs

Atovaquone + ProguanilMalarone
Approved
AtovaquoneMepron
Approved
ClindamycinCleocin
Approved
AzithromycinZithromax
Approved
TafenoquineKrintafelWR 238605
Phase 2

Barth Syndrome drugs

ElamipretideForzinityMTP-131
Approved

Bartonellosis drugs

AzithromycinZithromax
Approved
RifampinRifadin
Approved
DoxycyclineVibramycin
Approved

Behcet Disease drugs

ApremilastOtezlaCC-10004
Approved
AdalimumabHumiraD2E7
Approved
DusquetideSGX945
Phase 2

Beta-Thalassemia drugs

LuspaterceptReblozylACE-536
Approved

C3 Glomerulopathy drugs

IptacopanFabhaltaLNP023
Approved
PegcetacoplanEmpaveliAPL-2
Approved

CDKL5 Deficiency Disorder drugs

GanaxoloneZtalmy
Approved

Cerebral Adrenoleukodystrophy drugs

Elivaldogene autotemcelSkysonaLenti-D
Approved

Cerebrotendinous Xanthomatosis drugs

Chenodiol (chenodeoxycholic acid)Ctexli
Approved

Charcot-Marie-Tooth Disease drugs

GovorestatAT-007
Phase 3
PXT3003 (baclofen / sorbitol / naltrexone)PXT3003
Phase 3
IgnaseclantNMD670
Phase 2
EN001 (mesenchymal stem cell therapy)EN001
Phase 2
DTx-1252DTx-1252
Phase 1

Cholangiocarcinoma drugs

DurvalumabImfinziMEDI4736
Approved
IvosidenibTibsovoAG-120
Approved
ZanidatamabZiiheraZW25
Approved
FutibatinibLytgobiTAS-120
Approved
LirafugratinibLyrfigtuRLY-4008
Approved
PemigatinibPemazyreINCB054828
Approved
PembrolizumabKeytrudaMK-3475
Approved

Chronic Graft-versus-Host Disease drugs

RuxolitinibJakafiINCB018424
Approved
BelumosudilRezurockKD025
Approved
IbrutinibImbruvicaPCI-32765
Approved
AxatilimabNiktimvoSNDX-6352
Approved

Chronic Granulomatous Disease drugs

Interferon gamma-1bActimmune
Approved

Cold Agglutinin Disease drugs

Sutimlimab-jomeEnjaymoBIVV009
Approved

Congenital Adrenal Hyperplasia drugs

CrinecerfontCrenessityNBI-74788
Approved

Congenital Athymia drugs

Allogeneic processed thymus tissue-agdcRethymicRVT-802
Approved

Cushing Disease drugs

OsilodrostatIsturisaLCI699
Approved
MifepristoneKorlymRU-486
Approved
LevoketoconazoleRecorlevCOR-003
Approved
PasireotideSigniforSOM230
Approved

Cystic Fibrosis drugs

Elexacaftor / Tezacaftor / IvacaftorTrikaftaVX-445/VX-661/VX-770
Approved
Vanzacaftor / Tezacaftor / DeutivacaftorAlyftrekVX-121/VX-661/VX-561
Approved
IvacaftorKalydecoVX-770
Approved
Lumacaftor / IvacaftorOrkambiVX-809/VX-770
Approved
Tezacaftor / IvacaftorSymdekoVX-661/VX-770
Approved

Dermatomyositis drugs

BrepocitinibLisrayaPF-06700841
Approved
DazukibartPF-06823859
Phase 3
AnifrolumabSaphneloMEDI-546
Phase 3

Diffuse Intrinsic Pontine Glioma drugs

DordaviproneModeysoONC201
Approved

Dravet Syndrome drugs

StiripentolDiacomit
Approved
CannabidiolEpidiolexGWP42003
Approved
FenfluramineFintepla
Approved
ZorevunersenSTK-001
Phase 3
BexicaserinLP352
Phase 3
Clemizole hydrochlorideEPX-100
Phase 3
ETX101ETX101
Phase 2

Duchenne Muscular Dystrophy drugs

GivinostatDuvyzatITF2357
Approved
Delandistrogene moxeparvovecElevidysSRP-9001
Approved
DeflazacortEmflaza
Approved
EteplirsenExondys 51AVI-4658
Approved
GolodirsenVyondys 53SRP-4053
Approved
ViltolarsenViltepsoNS-065
Approved
CasimersenAmondys 45SRP-4045
Approved
VamoroloneAgamreeVBP15
Approved

Eosinophilic Granulomatosis with Polyangiitis drugs

MepolizumabNucala
Approved
BenralizumabFasenraMEDI-563
Approved

Epidermolysis Bullosa drugs

Beremagene geperpavec-svdtVyjuvekB-VEC
Approved
Prademagene zamikeracelZevaskynEB-101
Approved
Birch triterpenesFilsuvezOleogel-S10
Approved

Fabry Disease drugs

Agalsidase betaFabrazyme
Approved
MigalastatGalafoldAT1001
Approved
Pegunigalsidase alfaElfabrioPRX-102
Approved

Familial Chylomicronemia Syndrome drugs

OlezarsenTryngolzaISIS 678354
Approved
PlozasiranRedemploARO-APOC3
Approved

Familial Hypertrophic Cardiomyopathy drugs

AficamtenMyqorzoCK-3773274
Approved

Fibrodysplasia Ossificans Progressiva drugs

ZilurgisertibAtebriozINCB000928
Approved
Garetosmab-grtsPasatruREGN2477
Approved
PalovaroteneSohonos
Approved

Focal Epilepsy drugs

LevetiracetamKeppraUCB L059
Approved
LamotrigineLamictal
Approved
LacosamideVimpatSPM 927
Approved
BrivaracetamBriviactUCB 34714
Approved
PerampanelFycompaE2007
Approved
CenobamateXcopriYKP3089
Approved
AzetukalnerXEN1101
Phase 3
OpakalimBHV-7000
Clinical Hold

Focal Segmental Glomerulosclerosis drugs

SparsentanFilspariTSP-0005
Approved

Gaucher Disease drugs

EliglustatCerdelgaGENZ-112638
Approved
ImigluceraseCerezyme
Approved
Taliglucerase alfaElelysoprGCD
Approved
MiglustatZavescaOGT 918
Approved
Velaglucerase alfaVPRIVGene-Activated GCB
Approved
VenglustatGZ402671
Phase 3

Generalized Pustular Psoriasis drugs

ImsidolimabANB019
Phase 3

Geographic Atrophy drugs

Avacincaptad pegolIzervayARC1905
Approved

Giant Cell Arteritis drugs

UpadacitinibRinvoqABT-494
Approved
TocilizumabActemra
Approved

Graves' Disease drugs

BHV-1300BHV-1300
Phase 3

Hemophilia A drugs

ConcizumabAlhemoNN7415
Approved
MarstacimabHympavziPF-06741086
Approved
EmicizumabHemlibraACE910
Approved
FitusiranQfitliaALN-AT3
Approved
DenecimigMim8
Phase 3
Valoctocogene roxaparvovecRoctavianBMN 270
Discontinued

Hemophilia B drugs

ConcizumabAlhemoNN7415
Approved
MarstacimabHympavziPF-06741086
Approved
FitusiranQfitliaALN-AT3
Approved
Etranacogene dezaparvovecHemgenixAMT-061
Approved
Fidanacogene elaparvovecBeqvezPF-07055480
Discontinued

Heparin-Induced Thrombocytopenia drugs

CAD-1005CAD-1005
Phase 2

Hereditary Angioedema drugs

LanadelumabTakhzyroDX-2930
Approved
BerotralstatOrladeyoBCX7353
Approved
GaradacimabAndembryCSL312
Approved
SebetralstatEkterlyKVD900
Approved
DonidalorsenDawnzeraIONIS-PKK-LRx
Approved

Hunter Syndrome drugs

Tividenofusp alfaAvlayahDNL310
Approved
IdursulfaseElaprase
Approved

Huntington Disease drugs

DeutetrabenazineAustedoSD-809
Approved
TetrabenazineXenazine
Approved
ValbenazineIngrezzaNBI-98854
Approved

Hypertrophic Cardiomyopathy drugs

MavacamtenCamzyosMYK-461
Approved

Hypoparathyroidism drugs

PalopegteriparatideYorvipathTransCon PTH
Approved

Idiopathic Pulmonary Fibrosis drugs

NerandomilastJascaydBI 1015550
Approved
NintedanibOfevBIBF 1120
Approved
PirfenidoneEsbriet
Approved
AdmilparantBMS-986278
Phase 3

IgA Nephropathy drugs

Budesonide (delayed-release)Tarpeyo
Approved
SparsentanFilspariTSP-0005
Approved
IptacopanFabhaltaLNP023
Approved
AtrasentanVanrafia
Approved
AtaciceptTrutaknaTACI-Ig
Approved
SibeprenlimabVoyxact
Approved
FelzartamabMOR202
Phase 3
ZigakibartBION-1301
Phase 3
PovetaciceptALPN-303
Phase 3
SefaxersenRO7434656
Phase 3
RavulizumabUltomirisALXN1210
Phase 3
BHV-1400BHV-1400
Phase 1

IgG4-Related Disease drugs

InebilizumabUpliznaMEDI-551
Approved

Immune Thrombocytopenia drugs

RilzabrutinibWayrilzPRN1008
Approved

Leber Congenital Amaurosis drugs

Voretigene neparvovec-rzylLuxturnaAAV2-hRPE65v2
Approved

Light Chain Amyloidosis drugs

Daratumumab and hyaluronidase-fihjDarzalex Faspro
Approved

MCT8 Deficiency drugs

TiratricolEmcitateTriac
Approved

Membranous Nephropathy drugs

BHV-1420BHV-1420
Preclinical

Menkes Disease drugs

Copper histidinateZycuboCUTX-101
Approved

Merkel Cell Carcinoma drugs

Retifanlimab-dlwrZynyzINCMGA00012
Approved

Metachromatic Leukodystrophy drugs

Atidarsagene autotemcelLenmeldyOTL-200
Approved

Multiple Myeloma drugs

Anitocabtagene autoleucelanito-cel
Phase 3
MezigdomideCC-92480
Phase 3

Multiple Sclerosis drugs

UblituximabBriumviTG1101
Approved
FingolimodGilenyaFTY720
Approved
Glatiramer acetateCopaxoneCopolymer 1
Approved
TeriflunomideAubagioHMR1726
Approved
OzanimodZeposiaRPC1063
Approved
OcrelizumabOcrevus
Approved
NatalizumabTysabri
Approved
OfatumumabKesimpta
Approved
Dimethyl fumarateTecfideraBG-12
Approved
SiponimodMayzent
Approved
Fenebrutinib
Phase 3
FrexalimabSAR441344
Phase 3
TolebrutinibSAR442168
Not Approved (CRL)

Myasthenia Gravis drugs

EculizumabSolirish5G1.1
Approved
Efgartigimod alfaVyvgartARGX-113
Approved
RozanolixizumabRystiggoUCB7665
Approved
ZilucoplanZilbrysqRA101495
Approved
RavulizumabUltomirisALXN1210
Approved
InebilizumabUpliznaMEDI-551
Approved
NipocalimabImaavyM281
Approved
BHV-1450BHV-1450
Preclinical

Myelodysplastic Syndromes drugs

ImetelstatRyteloGRN163L
Approved

Myelofibrosis drugs

PacritinibVonjoSB1518
Approved
MomelotinibOjjaaraCYT387
Approved

Myotonic Dystrophy drugs

Zeleciment basivarsenDYNE-101
Phase 3
Delpacibart etedesiranAOC 1001
Missed Endpoint

Narcolepsy Type 1 drugs

OveporextonOrzeyfulTAK-861
Approved

Neurofibromatosis Type 1 drugs

SelumetinibKoselugoAZD6244
Approved
MirdametinibGomekliPD-0325901
Approved

Neuromyelitis Optica Spectrum Disorder drugs

SatralizumabEnspryngSA237
Approved

Niemann-Pick Disease drugs

ArimoclomolMiplyffa
Approved

Niemann-Pick Disease Type C drugs

ArimoclomolMiplyffa
Approved
LevacetylleucineAqneursaIB1001
Approved

NTRK Fusion Cancer drugs

LarotrectinibVitrakviLOXO-101
Approved
EntrectinibRozlytrekRXDX-101
Approved
RepotrectinibAugtyroTPX-0005
Approved
EratrectinibVelmarto (China)VC004
Phase 3
TL118TL118
Phase 2
ZurletrectinibICP-723
Phase 2
ANS03ANS03
Phase 1

Paraganglioma drugs

BelzutifanWeliregMK-6482
Approved

Paroxysmal Nocturnal Hemoglobinuria drugs

CrovalimabPiaskyRG6107
Approved
DanicopanVoydeyaALXN2040
Approved
IptacopanFabhaltaLNP023
Approved

Pemphigus Vulgaris drugs

BHV-1450BHV-1450
Preclinical

Peripartum Cardiomyopathy drugs

BHV-1600BHV-1600
Phase 1

Phenylketonuria drugs

SepiapterinSephiencePTC923
Approved

Pheochromocytoma drugs

BelzutifanWeliregMK-6482
Approved

Polycythemia Vera drugs

RusfertideMimryloPTG-300
Approved

Pompe Disease drugs

Avalglucosidase alfaNexviazymeneoGAA
Approved
Alglucosidase alfaLumizyme
Approved
Cipaglucosidase alfa + miglustatPombiliti + OpfoldaATB200/AT2221
Approved

Prader-Willi Syndrome drugs

Diazoxide cholineVykat XRDCCR
Approved

Primary Biliary Cholangitis drugs

SeladelparLivdelziMBX-8025
Approved
ElafibranorIqirvoGFT505
Approved
LinerixibatLynavoyGSK2330672
Approved

Primary Central Nervous System Lymphoma drugs

TirabrutinibVelexbru (Japan)ONO-4059
Phase 3

Primary Hyperoxaluria drugs

NedosiranRivflozaDCR-PHXC
Approved

Pulmonary Alveolar Proteinosis drugs

MolgramostimMolbreevi (proposed name)
Phase 3

Pulmonary Arterial Hypertension drugs

EpoprostenolFlolan
Approved
IloprostVentavis
Approved
BosentanTracleerRo 47-0203
Approved
RiociguatAdempasBAY 63-2521
Approved
SotaterceptWinrevairACE-011
Approved
TreprostinilTyvaso
Approved
Treprostinil inhalation powderYutrepiaLIQ861
Approved
SelexipagUptraviACT-293987
Approved
MacitentanOpsumitACT-064992
Approved
AmbrisentanLetairisBSF-208075
Approved
SildenafilRevatio
Approved
TadalafilAdcirca
Approved

Recurrent Respiratory Papillomatosis drugs

Zopapogene imadenovecPapzimeosPRGN-2012
Approved
INO-3107INO-3107
Phase 2

Retinitis Pigmentosa drugs

Voretigene neparvovec-rzylLuxturnaAAV2-hRPE65v2
Approved

Rett Syndrome drugs

TrofinetideDaybueNNZ-2566
Approved
BlarcamesineANAVEX 2-73
Phase 3
NGN-401NGN-401
Phase 2
TSHA-102TSHA-102
Phase 2

Sanfilippo Syndrome drugs

Rebisufligene etisparvovecFayuviUX111
Approved

SCN2A Encephalopathy drugs

RelutriginePRAX-562
Phase 3
ElsunersenPRAX-222
Phase 3

SCN8A Encephalopathy drugs

RelutriginePRAX-562
Phase 3

Sickle Cell Disease drugs

Exagamglogene autotemcelCasgevyCTX001
Approved
Lovotibeglogene autotemcelLyfgeniaLentiGlobin BB305
Approved
HydroxyureaDroxia
Approved
CrizanlizumabAdakveoSEG101
Approved
L-glutamineEndari
Approved

Sjögren's Syndrome drugs

PilocarpineSalagen
Approved
CevimelineEvoxacSNI-2011
Approved
IanalumabVAY736
Phase 3
DeucravacitinibSotyktuBMS-986165
Phase 3
TelitaciceptRC18
Phase 3
DazodalibepHZN-4920
Phase 3

Spinal Muscular Atrophy drugs

ApitegromabIsembyldSRK-015
Approved
NusinersenSpinrazaISIS-396443
Approved
Onasemnogene abeparvovecZolgensmaAVXS-101
Approved
Onasemnogene abeparvovec-brveItvismaOAV101 IT
Approved
RisdiplamEvrysdiRG7916
Approved
Taldefgrobep alfaRO7239361
Missed Endpoint

Spinocerebellar Ataxia drugs

TroriluzoleVyglxiaBHV-4157
Not Approved (CRL)

Systemic Juvenile Idiopathic Arthritis drugs

CanakinumabIlarisACZ885
Approved
TocilizumabActemra
Approved
AnakinraKineret
Approved
EmapalumabGamifantNI-0501
Approved

Systemic Sclerosis drugs

NintedanibOfevBIBF 1120
Approved
TocilizumabActemra
Approved

Thalassemia drugs

Betibeglogene autotemcelZyntegloLentiGlobin BB305
Approved
Exagamglogene autotemcelCasgevyCTX001
Approved

Thalassemia (alpha and beta) drugs

MitapivatAqvesmeAG-348
Approved

Thymidine Kinase 2 Deficiency drugs

Doxecitine and doxribtimineKygevviMT1621
Approved

Thyroid Eye Disease drugs

VeligrotugLumvoaVRDN-001
Approved
TeprotumumabTepezzaRV 001
Approved

Vascular Ehlers-Danlos Syndrome drugs

CeliprololEdsivo
Phase 3

Wild-Type ATTR Cardiac Amyloidosis drugs

VutrisiranAmvuttraALN-TTRSC02
Approved
TafamidisVyndamaxFx-1006A
Approved
AcoramidisAttrubyAG10
Approved
Nexiguran ziclumeranNTLA-2001
Phase 3

Wilson Disease drugs

Trientine tetrahydrochlorideCuvrior
Approved
Trientine hydrochlorideSyprine
Approved
PenicillamineCuprimine
Approved
Zinc acetateGalzin
Approved
UX701UX701
Phase 3
Bis-choline tetrathiomolybdateALXN1840
Phase 3
VTX-801VTX-801
Phase 2

Wiskott-Aldrich Syndrome drugs

Etuvetidigene autotemcelWaskyraOTL-103
Approved

Drug information is sourced from public databases and may not reflect the most recent updates. This page is for informational purposes only.