Accrufer
ferric maltol · Iron replacementRadiolabeled iron studies in rats (confirmed in dogs): iron transferred to transferrin and ferritin; literature on iron
Zavzpret: animal efficacy model or human cells?
zavegepant · CGRP receptor
Efficacy evidence
Suppression of CGRP-induced facial blood flow in cynomolgus monkeys; in vitro receptor binding
“An in vivo pharmacology study indicated suppression of CGRP-induced facial blood flow in monkeys without vasoconstrictive effects on coronary or cerebral arteries (as measured by laser doppler).”
Why that model
CGRP receptor affinity ~10,000-fold lower in dog, rabbit and rat; similar in cynomolgus monkey
“In vitro primary pharmacology studies indicated similar affinity of BHV-3500 for human and cynomolgus monkey CGRP receptors; however, affinity was approximately 10,000-fold lower for dog, rabbit, and rat CGRP.”
Reviewer's view of the efficacy data
FDA: in vitro and in vivo studies characterize zavegepant as a selective CGRP receptor antagonist (IC50 0.04 nM)
“The results of in vitro and in vivo studies characterize zavegepant as a calcitonin gene-related peptide (CGRP) receptor antagonist (IC50 of 0.04 nM), with selectivity for CGRP compared to other members of the calcitonin receptor family”
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)
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
Ztalmy
ganaxolone · GABA-A receptor (positive modulator)Traditional rodent seizure models (chemically induced convulsions, corneal kindling); not tested in a Cdkl5 disease model
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