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Atlas/Small molecules/Nulibry/Efficacy evidence

Nulibry: animal efficacy model or human cells?

fosdenopterin · cPMP replacement (MoCD type A)
Check my plan vs. these
Efficacy evidence
Knockout mouse model of MoCD Type A: reduced plasma and brain SSC, improved survival vs controls
“animal data from a knockout mouse model of MoCD Type A showing a reduction of plasma and brain SSC and improved mortality in treated mice compared to controls.”
✓ VerifiedFDA integrated review, p. 14Source ↗
Why that model
MoCo synthesis pathway highly conserved; cPMP identical between species
“The MoCo synthesis pathway is highly conserved, and cPMP is considered identical between species.”
✓ VerifiedEMA assessment report, p. 28Source ↗
Reviewer's view of the efficacy data
EMA: pharmacodynamic studies support cPMP replacement in MOCS1-/- knockout mice; treatment from PND1 prevented early death
“Overall, primary pharmacodynamic studies support that fosdenopterin can replace endogenous cPMP in a knock-out mouse model of MoCD (MOCS1-/-), showing phenotypes with similar characteristics and biochemical anomalies as human MoCD Type A. In these animals, fosdenopterin treatment from PND1 prevented early death.”
✓ VerifiedEMA assessment report, p. 28Source ↗

Same question, other small molecules

Accrufer

ferric maltol · Iron replacement
Radiolabeled iron studies in rats (confirmed in dogs): iron transferred to transferrin and ferritin; literature on iron

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 · CFTR
CF 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/TRK
Mouse 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; FAK
LGSOC patient-derived xenograft mice: avutometinib plus FAK-inhibitor surrogate VS-4718

Ayvakit

avapritinib · KIT/PDGFRA
Mouse P815 mastocytoma (KIT exon 17) model and imatinib-resistant GIST patient-derived xenograft (KIT exon 11/17)

Azstarys

serdexmethylphenidate and dexmethylphenidate · Dopamine/norepinephrine reuptake
In vitro radioligand binding only: SDX showed no significant binding to human DAT, NET or SERT

Balversa

erdafitinib · FGFR1-4
Human tumor cell-line xenografts with FGFR1, 2 and 3 alterations (bladder, lung, GI, liver) in immune-deficient rodents

Barhemsys

amisulpride · Dopamine D2/D3 receptor
Ferret 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-2
MAVER-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 · SGLT2
Diabetic db/db mouse and ZDF rat models: dose-dependent HbA1c reduction

Brexafemme

ibrexafungerp · Fungal glucan synthase
In 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 · BTK
REC-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 · IBAT
Mdr2-/- mouse cholestasis model: reduced liver weight and serum markers of liver damage and cholestasis

Cabenuva

cabotegravir and rilpivirine · HIV-1 integrase; NNRTI
In vitro antiviral activity in human MT4 T-cell line and HIV-1 primary isolates (rilpivirine)

Camzyos

mavacamten · Cardiac myosin
Transgenic troponin T-mutant HCM mice dosed 6 months: improved diastolic function, preserved fractional shortening

Caplyta

lumateperone · 5-HT2A / D2 receptor
Rodent models of antipsychotic activity: head twitch, conditioned avoidance, amphetamine-induced hyperlocomotion

Cardamyst

etripamil · L-type calcium channel
Isolated rabbit hearts, spontaneously hypertensive rats and normotensive rabbits (BP, AV conduction, ECG)

Cibinqo

abrocitinib · JAK1
Adjuvant-induced arthritis rat model (paw swelling; ex vivo STAT phosphorylation)

Cobenfy

xanomeline and trospium · Muscarinic M1/M4 agonist; peripheral antagonist
Rodent and monkey models of schizophrenia: conditioned avoidance, psychostimulant-induced hyperactivity, schedule-controlled behavior

Cosela

trilaciclib · CDK4/6
In vivo mouse studies of protection from chemotherapy-induced myelosuppression and of tumor protection

Crenessity

crinecerfont · CRF1 receptor
In vitro CRF1 binding, cAMP and ACTH assays; CRF- and stress-induced ACTH inhibition in normal rats

Cypsedo

cipepofol · GABA-A receptor
Anesthesia in normal rats, dogs and minipigs, similar depth to propofol with 6-fold higher potency

Daybue

trofinetide · IGF-1 tripeptide analogue
MeCP2 knockout mouse model of Rett syndrome (20 mg/kg IP daily, 5 or 20 weeks)

Dayvigo

lemborexant · Orexin OX1/OX2 receptors
Increased total sleep time in normal mice and rats (ED50 1 and 4.4 mg/kg)

Defencath

taurolidine and heparin · Antimicrobial catheter lock
In vitro antimicrobial activity of taurolidine against a panel of microorganisms

Dojolvi

triheptanoin · Anaplerotic energy substrate
Published 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 kallikrein
Carrageenan-induced paw oedema in Sprague-Dawley rats (non-HAE acute inflammation model)

Ensacove

ensartinib · ALK
H3122 (EML4-ALK) human NSCLC xenograft in nude mice

Evrysdi

risdiplam · SMN2 splicing
SMNΔ7 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 insertion
Patient-derived NSCLC xenograft LU0387 (EGFR exon 20 insertion) in immunocompromised mice

Exxua

gepirone · 5-HT1A receptor
Active in animal models predictive of antidepressant and anxiolytic activity

Fabhalta

iptacopan · Complement factor B
PNH patient erythrocytes in vitro: inhibition of hemolysis and C3 deposition

Fetroja

cefiderocol · Penicillin-binding proteins
Murine thigh and lung infection models with Gram-negative strains (dose-ranging, PK/PD target)

Fexinidazole

fexinidazole · Trypanosoma brucei
Acute and chronic murine T. brucei infection models of HAT

Filspari

sparsentan · Endothelin A / angiotensin II receptors
gddY and passive (EIC-induced) mouse IgAN models; anti-Thy-1 rat mesangioproliferative glomerulonephritis model

Fotivda

tivozanib · VEGFR1-3
Human 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 · VEGFR1-3
Human tumor xenografts in BALB/c nude mice (BGC-823, HT-29, Caki-1, NCI-H460)

Gavreto

pralsetinib · RET
RET-mutant and patient-derived RET-fusion (NSCLC, CRC) xenografts in mice

Gemtesa

vibegron · β3-adrenergic receptor
Acetic acid-induced bladder overactivity model (rat)

Gomekli

mirdametinib · MEK1/2
Genetically engineered Nf1fl/fl;Dhh-Cre neurofibroma mouse model

Grafapex

treosulfan · DNA alkylation
Murine 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 · NHE3
Stress-induced IBS-C rat model, mouse opioid-induced constipation model, neonatal acetic-acid IBS-like rat model

Ibtrozi

taletrectinib · ROS1
Subcutaneous ROS1-fusion xenografts (Ba/F3, NSCLC) and intracranial patient-derived NSCLC xenograft in mice

Idvynso

doravirine and islatravir · NNRTI; NRTTI
Islatravir 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/SGLT2
Metabolic 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 · JAK2
Mouse models of JAK2V617F-driven myeloproliferative disease: survival up to 100% at 42 days; reduced WBC, hematocrit, splenomegaly, fibrosis

Iqirvo

elafibranor · PPARα/δ
NASH 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 · PDE4B
Bleomycin 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 hydroxylase
Normal Sprague-Dawley rats: dose-related increases in RBC, hemoglobin and hematocrit

Jideytro

zidesamtinib · ROS1
Patient-derived xenograft (PDX) mouse models of ROS1-fusion NSCLC (SDC4-ROS1, CD74-ROS1 G2032R)

Joenja

leniolisib · PI3Kδ
Pik3cd E1020K/+ mouse model of APDS; rat TDAR and induced inflammation models

Journavx

suzetrigine · NaV1.8
Primary human DRG neurons in vitro (NaV1.8 current and action potential firing inhibition); no in vivo model

Kerendia

finerenone · Mineralocorticoid receptor
Animal models of heart failure and chronic kidney disease: heart and kidney protection

Klisyri

tirbanibulin · Tubulin / Src
Cultured keratinocytes in vitro: proliferation reduced 50% at 40 nM

Komzifti

ziftomenib · Menin-KMT2A
AML xenografts (MV4-11, MOLM-13 cell lines and patient-derived cells) in immunocompromised mice and rats

Koselugo

selumetinib · MEK1/2
Nf1fl/fl;DhhCre neurofibroma knockout mouse: reduced nerve root size and tumor number/size

Krazati

adagrasib · KRAS G12C
KRAS 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 cruzi
Acute 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/JAK2
In 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 kinases
C3H/HeJ skin-grafted alopecia areata mice: IV ritlecitinib prevented AA onset and hair loss

Livdelzi

seladelpar · PPARδ
Carbon 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 kinase
In vivo HCMV models using transplanted human tissue or surrogate animal viral strains, compared with ganciclovir

Lumakras

sotorasib · KRAS G12C
Mice bearing KRAS G12C human tumor cell line xenografts (incl. NSCLC): ERK inhibition and tumor growth inhibition

Lupkynis

voclosporin · Calcineurin
Animal 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 · IBAT
Fecal bile acid excretion in healthy mice and dogs; rat necrotizing enterocolitis model; no PBC model

Lynkuet

elinzanetant · NK1/NK3 receptors
Mongolian 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 receptor
Rodent EAE (experimental autoimmune encephalomyelitis) models; oral and ICV siponimod reduced clinical signs

Miebo

perfluorohexyloctane · Tear film evaporation barrier
Normal NZW rabbits: 7-day ocular dosing showed a trend to increased tear film breakup time (TFBUT)

Miplyffa

arimoclomol · Heat-shock response amplifier
NPC patient fibroblasts and Npc1-/- mutant mice (hepatic GSL, motor deficits)

Modeyso

dordaviprone · DRD2 antagonist / ClpP agonist
Orthotopic intracranial (brainstem) tumor-bearing mice; survival and pharmacodynamic endpoints

Myqorzo

aficamten · Cardiac myosin
Transgenic MYH7 R403Q HCM mouse and A31P MYBPC3 cat model of hereditary obstructive HCM

Nereus

tradipitant · NK1 receptor
Apomorphine-induced emesis in Beagle dogs (single oral dose reduced emesis incidence)

Nexletol

bempedoic acid · ATP citrate lyase
LDLR+/- and LDLR-/- Yucatan miniature pigs (160 days): LDL-C lowered vs vehicle

Nextstellis

drospirenone and estetrol · Estrogen / progestin receptors
Ovulation and implantation inhibition in normal rabbits

Nourianz

istradefylline · Adenosine A2A receptor
MPTP mouse and primate Parkinson's models, 6-OHDA-lesioned rat, haloperidol catalepsy

Nubeqa

darolutamide · Androgen receptor
Mouse xenograft models of human prostate cancer: tumor growth inhibition and suppressed PSA rise

Nurtec ODT

rimegepant · CGRP receptor
Marmoset hCGRP-induced facial blood flow: 75-80% inhibition (target engagement in normal animals)

Nuzolvence

zoliflodacin · Bacterial topoisomerase II
In vitro hollow fiber infection model (HFIM) with N. gonorrhoeae strains WHO-F and WHO-X; no animal infection studies

Ogsiveo

nirogacestat · Gamma secretase
Patient-derived desmoid tumor cell lines in vitro; in vivo only in non-desmoid Notch-driven xenografts (e.g., T-ALL)

Ohtuvayre

ensifentrine · PDE3/PDE4
Exploratory 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/ACVR1
Mouse 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 receptor
IOP lowering in normotensive rabbits and monkeys and in a monkey model of ocular hypertension

Ongentys

opicapone · COMT
MPTP-lesioned cynomolgus monkeys: augmented L-DOPA/benserazide effects on motor activity

Orgovyx

relugolix · GnRH receptor
Human GnRH receptor knock-in mice (testosterone reduction) and castrated monkeys (LH decrease)

Orladeyo

berotralstat · Plasma kallikrein
Ex vivo kallikrein inhibition in plasma from HAE patients

Orlynvah

sulopenem etzadroxil and probenecid · Penicillin-binding proteins
Neutropenic 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 agonist
OX/ataxin-3 transgenic NT1 mouse model (loss of orexin neurons): increased wakefulness

Oxbryta

voxelotor · HbS polymerisation
SCD (SS) mouse model: increased Hb-O2 affinity and reduced ex vivo RBC sickling

Palsonify

paltusotine · Somatostatin receptor 2
GHRH-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 · FGFR1-3
Human tumour xenografts in mice with FGFR1-3 activation, incl. patient-derived FGFR2-TRA2B cholangiocarcinoma xenografts

Piqray

alpelisib · PI3Kα
Breast cancer xenografts in mice, incl. ER-positive PIK3CA-mutant models and PDX

Pizensy

lactitol · Osmotic laxative
Normal rats: increased small intestine contents (osmotic effect), independent of fermentation

Ponvory

ponesimod · S1P1 receptor
Mouse MOG-peptide experimental autoimmune encephalomyelitis (EAE) model

Pretomanid

pretomanid · Mycobacterial cell wall / respiration
Acute, 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 reuptake
Normal rats: increased brain norepinephrine (and 5-HT, DA) levels

Qinlock

ripretinib · KIT/PDGFRA
Mouse tumour xenografts: KIT exon 11 GIST T1, imatinib-resistant exon 17 GIST patient-derived xenograft

Qulipta

atogepant · CGRP receptor
Capsaicin-induced dermal vasodilatation (CIDV) inhibition as surrogate pharmacodynamic marker; no animal migraine model

Quviviq

daridorexant · Orexin OX1/OX2 receptors
Sleep-wake EEG studies in naive rats and dogs (decreased wake, increased REM/non-REM sleep); no disease model

Rapiblyk

landiolol · β1-adrenergic receptor
Dog halothane-epinephrine, electrical stimulation and aconitine-induced arrhythmia/tachycardia models

Rasonque

daraxonrasib · RAS(ON) multi-selective
KRAS 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 · RET
TT medullary thyroid carcinoma and LC-2/ad lung adenocarcinoma xenografts in mice

Revtorpyk

gedatolisib · PI3K/mTOR
ER+ breast cancer PDX models and MCF7 orthotopic xenograft (triplet with palbociclib and fulvestrant)

Revuforj

revumenib · Menin-KMT2A
MV4;11 and MOLM-13 human AML xenografts in rats and immunocompromised mice

Reyvow

lasmiditan · 5-HT1F receptor
Rat trigeminovascular models: dural protein extravasation, c-fos induction, dural-evoked nociception, dural vasodilation

Rezdiffra

resmetirom · Thyroid hormone receptor β
High-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 · ROCK2
cGVHD mouse models (lung and skin endpoints) and murine pulmonary fibrosis model

Rezzayo

rezafungin · Fungal glucan synthase
Neutropenic murine candidiasis model (C. albicans, C. glabrata, C. parapsilosis, C. tropicalis, C. dubliniensis)

Rhapsido

remibrutinib · BTK
Mouse reverse passive Arthus and passive cutaneous anaphylaxis models of skin mast cell degranulation

Rinvoq

upadacitinib · JAK1
Rat adjuvant-induced arthritis model (paw swelling, bone erosion, ankle histology)

Romvimza

vimseltinib · CSF1R
CSF1R-dependent in vivo primary PD models; no TGCT models

Rozlytrek

entrectinib · TRK/ROS1/ALK
Mouse 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 precursor
Pah-R261Q PKU mouse (single dose) plus COS-7 cells expressing PAH variants

Simtriyo

centanafadine · Triple monoamine reuptake
Microdialysis in normal rats: increased cortical NE, DA and 5-HT

Skyclarys

omaveloxolone · Nrf2 activator
Nrf2 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 · RARγ
Injury-induced HO and FOP mouse models: BMP-2 implant, ALK2Q207D and ALK2R206H transgenic mice

Sotyktu

deucravacitinib · TYK2
Mouse IL-23-induced acanthosis (psoriasis), anti-CD40 and T-cell transfer colitis, NZB/W lupus nephritis

Sunlenca

lenacapavir · HIV-1 capsid
Cell-culture antiviral activity in human MT-4 T-cell line (EC50 0.19 nM)

Sunosi

solriamfetol · Dopamine/norepinephrine reuptake
Hypocretin/ataxin-3 transgenic narcolepsy mice: continuous wakefulness induced

Tabrecta

capmatinib · MET
Patient-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 · EZH2
Patient-derived synovial sarcoma (SS18 fusion) xenografts in nude mice

Tepmetko

tepotinib · MET
Mouse xenografts of human METex14-skipping tumor cell lines; complete regression at 6 mg/kg with MET amplification

Trikafta

elexacaftor, tezacaftor and ivacaftor · CFTR
CF patient-derived human bronchial epithelial (HBE) cells; increased CFTR processing and chloride transport

Truqap

capivasertib · AKT1-3
Human cancer xenografts including ER+ breast cancer models with PIK3CA, AKT1 or PTEN alterations

Truseltiq

infigratinib · FGFR1-3
Mouse and rat xenografts of human FGFR2/3-altered tumors, including cholangiocarcinoma PDX with FGFR2 fusions

Tryptyr

acoltremon · TRPM8 agonist
Increased blink rate after a single topical ocular dose in guinea pigs (EC50 9.1 µM)

Tryvio

aprocitentan · Endothelin A/B receptors
Hypertensive rat models: DOCA-salt, Dahl-S and SHR rats

Tukysa

tucatinib · HER2
Mouse xenograft models of HER2-expressing tumors (anti-tumor activity, HER2 phosphorylation inhibition)

Turalio

pexidartinib · CSF1R
Mouse model of splenomegaly from injected CSF1R signaling-dependent cells: inhibited 79%

Ubrelvy

ubrogepant · CGRP receptor
Capsaicin-induced dermal vasodilation in rat, rabbit and monkey; CGRP-induced relaxation of human cranial arteries ex vivo

Ukoniq

umbralisib · PI3Kδ / CK1ε
MOLT-4 xenograft mice (150 mg/kg/day oral) plus 13 leukemia/lymphoma cell lines

Utebzi

tebipenem pivoxil · Penicillin-binding proteins
Neutropenic mouse and immunocompetent rat pyelonephritis infection models

Vafseo

vadadustat · HIF prolyl hydroxylase
Healthy normoxic mice and rats (EPO and erythropoiesis increases); no anemia model

Vanflyta

quizartinib · FLT3
MV4-11 (FLT3-ITD) mouse xenograft: tumor growth inhibition and increased lifespan

Vanrafia

atrasentan · Endothelin A receptor
Spontaneous IgAN mouse (grouped-ddY): dose-dependent albuminuria reduction

Veklury

remdesivir · SARS-CoV-2 RNA polymerase
Infection challenge: mouse SARS-CoV/MERS-CoV and non-human primate SARS-CoV-2/MERS-CoV models

Velsipity

etrasimod · S1P1/S1P4/S1P5 receptors
T-cell transfer colitis in SCID mice: reduced colonic T-cell and monocyte markers

Veozah

fezolinetant · Neurokinin-3 receptor
Ovariectomized 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 cyclase
Spontaneously hypertensive rats, renin-transgenic rat heart failure model, dog/minipig PAH and dog PH-LVD models

Vivjoa

oteseconazole · Fungal CYP51
Estrogen-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/FLT3
Two 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/IDH2
Mouse xenografts: subcutaneous U87 IDH2-R140Q and orthotopic TS603 IDH1-R132H glioma (tumor 2-HG reduction)

Voydeya

danicopan · Complement factor D
Ex vivo alternative-pathway inhibition (80-90%) in serum of orally dosed cynomolgus monkeys

Vtama

tapinarof · Aryl hydrocarbon receptor
Imiquimod 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 receptor
Orexin-/- 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-2α
VHL-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-A
Rodent seizure models (battery); antiepileptic effects in mice and rats at 0.3-30 mg/kg

Xdemvy

lotilaner · Mite GABA-Cl channel
No proof-of-concept studies for Demodex blepharitis; mechanism (mite GABA-Cl inhibition) only

Xeglyze

abametapir · Lice metalloproteinases
In 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 · CXCR4
Human K562 cells expressing WT and WHIM-mutant CXCR4 (CXCL12-binding IC50 6.1 nM WT; 3.2-11 nM mutants)

Xpovio

selinexor · XPO1
Mouse xenograft models of multiple myeloma (selinexor alone and with dexamethasone)

Zavzpret

zavegepant · CGRP receptor
Suppression 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 insertion
Patient-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 receptor
Mouse 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 proteins
Infection models: murine septicemia and acute pneumonia, rat/rabbit endocarditis and osteomyelitis (MSSA, MRSA, S. pneumoniae)

Zokinvy

lonafarnib · Farnesyltransferase
Hutchinson-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 protease
Not stated in public reviews

Summary across all 211 small molecules →