Accrufer
ferric maltol · Iron replacementRadiolabeled iron studies in rats (confirmed in dogs): iron transferred to transferrin and ferritin; literature on iron
Ztalmy: animal efficacy model or human cells?
ganaxolone · GABA-A receptor (positive modulator)
Efficacy evidence
Traditional rodent seizure models (chemically induced convulsions, corneal kindling); not tested in a Cdkl5 disease model
“GNX (IP or oral) was active in several traditional rodent seizure models (eg, chemically-induced convulsions, corneal kindling). GNX was not tested for activity in a Cdkl5 disease model.”
Why that model
Cdkl5 knockout mouse models do not exhibit overt seizures
“While Cdkl5 knockout mouse models have been generated, these do not exhibit overt seizures.”
Reviewer's view of the efficacy data
EMA: proof of concept that ganaxolone protects against convulsions adequately demonstrated in several seizure models
“GNX demonstrated potent, broad, GABAA-mediated anticonvulsant activity in several types of seizure models tested. The proof of concept that GNX is protective of convulsion is adequately demonstrated.”
Same question, other small molecules
Adquey
difamilast · PDE4 (topical)Mouse model of chronic contact dermatitis; in vitro PDE-4 inhibition and cytokine suppression in inflammatory cells
Agamree
vamorolone · Glucocorticoid receptor (dissociative)mdx mouse (dystrophin-deficient) proof-of-concept studies with vamorolone
Alyftrek
vanzacaftor, tezacaftor and deutivacaftor · CFTRCF patient-derived HBE cells (F508del/F508del, F508del/MF): increased F508del-CFTR processing and trafficking
Anzupgo
delgocitinib · Pan-JAK (topical)Rat DNCB-induced dermatitis model (topical): dose-dependent suppression of ear swelling
Aqneursa
levacetylleucine · Neuronal metabolism (proposed)Published data: Npc1-/- CHO cells, NPC patient fibroblasts and Npc1-/- mice
Artesunate
artesunate · Plasmodium (antimalarial)Malaria infection models: P. berghei rodents, P. falciparum-infected immunocompromised mice, Plasmodium-infected nonhuman primates
Attruby
acoramidis · Transthyretin (stabiliser)TTR stabilisation measured in serum from acoramidis-dosed dogs and monkeys (in vivo pharmacodynamics)
Augtyro
repotrectinib · ROS1/TRKMouse subcutaneous allograft/xenograft tumors (engineered NIH/3T3 or Ba/F3 cells expressing ROS1 mutants/fusions)
Avmapki Fakzynja Co-pack
avutometinib and defactinib · RAF/MEK clamp; FAKLGSOC patient-derived xenograft mice: avutometinib plus FAK-inhibitor surrogate VS-4718
Ayvakit
avapritinib · KIT/PDGFRAMouse P815 mastocytoma (KIT exon 17) model and imatinib-resistant GIST patient-derived xenograft (KIT exon 11/17)
Azstarys
serdexmethylphenidate and dexmethylphenidate · Dopamine/norepinephrine reuptakeIn vitro radioligand binding only: SDX showed no significant binding to human DAT, NET or SERT
Balversa
erdafitinib · FGFR1-4Human tumor cell-line xenografts with FGFR1, 2 and 3 alterations (bladder, lung, GI, liver) in immune-deficient rodents
Barhemsys
amisulpride · Dopamine D2/D3 receptorFerret emesis models (apomorphine-, morphine- and cisplatin-induced)
Baxfendy
baxdrostat · Aldosterone synthase (CYP11B2)Telemetered cynomolgus monkeys on low-sodium diet plus furosemide (maximal RAAS activation)
Beqalzi
sonrotoclax · BCL-2MAVER-1 mantle cell lymphoma subcutaneous xenograft in immunocompromised mice
Blujepa
gepotidacin · Bacterial topoisomerases (gyrase/topo IV)Neutropenic murine thigh infection models (Enterobacterales, S. aureus); in vitro E. coli chemostat model
Brenzavvy
bexagliflozin · SGLT2Diabetic db/db mouse and ZDF rat models: dose-dependent HbA1c reduction
Brexafemme
ibrexafungerp · Fungal glucan synthaseIn vitro antifungal activity against Candida species (MIC90 0.015-4 ug/mL)
Brinsupri
brensocatib · DPP1 (cathepsin C)LPS-induced acute lung injury model (no established NCFB model)
Brukinsa
zanubrutinib · BTKREC-1 mantle cell lymphoma subcutaneous xenograft in mice; dose-dependent growth suppression
Byfavo
remimazolam · GABA-A receptor (benzodiazepine site)Sedation in normal animals (rodents, monkeys), reversed by flumazenil; GABA-A benzodiazepine-site binding in vitro
Bylvay
odevixibat · IBATMdr2-/- mouse cholestasis model: reduced liver weight and serum markers of liver damage and cholestasis
Cabenuva
cabotegravir and rilpivirine · HIV-1 integrase; NNRTIIn vitro antiviral activity in human MT4 T-cell line and HIV-1 primary isolates (rilpivirine)
Camzyos
mavacamten · Cardiac myosinTransgenic troponin T-mutant HCM mice dosed 6 months: improved diastolic function, preserved fractional shortening
Caplyta
lumateperone · 5-HT2A / D2 receptorRodent models of antipsychotic activity: head twitch, conditioned avoidance, amphetamine-induced hyperlocomotion
Cardamyst
etripamil · L-type calcium channelIsolated rabbit hearts, spontaneously hypertensive rats and normotensive rabbits (BP, AV conduction, ECG)
Cibinqo
abrocitinib · JAK1Adjuvant-induced arthritis rat model (paw swelling; ex vivo STAT phosphorylation)
Cobenfy
xanomeline and trospium · Muscarinic M1/M4 agonist; peripheral antagonistRodent and monkey models of schizophrenia: conditioned avoidance, psychostimulant-induced hyperactivity, schedule-controlled behavior
Cosela
trilaciclib · CDK4/6In vivo mouse studies of protection from chemotherapy-induced myelosuppression and of tumor protection
Crenessity
crinecerfont · CRF1 receptorIn vitro CRF1 binding, cAMP and ACTH assays; CRF- and stress-induced ACTH inhibition in normal rats
Cypsedo
cipepofol · GABA-A receptorAnesthesia in normal rats, dogs and minipigs, similar depth to propofol with 6-fold higher potency
Daybue
trofinetide · IGF-1 tripeptide analogueMeCP2 knockout mouse model of Rett syndrome (20 mg/kg IP daily, 5 or 20 weeks)
Dayvigo
lemborexant · Orexin OX1/OX2 receptorsIncreased total sleep time in normal mice and rats (ED50 1 and 4.4 mg/kg)
Defencath
taurolidine and heparin · Antimicrobial catheter lockIn vitro antimicrobial activity of taurolidine against a panel of microorganisms
Dojolvi
triheptanoin · Anaplerotic energy substratePublished studies: VLCAD-/- knockout mice fed triheptanoin diet (cardiac function), plus GLUT-1 KO and seizure mouse models
Duvyzat
givinostat · Histone deacetylase (class I/II)B10.mdx (mild) and D2.mdx (severe) mouse models of DMD; DMD patient-derived fibroblasts
Egaten
triclabendazole · Fasciola (tubulin)Published studies in animals experimentally infected with F. hepatica or F. gigantica; in vitro fluke motility and tegument assays
Ekterly
sebetralstat · Plasma kallikreinCarrageenan-induced paw oedema in Sprague-Dawley rats (non-HAE acute inflammation model)
Ensacove
ensartinib · ALKH3122 (EML4-ALK) human NSCLC xenograft in nude mice
Evrysdi
risdiplam · SMN2 splicingSMNΔ7 and C/C SMA mouse models carrying the human SMN2 transgene
Exblifep
cefepime and enmetazobactam · PBPs; beta-lactamase (enmetazobactam)Neutropenic murine thigh infection model (ESBL K. pneumoniae) and hollow-fiber infection model
Exkivity
mobocertinib · EGFR exon 20 insertionPatient-derived NSCLC xenograft LU0387 (EGFR exon 20 insertion) in immunocompromised mice
Exxua
gepirone · 5-HT1A receptorActive in animal models predictive of antidepressant and anxiolytic activity
Fabhalta
iptacopan · Complement factor BPNH patient erythrocytes in vitro: inhibition of hemolysis and C3 deposition
Fetroja
cefiderocol · Penicillin-binding proteinsMurine thigh and lung infection models with Gram-negative strains (dose-ranging, PK/PD target)
Fexinidazole
fexinidazole · Trypanosoma bruceiAcute and chronic murine T. brucei infection models of HAT
Filspari
sparsentan · Endothelin A / angiotensin II receptorsgddY and passive (EIC-induced) mouse IgAN models; anti-Thy-1 rat mesangioproliferative glomerulonephritis model
Fotivda
tivozanib · VEGFR1-3Human and murine tumor xenografts in mice and rats (tumor growth inhibition, anti-angiogenesis)
Foundayo
orforglipron · GLP-1 receptor (non-peptide)Cynomolgus monkeys and humanized GLP-1R knock-in (Glp1rS33W) rats, including diet-induced obese
Fruzaqla
fruquintinib · VEGFR1-3Human tumor xenografts in BALB/c nude mice (BGC-823, HT-29, Caki-1, NCI-H460)
Gavreto
pralsetinib · RETRET-mutant and patient-derived RET-fusion (NSCLC, CRC) xenografts in mice
Gemtesa
vibegron · β3-adrenergic receptorAcetic acid-induced bladder overactivity model (rat)
Gomekli
mirdametinib · MEK1/2Genetically engineered Nf1fl/fl;Dhh-Cre neurofibroma mouse model
Grafapex
treosulfan · DNA alkylationMurine allogeneic bone marrow transplant model (myeloablation, immunosuppression)
Hernexeos
zongertinib · HER2 (mutant)Mice implanted with patient-derived lung cancer cells (ST-3107, HER2 exon 20-YVMA): dose-dependent tumor growth inhibition
Hyrnuo
sevabertinib · HER2 (mutant)NSCLC patient-derived xenograft models with HER2 exon 20 insertion YVMA: antitumor activity
Ibsrela
tenapanor · NHE3Stress-induced IBS-C rat model, mouse opioid-induced constipation model, neonatal acetic-acid IBS-like rat model
Ibtrozi
taletrectinib · ROS1Subcutaneous ROS1-fusion xenografts (Ba/F3, NSCLC) and intracranial patient-derived NSCLC xenograft in mice
Idvynso
doravirine and islatravir · NNRTI; NRTTIIslatravir anti-HIV-1 activity in human PBMCs (IC50 0.21 nM)
Inluriyo
imlunestrant · Estrogen receptor (SERD)ER+ breast cancer xenograft and PDX mouse models, including ESR1-Y537S mutant PDX
Inpefa
sotagliflozin · SGLT1/SGLT2Metabolic rat model of HFpEF: improved diastolic dysfunction markers (LVEDP, left atrial remodeling, IVRT)
Inqovi
decitabine and cedazuridine · DNMT (decitabine); cytidine deaminase (cedazuridine)L1210 and KG-1 disseminated leukemia mouse models (oral cedazuridine plus oral decitabine)
Inrebic
fedratinib · JAK2Mouse models of JAK2V617F-driven myeloproliferative disease: survival up to 100% at 42 days; reduced WBC, hematocrit, splenomegaly, fibrosis
Iqirvo
elafibranor · PPARα/δNASH in db/db mice on MCD diet and chemically induced (CCl4) liver fibrosis in rats; no PBC model
Isturisa
osilodrostat · 11β-hydroxylase (CYP11B1)In vitro human CYP11B1 inhibition; cortisol synthesis inhibition in ACTH-stimulated normal monkeys
Itovebi
inavolisib · PI3Kα (mutant-selective)PIK3CA-mutant human breast cancer xenografts in mice (KPL-4, HCC1954X1, MCF7)
Jascayd
nerandomilast · PDE4BBleomycin lung fibrosis in mice and rats; IPF patient lung fibroblasts in vitro
Jaypirca
pirtobrutinib · BTK (non-covalent)Mouse xenograft models of lymphoma with WT or C481S mutant BTK
Jesduvroq
daprodustat · HIF prolyl hydroxylaseNormal Sprague-Dawley rats: dose-related increases in RBC, hemoglobin and hematocrit
Jideytro
zidesamtinib · ROS1Patient-derived xenograft (PDX) mouse models of ROS1-fusion NSCLC (SDC4-ROS1, CD74-ROS1 G2032R)
Joenja
leniolisib · PI3KδPik3cd E1020K/+ mouse model of APDS; rat TDAR and induced inflammation models
Journavx
suzetrigine · NaV1.8Primary human DRG neurons in vitro (NaV1.8 current and action potential firing inhibition); no in vivo model
Kerendia
finerenone · Mineralocorticoid receptorAnimal models of heart failure and chronic kidney disease: heart and kidney protection
Klisyri
tirbanibulin · Tubulin / SrcCultured keratinocytes in vitro: proliferation reduced 50% at 40 nM
Komzifti
ziftomenib · Menin-KMT2AAML xenografts (MV4-11, MOLM-13 cell lines and patient-derived cells) in immunocompromised mice and rats
Koselugo
selumetinib · MEK1/2Nf1fl/fl;DhhCre neurofibroma knockout mouse: reduced nerve root size and tumor number/size
Krazati
adagrasib · KRAS G12CKRAS G12C-mutant human tumor xenografts in mice: activity in 14 of 17 lung xenograft models, none in G12C-negative models
Kygevvi
doxecitine and doxribtimine · Pyrimidine nucleoside replacement (TK2 deficiency)Tk2 knock-in (Tk2KI) and knockout (Tk2KO) mouse models of TK2 deficiency
Lampit
nifurtimox · Trypanosoma cruziAcute and chronic T. cruzi infection models in mice, rats, hamsters, guinea pigs, rabbits and dogs
Lazcluze
lazertinib · EGFR (mutant-selective)Human NSCLC xenografts (EGFR del19, L858R/T790M) in nude mice, including intracranial model
Leqselvi
deuruxolitinib · JAK1/JAK2In vitro kinase assays and IL-6-stimulated STAT3 inhibition, benchmarked against ruxolitinib
Lifyorli
relacorilant · Glucocorticoid receptor (antagonist)Mouse xenografts of human ovarian, endometrial and cervical cancer with relacorilant plus paclitaxel
Litfulo
ritlecitinib · JAK3/TEC kinasesC3H/HeJ skin-grafted alopecia areata mice: IV ritlecitinib prevented AA onset and hair loss
Livdelzi
seladelpar · PPARδCarbon tetrachloride (CCl4) liver fibrosis model in mice
Livmarli
maralixibat · IBAT (ASBT)Rat partial bile duct ligation (pBDL) cholestasis model: reduced serum bile acids, liver enzymes and bilirubin
Livtencity
maribavir · CMV UL97 protein kinaseIn vivo HCMV models using transplanted human tissue or surrogate animal viral strains, compared with ganciclovir
Lumakras
sotorasib · KRAS G12CMice bearing KRAS G12C human tumor cell line xenografts (incl. NSCLC): ERK inhibition and tumor growth inhibition
Lupkynis
voclosporin · CalcineurinAnimal models of autoimmune uveitis, arthritis and organ transplantation; calcineurin inhibition in monkeys
Lybalvi
olanzapine and samidorphan · D2/5-HT2A; opioid receptors (samidorphan)Olanzapine-induced weight gain and adiposity in rats and nonhuman primates mitigated by samidorphan
Lynavoy
linerixibat · IBATFecal bile acid excretion in healthy mice and dogs; rat necrotizing enterocolitis model; no PBC model
Lynkuet
elinzanetant · NK1/NK3 receptorsMongolian gerbil model: elinzanetant reduced NK-1 agonist (GR-73632)-induced foot tapping
Lytgobi
futibatinib · FGFR1-4 (covalent)Human tumor xenografts with FGFR2 alterations: AN3 CA endometrial carcinoma in nude mice and SNU-16 gastric carcinoma in nude rats
Mayzent
siponimod · S1P1/S1P5 receptorRodent EAE (experimental autoimmune encephalomyelitis) models; oral and ICV siponimod reduced clinical signs
Miebo
perfluorohexyloctane · Tear film evaporation barrierNormal NZW rabbits: 7-day ocular dosing showed a trend to increased tear film breakup time (TFBUT)
Miplyffa
arimoclomol · Heat-shock response amplifierNPC patient fibroblasts and Npc1-/- mutant mice (hepatic GSL, motor deficits)
Modeyso
dordaviprone · DRD2 antagonist / ClpP agonistOrthotopic intracranial (brainstem) tumor-bearing mice; survival and pharmacodynamic endpoints
Myqorzo
aficamten · Cardiac myosinTransgenic MYH7 R403Q HCM mouse and A31P MYBPC3 cat model of hereditary obstructive HCM
Nereus
tradipitant · NK1 receptorApomorphine-induced emesis in Beagle dogs (single oral dose reduced emesis incidence)
Nexletol
bempedoic acid · ATP citrate lyaseLDLR+/- and LDLR-/- Yucatan miniature pigs (160 days): LDL-C lowered vs vehicle
Nextstellis
drospirenone and estetrol · Estrogen / progestin receptorsOvulation and implantation inhibition in normal rabbits
Nourianz
istradefylline · Adenosine A2A receptorMPTP mouse and primate Parkinson's models, 6-OHDA-lesioned rat, haloperidol catalepsy
Nubeqa
darolutamide · Androgen receptorMouse xenograft models of human prostate cancer: tumor growth inhibition and suppressed PSA rise
Nulibry
fosdenopterin · cPMP replacement (MoCD type A)Knockout mouse model of MoCD Type A: reduced plasma and brain SSC, improved survival vs controls
Nurtec ODT
rimegepant · CGRP receptorMarmoset hCGRP-induced facial blood flow: 75-80% inhibition (target engagement in normal animals)
Nuzolvence
zoliflodacin · Bacterial topoisomerase IIIn vitro hollow fiber infection model (HFIM) with N. gonorrhoeae strains WHO-F and WHO-X; no animal infection studies
Ogsiveo
nirogacestat · Gamma secretasePatient-derived desmoid tumor cell lines in vitro; in vivo only in non-desmoid Notch-driven xenografts (e.g., T-ALL)
Ohtuvayre
ensifentrine · PDE3/PDE4Exploratory in vivo models of allergic conditions and asthma; no COPD animal model
Ojemda
tovorafenib · RAF (type II pan-RAF)Mouse tumour xenografts (BRAF-mutant WM-266-4 melanoma; Calu-6, BxPC-3): tumour growth inhibition or regression
Ojjaara
momelotinib · JAK1/JAK2/ACVR1Mouse model of myeloproliferative neoplasms and rat model of anemia of chronic disease
Olinvyk
oliceridine · μ-opioid receptor (G-protein biased)Rodent (mouse, rat) spinal reflexive and supraspinal affective nociceptive pain models
Omlonti
omidenepag isopropyl · EP2 receptorIOP lowering in normotensive rabbits and monkeys and in a monkey model of ocular hypertension
Ongentys
opicapone · COMTMPTP-lesioned cynomolgus monkeys: augmented L-DOPA/benserazide effects on motor activity
Orgovyx
relugolix · GnRH receptorHuman GnRH receptor knock-in mice (testosterone reduction) and castrated monkeys (LH decrease)
Orladeyo
berotralstat · Plasma kallikreinEx vivo kallikrein inhibition in plasma from HAE patients
Orlynvah
sulopenem etzadroxil and probenecid · Penicillin-binding proteinsNeutropenic and immune-competent murine thigh infection models plus in vitro hollow-fiber infection model
Orserdu
elacestrant · Estrogen receptor (SERD)ER+ breast cancer xenograft and patient-derived xenograft mouse models (≥96% TGI at ≥30 mg/kg/day)
Orzeyful
oveporexton · Orexin OX2 receptor agonistOX/ataxin-3 transgenic NT1 mouse model (loss of orexin neurons): increased wakefulness
Oxbryta
voxelotor · HbS polymerisationSCD (SS) mouse model: increased Hb-O2 affinity and reduced ex vivo RBC sickling
Palsonify
paltusotine · Somatostatin receptor 2GHRH-challenged rats: dose-dependent suppression of GH elevation
Paxlovid
nirmatrelvir and ritonavir · SARS-CoV-2 Mpro (3CL protease)Mouse-adapted SARS-CoV-2 (MA10) infection in BALB/c and 129 mice: reduced lung viral titres and disease
Pemazyre
pemigatinib · FGFR1-3Human tumour xenografts in mice with FGFR1-3 activation, incl. patient-derived FGFR2-TRA2B cholangiocarcinoma xenografts
Piqray
alpelisib · PI3KαBreast cancer xenografts in mice, incl. ER-positive PIK3CA-mutant models and PDX
Pizensy
lactitol · Osmotic laxativeNormal rats: increased small intestine contents (osmotic effect), independent of fermentation
Ponvory
ponesimod · S1P1 receptorMouse MOG-peptide experimental autoimmune encephalomyelitis (EAE) model
Pretomanid
pretomanid · Mycobacterial cell wall / respirationAcute, chronic and latent murine TB infection models (lung/spleen CFU, relapse)
Pyrukynd
mitapivat · Pyruvate kinase (activator)Hbbth3/+ β-thalassemia mouse (another hemolytic anemia), normal mice, and RBCs from PKD patients ex vivo
Qelbree
viloxazine · Norepinephrine reuptakeNormal rats: increased brain norepinephrine (and 5-HT, DA) levels
Qinlock
ripretinib · KIT/PDGFRAMouse tumour xenografts: KIT exon 11 GIST T1, imatinib-resistant exon 17 GIST patient-derived xenograft
Qulipta
atogepant · CGRP receptorCapsaicin-induced dermal vasodilatation (CIDV) inhibition as surrogate pharmacodynamic marker; no animal migraine model
Quviviq
daridorexant · Orexin OX1/OX2 receptorsSleep-wake EEG studies in naive rats and dogs (decreased wake, increased REM/non-REM sleep); no disease model
Rapiblyk
landiolol · β1-adrenergic receptorDog halothane-epinephrine, electrical stimulation and aconitine-induced arrhythmia/tachycardia models
Rasonque
daraxonrasib · RAS(ON) multi-selectiveKRAS G12X PDAC cell-line xenografts in mice (mean regressions 53%–93% at 25 mg/kg daily)
Recarbrio
imipenem, cilastatin and relebactam · Beta-lactamase (relebactam)Neutropenic mouse infection models with relebactam plus imipenem
Relyvrio
sodium phenylbutyrate and taurursodiol · ER stress / mitochondrial (proposed)SOD1 G93A transgenic mouse model of ALS (no effect on lifespan or disease progression)
Retevmo
selpercatinib · RETTT medullary thyroid carcinoma and LC-2/ad lung adenocarcinoma xenografts in mice
Revtorpyk
gedatolisib · PI3K/mTORER+ breast cancer PDX models and MCF7 orthotopic xenograft (triplet with palbociclib and fulvestrant)
Revuforj
revumenib · Menin-KMT2AMV4;11 and MOLM-13 human AML xenografts in rats and immunocompromised mice
Reyvow
lasmiditan · 5-HT1F receptorRat trigeminovascular models: dural protein extravasation, c-fos induction, dural-evoked nociception, dural vasodilation
Rezdiffra
resmetirom · Thyroid hormone receptor βHigh-fat diet-induced obese (DIO) mice for 25 weeks: reduced liver weight and vacuolation
Rezlidhia
olutasidenib · IDH1 (mutant)Mouse xenografts of cell lines engineered to express mutant IDH1 (tumor 2-HG measured)
Rezurock
belumosudil · ROCK2cGVHD mouse models (lung and skin endpoints) and murine pulmonary fibrosis model
Rezzayo
rezafungin · Fungal glucan synthaseNeutropenic murine candidiasis model (C. albicans, C. glabrata, C. parapsilosis, C. tropicalis, C. dubliniensis)
Rhapsido
remibrutinib · BTKMouse reverse passive Arthus and passive cutaneous anaphylaxis models of skin mast cell degranulation
Rinvoq
upadacitinib · JAK1Rat adjuvant-induced arthritis model (paw swelling, bone erosion, ankle histology)
Romvimza
vimseltinib · CSF1RCSF1R-dependent in vivo primary PD models; no TGCT models
Rozlytrek
entrectinib · TRK/ROS1/ALKMouse xenograft models (cell line- and patient-derived) with TRK, ROS1 and ALK fusions, including an orthotopic glioma model
Rukobia
fostemsavir · HIV-1 gp120 (attachment)Cell-culture antiviral activity of temsavir against HIV-1 clinical isolates in PBMCs
Scemblix
asciminib · BCR-ABL1 (STAMP, myristoyl pocket)KCL-22 CML xenograft mice, including a T315I-mutant model
Sephience
sepiapterin · Tetrahydrobiopterin precursorPah-R261Q PKU mouse (single dose) plus COS-7 cells expressing PAH variants
Simtriyo
centanafadine · Triple monoamine reuptakeMicrodialysis in normal rats: increased cortical NE, DA and 5-HT
Skyclarys
omaveloxolone · Nrf2 activatorNrf2 target-gene induction in healthy mice, rats and monkeys; no FA animal model
Sofdra
sofpironium · Muscarinic receptors (topical)Pilocarpine-induced perspiration in normal SD rats inhibited by topical 20% gel
Sohonos
palovarotene · RARγInjury-induced HO and FOP mouse models: BMP-2 implant, ALK2Q207D and ALK2R206H transgenic mice
Sotyktu
deucravacitinib · TYK2Mouse IL-23-induced acanthosis (psoriasis), anti-CD40 and T-cell transfer colitis, NZB/W lupus nephritis
Sunlenca
lenacapavir · HIV-1 capsidCell-culture antiviral activity in human MT-4 T-cell line (EC50 0.19 nM)
Sunosi
solriamfetol · Dopamine/norepinephrine reuptakeHypocretin/ataxin-3 transgenic narcolepsy mice: continuous wakefulness induced
Tabrecta
capmatinib · METPatient-derived NSCLC xenografts (MET exon 14 skipping or MET amplification) in nude mice
Tavneos
avacopan · C5a receptor (C5aR1)Anti-MPO ANCA glomerulonephritis model in hC5aR knock-in mice
Tazverik
tazemetostat · EZH2Patient-derived synovial sarcoma (SS18 fusion) xenografts in nude mice
Tepmetko
tepotinib · METMouse xenografts of human METex14-skipping tumor cell lines; complete regression at 6 mg/kg with MET amplification
Trikafta
elexacaftor, tezacaftor and ivacaftor · CFTRCF patient-derived human bronchial epithelial (HBE) cells; increased CFTR processing and chloride transport
Truqap
capivasertib · AKT1-3Human cancer xenografts including ER+ breast cancer models with PIK3CA, AKT1 or PTEN alterations
Truseltiq
infigratinib · FGFR1-3Mouse and rat xenografts of human FGFR2/3-altered tumors, including cholangiocarcinoma PDX with FGFR2 fusions
Tryptyr
acoltremon · TRPM8 agonistIncreased blink rate after a single topical ocular dose in guinea pigs (EC50 9.1 µM)
Tryvio
aprocitentan · Endothelin A/B receptorsHypertensive rat models: DOCA-salt, Dahl-S and SHR rats
Tukysa
tucatinib · HER2Mouse xenograft models of HER2-expressing tumors (anti-tumor activity, HER2 phosphorylation inhibition)
Turalio
pexidartinib · CSF1RMouse model of splenomegaly from injected CSF1R signaling-dependent cells: inhibited 79%
Ubrelvy
ubrogepant · CGRP receptorCapsaicin-induced dermal vasodilation in rat, rabbit and monkey; CGRP-induced relaxation of human cranial arteries ex vivo
Ukoniq
umbralisib · PI3Kδ / CK1εMOLT-4 xenograft mice (150 mg/kg/day oral) plus 13 leukemia/lymphoma cell lines
Utebzi
tebipenem pivoxil · Penicillin-binding proteinsNeutropenic mouse and immunocompetent rat pyelonephritis infection models
Vafseo
vadadustat · HIF prolyl hydroxylaseHealthy normoxic mice and rats (EPO and erythropoiesis increases); no anemia model
Vanflyta
quizartinib · FLT3MV4-11 (FLT3-ITD) mouse xenograft: tumor growth inhibition and increased lifespan
Vanrafia
atrasentan · Endothelin A receptorSpontaneous IgAN mouse (grouped-ddY): dose-dependent albuminuria reduction
Veklury
remdesivir · SARS-CoV-2 RNA polymeraseInfection challenge: mouse SARS-CoV/MERS-CoV and non-human primate SARS-CoV-2/MERS-CoV models
Velsipity
etrasimod · S1P1/S1P4/S1P5 receptorsT-cell transfer colitis in SCID mice: reduced colonic T-cell and monocyte markers
Veozah
fezolinetant · Neurokinin-3 receptorOvariectomized rat (postmenopause model): attenuated hot flash-like symptoms
Veppanu
vepdegestrant · Estrogen receptor (PROTAC degrader)MCF7 xenograft mice: tumor shrinkage (124% TGI) and >94% ER reduction at 30 mg/kg/day
Verquvo
vericiguat · Soluble guanylate cyclaseSpontaneously hypertensive rats, renin-transgenic rat heart failure model, dog/minipig PAH and dog PH-LVD models
Vivjoa
oteseconazole · Fungal CYP51Estrogen-dependent murine model of vaginal candidiasis (C. albicans infection)
Vizz
aceclidine · Muscarinic agonist (miotic)Pupil response (miosis) in normal New Zealand White rabbits after topical aceclidine 1.75%
Vonjo
pacritinib · JAK2/FLT3Two murine myelofibrosis models driven by excessive JAK2 activity (nude mice bearing Ba/F3-JAK2 cells)
Voquezna
vonoprazan · H+/K+-ATPase (potassium-competitive)Rat NSAID-induced gastric lesion and experimental reflux esophagitis models
Voranigo
vorasidenib · IDH1/IDH2Mouse xenografts: subcutaneous U87 IDH2-R140Q and orthotopic TS603 IDH1-R132H glioma (tumor 2-HG reduction)
Voydeya
danicopan · Complement factor DEx vivo alternative-pathway inhibition (80-90%) in serum of orally dosed cynomolgus monkeys
Vtama
tapinarof · Aryl hydrocarbon receptorImiquimod mouse psoriasis model: reduced epidermal thickness and IL-17a/IL-17f/IL-19/IL-1β/IL-23a mRNA
Vyndaqel
tafamidis · Transthyretin (stabiliser)In vitro only: TTR binding and tetramer stabilisation under acid/urea denaturation and physiologic conditions
Wakix
pitolisant · Histamine H3 receptorOrexin-/- narcolepsy mouse: fewer narcoleptic attacks and shorter total narcolepsy duration
Wayrilz
rilzabrutinib · BTK (reversible covalent)Mouse anti-CD41 antibody-induced ITP model; also rat collagen-induced arthritis and canine pemphigus
Welireg
belzutifan · HIF-2αVHL-deficient 786-O ccRCC xenograft and patient-derived ccRCC xenograft in immunodeficient mice
Winlevi
clascoterone · Androgen receptor (topical)Golden Syrian hamster flank organ (testosterone-induced) model with topical clascoterone
Xacduro
sulbactam and durlobactam · PBPs; beta-lactamase (durlobactam)Neutropenic murine thigh and lung (pneumonia) infection models with MDR A. baumannii
Xcopri
cenobamate · Sodium channel / GABA-ARodent seizure models (battery); antiepileptic effects in mice and rats at 0.3-30 mg/kg
Xdemvy
lotilaner · Mite GABA-Cl channelNo proof-of-concept studies for Demodex blepharitis; mechanism (mite GABA-Cl inhibition) only
Xeglyze
abametapir · Lice metalloproteinasesIn vitro ovicidal assay on resistant head lice eggs (SF-HL strain) with the lotion
Xenleta
lefamulin · Bacterial ribosome (50S PTC)Animal infection models of S. pneumoniae and S. aureus pneumonia (PK-PD index fAUC/MIC)
Xolremdi
mavorixafor · CXCR4Human K562 cells expressing WT and WHIM-mutant CXCR4 (CXCL12-binding IC50 6.1 nM WT; 3.2-11 nM mutants)
Xpovio
selinexor · XPO1Mouse xenograft models of multiple myeloma (selinexor alone and with dexamethasone)
Zavzpret
zavegepant · CGRP receptorSuppression of CGRP-induced facial blood flow in cynomolgus monkeys; in vitro receptor binding
Zaynich
cefepime and zidebactam · PBPs; beta-lactam enhancer (zidebactam)Neutropenic mouse lung, thigh and cUTI infection models with human-simulated regimens
Zegfrovy
sunvozertinib · EGFR exon 20 insertionPatient-derived NSCLC xenografts with EGFR exon20ins (LU0387, LU3075) and A431 WT EGFR xenograft in nude mice
Zenbexus
iberdomide · Cereblon (CELMoD)Multiple myeloma xenograft models in mice, including lenalidomide-resistant models
Zeposia
ozanimod · S1P1/S1P5 receptorMouse EAE model and cuprizone demyelination model; lymphocyte reduction in rats, mice, dogs and monkeys
Zepzelca
lurbinectedin · DNA minor groove (transcription)Subcutaneous/orthotopic human CDX and PDX tumour xenografts in mice (lung, breast, prostate, ovary, pancreas, sarcoma and others)
Zevtera
ceftobiprole medocaril · Penicillin-binding proteinsInfection models: murine septicemia and acute pneumonia, rat/rabbit endocarditis and osteomyelitis (MSSA, MRSA, S. pneumoniae)
Zokinvy
lonafarnib · FarnesyltransferaseHutchinson-Gilford progeria mouse model (homozygous LmnaG609G/G609G): lonafarnib prolonged survival
Zulresso
brexanolone · GABA-A receptor (positive modulator)Behavioral depressant effects (sedation, muscle relaxation, decreased locomotion) in rats, dogs and mice; no PPD model
Zurzuvae
zuranolone · GABA-A receptor (positive modulator)Rodent anticonvulsant, EEG, anxiolytic-like and sedation models (target engagement)
Zycubo
copper histidinate · Copper replacement (Menkes disease)Published Menkes mouse model only (CuHis with AAV9-ATP7A); CuHis alone gave no survival benefit
Xocova
ensitrelvir · SARS-CoV-2 3CL proteaseNot stated in public reviews