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Atlas/Small molecules/Orladeyo/Risks

Orladeyo: which risks did reviewers probe?

berotralstat · Plasma kallikrein
Check my plan vs. these
Immune & cytokine safety
Foamy macrophages/histiocytes in spleen, thymus and intestine (phospholipidosis)
“Phospholipidosis-related findings included vacuoles in the epithelium and mucosa of the intestine, kidney, and choroid plexus (rats); foamy macrophages and histiocytes in the spleen, thymus, and intestine; and foamy Kupffer cells in the liver.”
✓ VerifiedFDA multi-discipline review, p. 28Source ↗
Biodistribution & persistence
Binds melanin-containing tissues; highest late radioactivity in uveal tract of pigmented rats
“BCX7353 was found to bind to melanin-containing tissues. At 1440 hours post-dose, the highest concentration of radioactivity was quantified in the uveal tract of the eye.”
✓ VerifiedEMA assessment report, p. 32Source ↗
Modality-specific risk
Phospholipidosis and cardiovascular effects (QT prolongation)
“Toxicity studies indicated that berotralstat can result in phospholipidosis and affect the cardiovascular (CV) system of animals.”
✓ VerifiedFDA multi-discipline review, p. 28Source ↗

Same question, other small molecules

Adquey

difamilast · PDE4 (topical)
No skin sensitisation potential in guinea pigs

Agamree

vamorolone · Glucocorticoid receptor (dissociative)
Glucocorticoid immunosuppression: TDAR inhibited in female mice and abolished in male mice at 100 mg/kg/day

Alyftrek

vanzacaftor, tezacaftor and deutivacaftor · CFTR
26-week rat: decreased lymphocytes in spleen, thymus and lymph nodes

Anzupgo

delgocitinib · Pan-JAK (topical)
Immunotoxic in a 4-week oral rat study, an expected immunosuppressive effect

Artesunate

artesunate · Plasmodium (antimalarial)
Increased leukocytes and fibrinogen, indicating an inflammatory response

Augtyro

repotrectinib · ROS1/TRK
Non-adverse decreased lymphocytes in blood and lymphoid organs in rats and monkeys

Avmapki Fakzynja Co-pack

avutometinib and defactinib · RAF/MEK clamp; FAK
Avutometinib: lymph node follicular hyperplasia, plasma cells and inflammatory infiltrates in rats and monkeys

Ayvakit

avapritinib · KIT/PDGFRA
Lymphoid system effects (spleen, thymus) did not reverse

Balversa

erdafitinib · FGFR1-4
Skin sensitiser in the murine local lymph node assay

Barhemsys

amisulpride · Dopamine D2/D3 receptor
High-dose males: 24% decrease in lymphocytes and 19% decrease in total white blood cells

Baxfendy

baxdrostat · Aldosterone synthase (CYP11B2)
Decreased lymphocytes in spleen, thymus and mesenteric lymph nodes in 39-week monkey study (non-adverse)

Beqalzi

sonrotoclax · BCL-2
Lymphoid depletion; one mouse death from opportunistic infection secondary to lymphoid depletion

Blujepa

gepotidacin · Bacterial topoisomerases (gyrase/topo IV)
Thymic lymphoid depletion in high-dose rats, considered stress-related

Brexafemme

ibrexafungerp · Fungal glucan synthase
Thymic lymphoid necrosis in high-dose rats (1-month study)

Brinsupri

brensocatib · DPP1 (cathepsin C)
No dedicated immunotoxicity studies; weight-of-evidence evaluation showed no concerning immunotoxic risk

Brukinsa

zanubrutinib · BTK
Lymphoid depletion in dogs (also rats); inflammatory responses with elevated WBCs

Byfavo

remimazolam · GABA-A receptor (benzodiazepine site)
No immune system findings

Bylvay

odevixibat · IBAT
No effects on thymus, spleen, lymph nodes or bone marrow; dedicated immunotoxicity studies not performed

Cabenuva

cabotegravir and rilpivirine · HIV-1 integrase; NNRTI
Negative rat TDAR; no skin sensitization

Camzyos

mavacamten · Cardiac myosin
Thymus decreased in size from decreased cellularity in rats and dogs

Cibinqo

abrocitinib · JAK1
Monkey KLH assay: dose-related reduction in IgG and IgM from 15 mg/kg/day, with partial recovery

Cobenfy

xanomeline and trospium · Muscarinic M1/M4 agonist; peripheral antagonist
Moderate skin sensitizer in mouse LLNA (EC3 125 µg/cm2)

Cosela

trilaciclib · CDK4/6
Failure to clear catheter-related bacterial infections, suggesting compromised immune response from leukocyte suppression

Crenessity

crinecerfont · CRF1 receptor
Rat TDAR study: small changes in leukocytes, lymphocyte subsets and T-cell-dependent antibody response (males); no remarkable immunotoxicity

Dayvigo

lemborexant · Orexin OX1/OX2 receptors
No drug-related findings in immune function assessments (immunophenotyping, NK activity, granulocyte function, cytokine release)

Defencath

taurolidine and heparin · Antimicrobial catheter lock
Not irritating intradermally and nonsensitizing in guinea pigs

Dojolvi

triheptanoin · Anaplerotic energy substrate
Not a dermal sensitizer in guinea pigs

Duvyzat

givinostat · Histone deacetylase (class I/II)
Lymphoid depletion with atrophy of thymus, lymph nodes, bone marrow and spleen; partially reversible

Egaten

triclabendazole · Fasciola (tubulin)
Guinea pig sensitization: no reaction after epidermal challenge; contact dermatitis unlikely

Ekterly

sebetralstat · Plasma kallikrein
Thymus microscopic findings in monkeys at 300 mg/kg/day and above

Ensacove

ensartinib · ALK
Splenic lymphoid depletion and marked bone marrow hypocellularity in the 4-week dog study

Evrysdi

risdiplam · SMN2 splicing
Decreased B lymphocytes in juvenile rats

Exblifep

cefepime and enmetazobactam · PBPs; beta-lactamase (enmetazobactam)
Guinea pig maximization test: no skin sensitization

Exkivity

mobocertinib · EGFR exon 20 insertion
Increased lymphoid cellularity in lymph nodes in rats and dogs

Exxua

gepirone · 5-HT1A receptor
Increased mesenteric lymph node plasmablasts at end of dosing (highest at 70 mg/kg), reversible after recovery

Fabhalta

iptacopan · Complement factor B
Minimal TDAR effects in 39-week dog study: reduced IgM/IgG titers to KLH

Fetroja

cefiderocol · Penicillin-binding proteins
Splenic germinal center development in 1- and 3-month rat studies, a response to weak antigenic stimulation; no anaphylaxis

Fexinidazole

fexinidazole · Trypanosoma brucei
Dog: decreased thymus weights at all doses

Fotivda

tivozanib · VEGFR1-3
Lymphoid depletion of thymus, lymph nodes and spleen

Fruzaqla

fruquintinib · VEGFR1-3
Immune system (thymus, spleen, lymph nodes) a target organ in dog studies

Gavreto

pralsetinib · RET
No dedicated immunotoxicity studies; decreased cellularity of haematopoietic and lymphoid organs in rats and monkeys

Gomekli

mirdametinib · MEK1/2
Lymphoid hyperplasia, lymphoid depletion and increased plasma cells in lymph nodes of rats and dogs

Grafapex

treosulfan · DNA alkylation
Myeloablative and immunosuppressive in a murine allogeneic bone marrow transplant model

Hernexeos

zongertinib · HER2 (mutant)
4-week rat: slight to moderate fluctuations in blood, spleen and thymocyte populations

Hyrnuo

sevabertinib · HER2 (mutant)
Thymic atrophy and mesenteric lymph node changes in monkeys

Ibsrela

tenapanor · NHE3
Lymphoid depletion in spleen and thymus in high-dose rat decedents

Ibtrozi

taletrectinib · ROS1
Thymic atrophy and lymph node erythrophagocytosis and foamy macrophages in monkeys

Idvynso

doravirine and islatravir · NNRTI; NRTTI
Dose- and duration-dependent lymphocyte decreases in two mouse strains

Inluriyo

imlunestrant · Estrogen receptor (SERD)
No dedicated immunotoxicity studies; TDAR effects in rats judged non-adverse as immune responses were still mounted

Inqovi

decitabine and cedazuridine · DNMT (decitabine); cytidine deaminase (cedazuridine)
Reversible thymic lymphoid depletion attributed to cedazuridine

Inrebic

fedratinib · JAK2
Bone marrow hypoplasia and lymphocyte depletion (spleen, thymus, lymph nodes) in all species; immunosuppressive

Iqirvo

elafibranor · PPARα/δ
Thymic atrophy in dogs from 30 mg/kg/day (females) and 100 mg/kg/day (males)

Isturisa

osilodrostat · 11β-hydroxylase (CYP11B1)
Strong skin sensitizer (Category 1A) in murine local lymph node assay

Itovebi

inavolisib · PI3Kα (mutant-selective)
Lymphoid depletion in dogs after 4 weeks of dosing

Jascayd

nerandomilast · PDE4B
IL-6 induction by high-dose PDE4 inhibition linked to rat mesenteric vasculitis

Jaypirca

pirtobrutinib · BTK (non-covalent)
Decreased B lymphocytes and TDAR response to KLH; adverse only for the anti-KLH TDAR in male rats

Jesduvroq

daprodustat · HIF prolyl hydroxylase
Thymic lymphoid atrophy in the 13-week monkey study

Jideytro

zidesamtinib · ROS1
Dose-dependent lymphoid depletion (GALT, lymph nodes, spleen, thymus) in dogs, incompletely reversible at 4 mg/kg/day

Joenja

leniolisib · PI3Kδ
Lymphoid atrophy (lymph node, thymus, GALT) and immunosuppression-related skin lesions/infections in rats

Journavx

suzetrigine · NaV1.8
Not a skin sensitizer in the mouse local lymph node assay

Klisyri

tirbanibulin · Tubulin / Src
Some skin sensitisation potential in guinea pig Buehler assay

Komzifti

ziftomenib · Menin-KMT2A
Thymic white blood cell infiltration, vacuolation and lymphoid depletion, persisting after recovery

Koselugo

selumetinib · MEK1/2
Secondary lymphoreticular changes with inflammatory/ulcerative GI findings in mice (~19.5x clinical free AUC)

Krazati

adagrasib · KRAS G12C
Lymphoid tissue changes not considered an immunotoxicity risk; no dedicated immunotoxicity studies

Kygevvi

doxecitine and doxribtimine · Pyrimidine nucleoside replacement (TK2 deficiency)
No hormonal or immune modulation in 26-week juvenile rat and 13-week dog studies

Lampit

nifurtimox · Trypanosoma cruzi
Decreased thymus weights in high-dose rats without correlating histopathology

Lazcluze

lazertinib · EGFR (mutant-selective)
Skin sensitizer in mouse local lymph node assay

Leqselvi

deuruxolitinib · JAK1/JAK2
Reduced WBC and lymphocytes; lymphoid depletion in spleen, thymus, lymph nodes and GALT

Lifyorli

relacorilant · Glucocorticoid receptor (antagonist)
Generalized lymphoid depletion contributed to deaths in 2-week rat studies at ≥200 mg/kg/day

Litfulo

ritlecitinib · JAK3/TEC kinases
Adverse immunosuppression (skin mites, lung inflammation) at 40 mg/kg/day in the 39-week dog study

Livdelzi

seladelpar · PPARδ
No immunotoxicity in repeat-dose studies; negative mouse local lymph node assay

Livtencity

maribavir · CMV UL97 protein kinase
Not immunotoxic in rats up to 100 mg/kg/day

Lumakras

sotorasib · KRAS G12C
Decreased thymus weight and lymphoid cellularity in dogs

Lupkynis

voclosporin · Calcineurin
Immunosuppression: thymic medullary atrophy, absent germinal centers, lymphomas, decreased lymphocytes

Lybalvi

olanzapine and samidorphan · D2/5-HT2A; opioid receptors (samidorphan)
Non-adverse, partially reversible thymus weight decrease with lymphoid depletion in male dogs

Lynkuet

elinzanetant · NK1/NK3 receptors
Thymus atrophy and lymphoid depletion, attributed to treatment-induced stress

Mayzent

siponimod · S1P1/S1P5 receptor
Lymphoid depletion with reduced WBC and lymphocytes in all species, attributed to pharmacology

Miplyffa

arimoclomol · Heat-shock response amplifier
Lymphoid and bone marrow depletion, spleen and thymus atrophy at MTD or higher

Myqorzo

aficamten · Cardiac myosin
Guinea pig dermal sensitisation study (modified Buehler) negative: no erythema after topical application

Nextstellis

drospirenone and estetrol · Estrogen / progestin receptors
Thymus weight reduction with minimal to severe lymphoid atrophy in all species

Nourianz

istradefylline · Adenosine A2A receptor
No antigenicity: no anaphylaxis (ASA/PCA) reactions or drug-specific antibodies

Nubeqa

darolutamide · Androgen receptor
Increased thymus weight in male rats and dogs, possibly reduced thymic involution; no immunotoxicity studies

Nulibry

fosdenopterin · cPMP replacement (MoCD type A)
No antigenicity or immunotoxicity studies conducted

Nurtec ODT

rimegepant · CGRP receptor
No effects on the immune system in pivotal studies

Nuzolvence

zoliflodacin · Bacterial topoisomerase II
One male dog at 500 mg/kg/day euthanized with marked necrosis and inflammation of ileum and gut-associated lymphoid tissue

Ogsiveo

nirogacestat · Gamma secretase
Decreased cellularity of gut-associated lymphoid tissue and mesenteric lymph nodes in rats

Ojemda

tovorafenib · RAF (type II pan-RAF)
Rat: minimal to mild thymus and mesenteric lymph node depletion, not reversed after recovery

Ojjaara

momelotinib · JAK1/JAK2/ACVR1
Hematopoietic and lymphoid tissue targets: bone marrow, spleen, lymph node and thymus

Olinvyk

oliceridine · μ-opioid receptor (G-protein biased)
Stress-related reductions in white blood cells and lymphocytes; minimal thymus atrophy

Ongentys

opicapone · COMT
Slight increase in T-helper lymphocytes in rats, with no functional change (sheep RBC response)

Orgovyx

relugolix · GnRH receptor
No immunotoxicity studies; no immune-system findings in general toxicity studies

Orlynvah

sulopenem etzadroxil and probenecid · Penicillin-binding proteins
Dose-related positive Coombs test in monkeys, likely antibody-mediated minimal erythrocyte loss

Orserdu

elacestrant · Estrogen receptor (SERD)
Macrophage and neutrophilic infiltrates in mesenteric lymph node, lung and intestines of rats and monkeys

Oxbryta

voxelotor · HbS polymerisation
Immunosuppression: opportunistic infections in monkeys, reduced IgG/IgM titres, delayed antibody response

Palsonify

paltusotine · Somatostatin receptor 2
No dedicated immunotoxicity study; no immunotoxicity findings in the nonclinical or clinical program

Pemazyre

pemigatinib · FGFR1-3
Multi-organ mononuclear cell infiltration in rats and monkeys

Piqray

alpelisib · PI3Kα
Inflammation and/or suppression in bone marrow, spleen, thymus and lymph nodes

Ponvory

ponesimod · S1P1 receptor
Reversible suppression of T-cell dependent antibody response during dosing

Pretomanid

pretomanid · Mycobacterial cell wall / respiration
Lymphoid depletion (spleen, thymus, Peyer's patches, BALT) in all three species, at toxic doses

Qelbree

viloxazine · Norepinephrine reuptake
Decreased thymus weight, considered secondary to stress and poor condition; reversible

Qinlock

ripretinib · KIT/PDGFRA
Reduced thymus weight with decreased lymphocytes in dogs

Qulipta

atogepant · CGRP receptor
ICH S8 weight-of-evidence review raised no immunotoxicity concerns

Rapiblyk

landiolol · β1-adrenergic receptor
Decreased thymus weight at the high dose in the rat fertility study

Rasonque

daraxonrasib · RAS(ON) multi-selective
Monkey immune activation with fibrinogen elevation, thymic toxicity and myelosuppression

Retevmo

selpercatinib · RET
Hematopoietic and lymphoid system changes

Revtorpyk

gedatolisib · PI3K/mTOR
Decreased white blood cells and lymphocytes, reduced bone marrow cellularity (rat) and lymphocyte depletion in thymus and spleen

Revuforj

revumenib · Menin-KMT2A
Lymphoid atrophy with decreased thymus weights in dogs at all doses

Reyvow

lasmiditan · 5-HT1F receptor
Splenic marginal-zone lymphoid depletion and thymic involution at high dose in rats

Rezdiffra

resmetirom · Thyroid hormone receptor β
Increased spleen weight in female mice without other immune findings; immunotoxicity risk low

Rezlidhia

olutasidenib · IDH1 (mutant)
Lymph node cellular depletion and increased white blood cells

Rezurock

belumosudil · ROCK2
Thymic lymphoid depletion with reduced thymus weight at high dose; not seen after recovery

Rezzayo

rezafungin · Fungal glucan synthase
Acute transient histamine-release response in rats; mild histamine-like response in monkeys

Rhapsido

remibrutinib · BTK
Inhibited primary antibody responses to some antigens, attributed to BTK role in B cells

Rinvoq

upadacitinib · JAK1
Decreased circulating lymphocytes and lymphoid depletion in spleen, thymus and lymph nodes (JAK inhibition)

Romvimza

vimseltinib · CSF1R
Rat: bone marrow suppression and lymphoid tissue hypocellularity/necrosis at the highest 28-day dose

Rozlytrek

entrectinib · TRK/ROS1/ALK
Lymphoid depletion in rat spleen (≥25 mg/kg/day), lymph nodes and thymus (≥200 mg/kg/day)

Rukobia

fostemsavir · HIV-1 gp120 (attachment)
No immunotoxicity in TDAR assay within the 26-week rat study

Scemblix

asciminib · BCR-ABL1 (STAMP, myristoyl pocket)
Thymic lymphocyte necrosis and splenic lymphoid depletion in one rat at 200 mg/kg/day

Sephience

sepiapterin · Tetrahydrobiopterin precursor
No immune-system effects in repeat-dose studies; no immunotoxicity studies

Skyclarys

omaveloxolone · Nrf2 activator
No dedicated immunotoxicity studies; not required for this small molecule

Sofdra

sofpironium · Muscarinic receptors (topical)
Skin irritant but not a skin sensitizer

Sohonos

palovarotene · RARγ
No evidence of immunosuppression

Sotyktu

deucravacitinib · TYK2
Immunosuppression: lower lymphocyte counts, lymphoid depletion and reduced TDAR to KLH in rats; reduced TDAR in monkeys

Sunlenca

lenacapavir · HIV-1 capsid
Positive in two of three in vitro skin sensitisation assays; negative in vivo

Sunosi

solriamfetol · Dopamine/norepinephrine reuptake
Dose-dependent changes in immune cell counts and immune organ weight/morphology, most prominent in rats

Tabrecta

capmatinib · MET
No specific immunotoxicity studies conducted

Tavneos

avacopan · C5a receptor (C5aR1)
No effect on monkey TDAR, peripheral immunophenotyping or lymphoid organ histology

Tazverik

tazemetostat · EZH2
Monkey: lymphoid hyperplasia in mesenteric lymph node of a single male at 600 mg/kg/day

Tepmetko

tepotinib · MET
No immunotoxic potential in rat and dog repeat-dose studies

Trikafta

elexacaftor, tezacaftor and ivacaftor · CFTR
Lymphoid depletion in the thymus

Truqap

capivasertib · AKT1-3
Decreased white blood cells and lymphocytes in rats

Tryptyr

acoltremon · TRPM8 agonist
Not a skin sensitizer in mouse local lymph node assay

Tukysa

tucatinib · HER2
Lymphoid depletion (hemolymphoid system) in rats

Turalio

pexidartinib · CSF1R
Thymic lymphoid depletion with decreased thymus weight and lymphocytes; lymphoid hyperplasia with lymph node enlargement

Ukoniq

umbralisib · PI3Kδ / CK1ε
Decreased white blood cells and lymphoid depletion

Utebzi

tebipenem pivoxil · Penicillin-binding proteins
Negative in guinea pig delayed hypersensitivity and mouse/guinea pig antigenicity studies

Vafseo

vadadustat · HIF prolyl hydroxylase
No immunotoxic signals in rat and dog repeat-dose studies; no dedicated immunotoxicity studies

Vanflyta

quizartinib · FLT3
Hematologic and lymphoid toxicities from exaggerated pharmacology

Vanrafia

atrasentan · Endothelin A receptor
No immunosuppressive effect in 6-month rat and 9-month dog studies

Veklury

remdesivir · SARS-CoV-2 RNA polymerase
No dedicated immunotoxicity studies; no concerns in repeat-dose studies

Velsipity

etrasimod · S1P1/S1P4/S1P5 receptors
Dose-dependent lymphocyte decreases in rats and dogs; lymphoid depletion in rat lymph nodes and spleen (pharmacological)

Veozah

fezolinetant · Neurokinin-3 receptor
No antigenicity or immunotoxicity studies conducted (not considered necessary)

Verquvo

vericiguat · Soluble guanylate cyclase
No indicators of immune-system effects; no further immunotoxicity evaluation

Vonjo

pacritinib · JAK2/FLT3
Lymphoid tissue depletion with white blood cell changes consistent with bone marrow suppression in mice, rats and dogs

Voranigo

vorasidenib · IDH1/IDH2
Lymphoid organs (lymph nodes, Peyer's patches, spleen, thymus) among rat target organs

Voydeya

danicopan · Complement factor D
Alternative-pathway blockade may impair serum bactericidal activity (meningococcal infection risk) when antibody titers are low

Vtama

tapinarof · Aryl hydrocarbon receptor
Lymphoid depletion with decreased thymus weights in rats at 10 and 40 mg/kg/day subcutaneously

Vyndaqel

tafamidis · Transthyretin (stabiliser)
No specific immunotoxicity studies submitted

Wakix

pitolisant · Histamine H3 receptor
No changes in haematology, immune organ weights/histology or infections in 6-month rat and 9-month monkey studies; not investigated further

Wayrilz

rilzabrutinib · BTK (reversible covalent)
No dedicated immunotoxicity studies submitted

Welireg

belzutifan · HIF-2α
No immunosuppression in repeat-dose toxicity studies

Winlevi

clascoterone · Androgen receptor (topical)
Lymphocyte decreases and lymphoid organ atrophy attributed to weak glucocorticoid activity

Xcopri

cenobamate · Sodium channel / GABA-A
Bone marrow lymphoid hyperplasia in 52-week monkey study, persisting after recovery at high dose

Xdemvy

lotilaner · Mite GABA-Cl channel
Not a skin sensitizer in the mouse local lymph node assay

Xeglyze

abametapir · Lice metalloproteinases
Negative for skin sensitization in mouse LLNA

Xenleta

lefamulin · Bacterial ribosome (50S PTC)
Splenic lymphoid sheath reduction and thymic cortical atrophy in rats after IV dosing

Xocova

ensitrelvir · SARS-CoV-2 3CL protease
20-40% decreases in T- and B-cell populations in 4-week monkey study

Xolremdi

mavorixafor · CXCR4
No immunosuppression in 28-day rat KLH antibody response study

Xpovio

selinexor · XPO1
Mild grade II dermal contact sensitivity response

Zavzpret

zavegepant · CGRP receptor
Non-dose-related lower spleen and thymus weights in monkey without histopathological correlates

Zegfrovy

sunvozertinib · EGFR exon 20 insertion
Immune organs among the main target organs

Zenbexus

iberdomide · Cereblon (CELMoD)
Immune suppression from lymphoid depletion; decreased neutrophils and lymphocytes, matching clinical neutropenia and infections

Zeposia

ozanimod · S1P1/S1P5 receptor
Decreased T and B lymphocytes; suppressed T-dependent IgM and IgG antibody responses

Zepzelca

lurbinectedin · DNA minor groove (transcription)
Lymphocyte and neutrophil decreases suggest a general decrease in immune responsiveness

Zokinvy

lonafarnib · Farnesyltransferase
Lymphoid hypocellularity in spleen, thymus and mesenteric lymph node after 3 months in rats

Ztalmy

ganaxolone · GABA-A receptor (positive modulator)
No antigenicity or immunogenicity studies conducted

Zulresso

brexanolone · GABA-A receptor (positive modulator)
No dermal irritation, skin sensitization or hemolytic potential

Accrufer

ferric maltol · Iron replacement
Not stated in public reviews

Aqneursa

levacetylleucine · Neuronal metabolism (proposed)
Not stated in public reviews

Attruby

acoramidis · Transthyretin (stabiliser)
Not stated in public reviews

Azstarys

serdexmethylphenidate and dexmethylphenidate · Dopamine/norepinephrine reuptake
Not stated in public reviews

Brenzavvy

bexagliflozin · SGLT2
Not stated in public reviews

Caplyta

lumateperone · 5-HT2A / D2 receptor
Not stated in public reviews

Cardamyst

etripamil · L-type calcium channel
Not stated in public reviews

Cypsedo

cipepofol · GABA-A receptor
Not stated in public reviews

Daybue

trofinetide · IGF-1 tripeptide analogue
Not stated in public reviews

Filspari

sparsentan · Endothelin A / angiotensin II receptors
Not stated in public reviews

Foundayo

orforglipron · GLP-1 receptor (non-peptide)
Not stated in public reviews

Gemtesa

vibegron · β3-adrenergic receptor
Not stated in public reviews

Inpefa

sotagliflozin · SGLT1/SGLT2
Not stated in public reviews

Kerendia

finerenone · Mineralocorticoid receptor
Not stated in public reviews

Livmarli

maralixibat · IBAT (ASBT)
Not stated in public reviews

Lynavoy

linerixibat · IBAT
Not stated in public reviews

Lytgobi

futibatinib · FGFR1-4 (covalent)
Not stated in public reviews

Miebo

perfluorohexyloctane · Tear film evaporation barrier
Not stated in public reviews

Modeyso

dordaviprone · DRD2 antagonist / ClpP agonist
Not stated in public reviews

Nereus

tradipitant · NK1 receptor
Not stated in public reviews

Nexletol

bempedoic acid · ATP citrate lyase
Not stated in public reviews

Ohtuvayre

ensifentrine · PDE3/PDE4
Not stated in public reviews

Omlonti

omidenepag isopropyl · EP2 receptor
Not stated in public reviews

Orzeyful

oveporexton · Orexin OX2 receptor agonist
Not stated in public reviews

Paxlovid

nirmatrelvir and ritonavir · SARS-CoV-2 Mpro (3CL protease)
Not stated in public reviews

Pizensy

lactitol · Osmotic laxative
Not stated in public reviews

Pyrukynd

mitapivat · Pyruvate kinase (activator)
Not stated in public reviews

Quviviq

daridorexant · Orexin OX1/OX2 receptors
Not stated in public reviews

Recarbrio

imipenem, cilastatin and relebactam · Beta-lactamase (relebactam)
Not stated in public reviews

Relyvrio

sodium phenylbutyrate and taurursodiol · ER stress / mitochondrial (proposed)
Not stated in public reviews

Simtriyo

centanafadine · Triple monoamine reuptake
Not stated in public reviews

Truseltiq

infigratinib · FGFR1-3
Not stated in public reviews

Tryvio

aprocitentan · Endothelin A/B receptors
Not stated in public reviews

Ubrelvy

ubrogepant · CGRP receptor
Not stated in public reviews

Veppanu

vepdegestrant · Estrogen receptor (PROTAC degrader)
Not stated in public reviews

Vivjoa

oteseconazole · Fungal CYP51
Not stated in public reviews

Vizz

aceclidine · Muscarinic agonist (miotic)
Not stated in public reviews

Voquezna

vonoprazan · H+/K+-ATPase (potassium-competitive)
Not stated in public reviews

Xacduro

sulbactam and durlobactam · PBPs; beta-lactamase (durlobactam)
Not stated in public reviews

Zaynich

cefepime and zidebactam · PBPs; beta-lactam enhancer (zidebactam)
Not stated in public reviews

Zevtera

ceftobiprole medocaril · Penicillin-binding proteins
Not stated in public reviews

Zurzuvae

zuranolone · GABA-A receptor (positive modulator)
Not stated in public reviews

Zycubo

copper histidinate · Copper replacement (Menkes disease)
Not stated in public reviews

Summary across all 211 small molecules →