Accrufer
ferric maltol · Iron replacement4 (28-day dog)
Azstarys: how long were the pivotal tox studies?
serdexmethylphenidate and dexmethylphenidate · Dopamine/norepinephrine reuptake
Longest pivotal study
26 (6-month juvenile rabbit)
“The Division agreed to review 28-day repeat-dose toxicology data in two species, an embryo-fetal development study in the rabbit, and 6- month juvenile rabbit study to support this position.”
Pivotal GLP studies
28-day rat and dog studies and 6-month juvenile rabbit study
“The Division agreed to review 28-day repeat-dose toxicology data in two species, an embryo-fetal development study in the rabbit, and 6- month juvenile rabbit study to support this position.”
Route in tox
Oral (gavage / stomach tube)
“A 6-Month Study of KP415 Administered by Oral (Stomach Tube) Administration in Juvenile Rabbits with a 1-Month Recovery Period”
Same question, other small molecules
Adquey
difamilast · PDE4 (topical)39 (39-week dog and minipig)
Agamree
vamorolone · Glucocorticoid receptor (dissociative)39 (9-month dog)
Alyftrek
vanzacaftor, tezacaftor and deutivacaftor · CFTR39 (39-week dog, vanzacaftor)
Anzupgo
delgocitinib · Pan-JAK (topical)39 (9-month oral dog and dermal minipig)
Aqneursa
levacetylleucine · Neuronal metabolism (proposed)39 (39-week dog)
Artesunate
artesunate · Plasmodium (antimalarial)4 (28-day rat and dog)
Attruby
acoramidis · Transthyretin (stabiliser)39 (39-week dog)
Augtyro
repotrectinib · ROS1/TRK13 (91-day rat and monkey)
Avmapki Fakzynja Co-pack
avutometinib and defactinib · RAF/MEK clamp; FAK13 (13-week rat, monkey and dog)
Ayvakit
avapritinib · KIT/PDGFRA39 (9-month dog)
Balversa
erdafitinib · FGFR1-413 (13-week rat and dog)
Barhemsys
amisulpride · Dopamine D2/D3 receptor4 (28-day rat)
Baxfendy
baxdrostat · Aldosterone synthase (CYP11B2)39 (9-month monkey)
Beqalzi
sonrotoclax · BCL-213 weeks
Blujepa
gepotidacin · Bacterial topoisomerases (gyrase/topo IV)13 (13-week rat and dog)
Brenzavvy
bexagliflozin · SGLT239 (39-week monkey)
Brexafemme
ibrexafungerp · Fungal glucan synthase26 (26-week rat)
Brinsupri
brensocatib · DPP1 (cathepsin C)39 (9-month dog)
Brukinsa
zanubrutinib · BTK39 (9-month dog)
Byfavo
remimazolam · GABA-A receptor (benzodiazepine site)4 (4-week minipig and monkey)
Bylvay
odevixibat · IBAT39 (39-week dog)
Cabenuva
cabotegravir and rilpivirine · HIV-1 integrase; NNRTI39 (39-week monkey)
Camzyos
mavacamten · Cardiac myosin39 (9-month dog)
Caplyta
lumateperone · 5-HT2A / D2 receptor39 (9-month dog)
Cardamyst
etripamil · L-type calcium channel26 (6-month monkey)
Cibinqo
abrocitinib · JAK139 (9-month monkey)
Cobenfy
xanomeline and trospium · Muscarinic M1/M4 agonist; peripheral antagonist52 (1-year monkey, xanomeline)
Cosela
trilaciclib · CDK4/61 (7-day rat and dog iv)
Crenessity
crinecerfont · CRF1 receptor52 (12-month dog)
Cypsedo
cipepofol · GABA-A receptor4 (rat and dog, dosing every other day)
Daybue
trofinetide · IGF-1 tripeptide analogue39 (39-week dog)
Dayvigo
lemborexant · Orexin OX1/OX2 receptors39 (9-month monkey)
Defencath
taurolidine and heparin · Antimicrobial catheter lock2 (14-day rat; not considered pivotal)
Dojolvi
triheptanoin · Anaplerotic energy substrate39 (9-month juvenile minipig)
Duvyzat
givinostat · Histone deacetylase (class I/II)39 (39-week monkey)
Egaten
triclabendazole · Fasciola (tubulin)13 (13-week rat and dog)
Ekterly
sebetralstat · Plasma kallikrein26 (6-month rat and monkey)
Ensacove
ensartinib · ALK13 (13-week rat and dog)
Evrysdi
risdiplam · SMN2 splicing39 (39-week monkey)
Exblifep
cefepime and enmetazobactam · PBPs; beta-lactamase (enmetazobactam)13 (13-week dog combination study)
Exkivity
mobocertinib · EGFR exon 20 insertion13 (3-month rat and dog)
Exxua
gepirone · 5-HT1A receptor52 (12-month dog)
Fabhalta
iptacopan · Complement factor B39 (39-week dog)
Fetroja
cefiderocol · Penicillin-binding proteins13 (3-month rat and monkey)
Fexinidazole
fexinidazole · Trypanosoma brucei4 (28-day rat and dog)
Filspari
sparsentan · Endothelin A / angiotensin II receptors39 (39-week monkey)
Fotivda
tivozanib · VEGFR1-339 (39-week monkey)
Foundayo
orforglipron · GLP-1 receptor (non-peptide)39 (9-month monkey)
Fruzaqla
fruquintinib · VEGFR1-313 (13-week rat and dog GLP studies)
Gavreto
pralsetinib · RET13 (13-week rat and monkey)
Gemtesa
vibegron · β3-adrenergic receptor39 (9-month monkey)
Gomekli
mirdametinib · MEK1/213 (3-month rat and dog)
Grafapex
treosulfan · DNA alkylation4 (4-week rat GLP study)
Hernexeos
zongertinib · HER2 (mutant)13 (13-week rat and dog)
Hyrnuo
sevabertinib · HER2 (mutant)13 (13-week rat and monkey)
Ibsrela
tenapanor · NHE339 (9-month dog)
Ibtrozi
taletrectinib · ROS112 (85-day rat and monkey)
Idvynso
doravirine and islatravir · NNRTI; NRTTI39 (9-month monkey)
Inluriyo
imlunestrant · Estrogen receptor (SERD)26 (6-month rat)
Inpefa
sotagliflozin · SGLT1/SGLT239 (39-week dog)
Inqovi
decitabine and cedazuridine · DNMT (decitabine); cytidine deaminase (cedazuridine)13 (92-day monkey, 4 cycles)
Inrebic
fedratinib · JAK239 (9-month dog)
Iqirvo
elafibranor · PPARα/δ52 (12-month monkey)
Isturisa
osilodrostat · 11β-hydroxylase (CYP11B1)39 (39-week dog)
Itovebi
inavolisib · PI3Kα (mutant-selective)13 (3-month rat and dog)
Jascayd
nerandomilast · PDE4B39 (39-week minipig and monkey)
Jaypirca
pirtobrutinib · BTK (non-covalent)13 (3-month rat and dog)
Jesduvroq
daprodustat · HIF prolyl hydroxylase39 (39-week monkey)
Jideytro
zidesamtinib · ROS113 (13-week rat and dog)
Joenja
leniolisib · PI3Kδ39 (39-week monkey)
Journavx
suzetrigine · NaV1.813 (13-week rat and monkey)
Kerendia
finerenone · Mineralocorticoid receptor39 (9-month dog)
Klisyri
tirbanibulin · Tubulin / Src4 (28-day oral rat and dog)
Komzifti
ziftomenib · Menin-KMT2A13 (13-week mouse and dog)
Koselugo
selumetinib · MEK1/226 (26-week mouse and monkey)
Krazati
adagrasib · KRAS G12C13 (13-week rat and dog)
Kygevvi
doxecitine and doxribtimine · Pyrimidine nucleoside replacement (TK2 deficiency)26 (6-month juvenile rat)
Lampit
nifurtimox · Trypanosoma cruzi52 (52-week dog)
Lazcluze
lazertinib · EGFR (mutant-selective)13 (13-week rat and dog)
Leqselvi
deuruxolitinib · JAK1/JAK252 (52-week dog)
Lifyorli
relacorilant · Glucocorticoid receptor (antagonist)39 (39-week monkey)
Litfulo
ritlecitinib · JAK3/TEC kinases39 (39-week dog)
Livdelzi
seladelpar · PPARδ52 (52-week monkey)
Livmarli
maralixibat · IBAT (ASBT)52 (1-year dog)
Livtencity
maribavir · CMV UL97 protein kinase52 (52-week monkey)
Lumakras
sotorasib · KRAS G12C13 (3-month rat and dog)
Lupkynis
voclosporin · Calcineurin39 (39-week monkey)
Lybalvi
olanzapine and samidorphan · D2/5-HT2A; opioid receptors (samidorphan)39 (9-month dog, samidorphan)
Lynavoy
linerixibat · IBAT39 (39-week dog)
Lynkuet
elinzanetant · NK1/NK3 receptors39 (39-week monkey)
Lytgobi
futibatinib · FGFR1-4 (covalent)13 (13-week rat and dog)
Mayzent
siponimod · S1P1/S1P5 receptor52 (52-week monkey)
Miebo
perfluorohexyloctane · Tear film evaporation barrier26 (26-week rabbit ocular)
Miplyffa
arimoclomol · Heat-shock response amplifier52 (52-week dog)
Modeyso
dordaviprone · DRD2 antagonist / ClpP agonist13 (13-week rat and dog)
Myqorzo
aficamten · Cardiac myosin39 (39-week dog)
Nereus
tradipitant · NK1 receptor26 (6-month rat)
Nexletol
bempedoic acid · ATP citrate lyase52 (52-week monkey)
Nextstellis
drospirenone and estetrol · Estrogen / progestin receptors39 (39-week monkey)
Nourianz
istradefylline · Adenosine A2A receptor52 (52-week dog)
Nubeqa
darolutamide · Androgen receptor39 (39-week dog)
Nulibry
fosdenopterin · cPMP replacement (MoCD type A)39 (juvenile dog)
Nurtec ODT
rimegepant · CGRP receptor39 (9-month monkey)
Nuzolvence
zoliflodacin · Bacterial topoisomerase II4 (4-week rat and dog)
Ogsiveo
nirogacestat · Gamma secretase13 (3-month rat and dog)
Ohtuvayre
ensifentrine · PDE3/PDE440 (40-week dog)
Ojemda
tovorafenib · RAF (type II pan-RAF)13 (3-month rat and monkey)
Ojjaara
momelotinib · JAK1/JAK2/ACVR139 (39-week dog)
Olinvyk
oliceridine · μ-opioid receptor (G-protein biased)4 (28-day rat)
Omlonti
omidenepag isopropyl · EP2 receptor39 (39-week monkey topical ocular)
Ongentys
opicapone · COMT52 (52-week monkey)
Orgovyx
relugolix · GnRH receptor39 (39-week monkey)
Orladeyo
berotralstat · Plasma kallikrein39 (9-month monkey)
Orlynvah
sulopenem etzadroxil and probenecid · Penicillin-binding proteins4 (1-month rat and monkey)
Orserdu
elacestrant · Estrogen receptor (SERD)39 (39-week monkey)
Orzeyful
oveporexton · Orexin OX2 receptor agonist39 (39-week monkey)
Oxbryta
voxelotor · HbS polymerisation39 (39-week monkey)
Palsonify
paltusotine · Somatostatin receptor 239 (9-month dog)
Paxlovid
nirmatrelvir and ritonavir · SARS-CoV-2 Mpro (3CL protease)4 (1-month rat and monkey)
Pemazyre
pemigatinib · FGFR1-313 (3-month rat and monkey)
Piqray
alpelisib · PI3Kα13 (13-week rat and dog)
Pizensy
lactitol · Osmotic laxative13 (91-day rat)
Ponvory
ponesimod · S1P1 receptor52 (52-week dog)
Pretomanid
pretomanid · Mycobacterial cell wall / respiration39 (9-month monkey)
Pyrukynd
mitapivat · Pyruvate kinase (activator)39 (9-month monkey)
Qelbree
viloxazine · Norepinephrine reuptake78 (18-month rat)
Qinlock
ripretinib · KIT/PDGFRA13 (13-week rat and dog)
Qulipta
atogepant · CGRP receptor39 (9-month monkey)
Quviviq
daridorexant · Orexin OX1/OX2 receptors39 (39-week dog)
Rapiblyk
landiolol · β1-adrenergic receptor4 (4-week rat and dog studies)
Rasonque
daraxonrasib · RAS(ON) multi-selective13 (13-week mouse and monkey)
Recarbrio
imipenem, cilastatin and relebactam · Beta-lactamase (relebactam)13 (3-month rat and monkey)
Relyvrio
sodium phenylbutyrate and taurursodiol · ER stress / mitochondrial (proposed)39 (9-month minipig)
Retevmo
selpercatinib · RET13 weeks
Revtorpyk
gedatolisib · PI3K/mTOR13 (3-month rat and dog)
Revuforj
revumenib · Menin-KMT2A13 (13-week rat and dog)
Reyvow
lasmiditan · 5-HT1F receptor39 (39-week dog)
Rezdiffra
resmetirom · Thyroid hormone receptor β39 weeks
Rezlidhia
olutasidenib · IDH1 (mutant)13 (13-week rat and monkey)
Rezurock
belumosudil · ROCK239 (39-week dog)
Rezzayo
rezafungin · Fungal glucan synthase26 (26-week rat and monkey)
Rhapsido
remibrutinib · BTK39 (39-week dog)
Rinvoq
upadacitinib · JAK139 (39-week dog)
Romvimza
vimseltinib · CSF1R39 (39-week dog)
Rozlytrek
entrectinib · TRK/ROS1/ALK13 (13-week rat and dog)
Rukobia
fostemsavir · HIV-1 gp120 (attachment)39 (9-month dog)
Scemblix
asciminib · BCR-ABL1 (STAMP, myristoyl pocket)39 (39-week monkey)
Sephience
sepiapterin · Tetrahydrobiopterin precursor39 (39-week marmoset)
Simtriyo
centanafadine · Triple monoamine reuptake43 (43-week juvenile dog)
Skyclarys
omaveloxolone · Nrf2 activator39 (39-week monkey)
Sofdra
sofpironium · Muscarinic receptors (topical)39 (39-week dermal minipig)
Sohonos
palovarotene · RARγ39 (39-week dog)
Sotyktu
deucravacitinib · TYK239 (9-month monkey)
Sunlenca
lenacapavir · HIV-1 capsid39 (9-month dog)
Sunosi
solriamfetol · Dopamine/norepinephrine reuptake52 (12-month dog)
Tabrecta
capmatinib · MET13 (13-week rat and monkey)
Tavneos
avacopan · C5a receptor (C5aR1)44 (44-week monkey)
Tazverik
tazemetostat · EZH213 (13-week rat and monkey)
Tepmetko
tepotinib · MET39 (39-week dog)
Trikafta
elexacaftor, tezacaftor and ivacaftor · CFTR39 (39-week dog, elexacaftor)
Truqap
capivasertib · AKT1-339 (9-month dog)
Truseltiq
infigratinib · FGFR1-339 (39-week dog)
Tryptyr
acoltremon · TRPM8 agonist26 (6-month rabbit and dog ocular)
Tryvio
aprocitentan · Endothelin A/B receptors39 (39-week dog)
Tukysa
tucatinib · HER213 weeks
Turalio
pexidartinib · CSF1R39 (9-month dog)
Ubrelvy
ubrogepant · CGRP receptor39 (9-month monkey)
Ukoniq
umbralisib · PI3Kδ / CK1ε39 (39-week dog)
Utebzi
tebipenem pivoxil · Penicillin-binding proteins4 (28-day GLP rat and monkey; non-GLP studies up to 90 days)
Vafseo
vadadustat · HIF prolyl hydroxylase39 (9-month dog)
Vanflyta
quizartinib · FLT313 (13-week rat, dog and monkey)
Vanrafia
atrasentan · Endothelin A receptor39 (9-month dog)
Veklury
remdesivir · SARS-CoV-2 RNA polymerase4 (4-week rat and monkey)
Velsipity
etrasimod · S1P1/S1P4/S1P5 receptors39 (9-month dog)
Veozah
fezolinetant · Neurokinin-3 receptor39 (39-week monkey)
Veppanu
vepdegestrant · Estrogen receptor (PROTAC degrader)39 (9-month dog)
Verquvo
vericiguat · Soluble guanylate cyclase39 (39-week dog)
Vivjoa
oteseconazole · Fungal CYP5139 (9-month dog)
Vizz
aceclidine · Muscarinic agonist (miotic)26 (6-month rabbit ocular)
Vonjo
pacritinib · JAK2/FLT339 (39-week dog)
Voquezna
vonoprazan · H+/K+-ATPase (potassium-competitive)39 (39-week dog)
Voranigo
vorasidenib · IDH1/IDH213 (13-week rat and monkey)
Voydeya
danicopan · Complement factor D39 (39-week dog)
Vtama
tapinarof · Aryl hydrocarbon receptor39 (9-month minipig)
Vyndaqel
tafamidis · Transthyretin (stabiliser)39 (39-week dog)
Wakix
pitolisant · Histamine H3 receptor39 (9-month monkey)
Wayrilz
rilzabrutinib · BTK (reversible covalent)39 (9-month dog)
Welireg
belzutifan · HIF-2α13 (3-month rat and dog)
Winlevi
clascoterone · Androgen receptor (topical)39 (39-week dermal minipig)
Xacduro
sulbactam and durlobactam · PBPs; beta-lactamase (durlobactam)4 (28-day dog)
Xcopri
cenobamate · Sodium channel / GABA-A52 (52-week monkey)
Xdemvy
lotilaner · Mite GABA-Cl channel39 (9-month dog ocular)
Xeglyze
abametapir · Lice metalloproteinases4 (28-day minipig dermal)
Xenleta
lefamulin · Bacterial ribosome (50S PTC)13 (13-week IV studies)
Xocova
ensitrelvir · SARS-CoV-2 3CL protease4 (4-week rat and monkey)
Xolremdi
mavorixafor · CXCR439 (39-week dog)
Xpovio
selinexor · XPO113 (13-week rat and monkey)
Zavzpret
zavegepant · CGRP receptor39 (39-week monkey)
Zaynich
cefepime and zidebactam · PBPs; beta-lactam enhancer (zidebactam)13 (13-week dog)
Zegfrovy
sunvozertinib · EGFR exon 20 insertion13 (13-week rat and dog)
Zenbexus
iberdomide · Cereblon (CELMoD)39 (9-month cynomolgus monkey)
Zeposia
ozanimod · S1P1/S1P5 receptor39 (39-week monkey)
Zepzelca
lurbinectedin · DNA minor groove (transcription)24 (8-cycle q3w rat and monkey)
Zevtera
ceftobiprole medocaril · Penicillin-binding proteins13 (13-week rat, marmoset and dog)
Zokinvy
lonafarnib · Farnesyltransferase52 (1-year monkey)
Ztalmy
ganaxolone · GABA-A receptor (positive modulator)52 (12-month dog)
Zulresso
brexanolone · GABA-A receptor (positive modulator)4 weeks
Zurzuvae
zuranolone · GABA-A receptor (positive modulator)39 (9-month dog)
Zycubo
copper histidinate · Copper replacement (Menkes disease)13 (13-week juvenile rat)