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Atlas/Small molecules/Trikafta/Risks

Trikafta: which risks did reviewers probe?

elexacaftor, tezacaftor and ivacaftor · CFTR
Check my plan vs. these
Immune & cytokine safety
Lymphoid depletion in the thymus
“Microscopic correlates consisted of cortical hypertrophy/vacuolation in the adrenal glands, atrophy in the prostate, secretory depletion in the seminal vesicles/coagulating glands, germ cell debris/oligospermia in the epididymides, and lymphoid depletion in the thymus.”
✓ VerifiedEMA assessment report, p. 43Source ↗
Biodistribution & persistence
Elexacaftor crosses blood-brain barrier (brain ~3-fold below plasma); no preferential melanin binding
“VX-445-related radioactivity crossed the blood-brain barrier, but measured concentrations in the brain were about 3-fold lower relative to plasma. A tissue distribution study in Long-Evans rats showed no significant distribution to melanin-containing tissues (skin, eyes), indicating that VX-445 did not preferentially associate with melanin.”
✓ VerifiedEMA assessment report, p. 39Source ↗
Modality-specific risk
Major active human metabolite M23 with low nonclinical exposure margins (MIST)
“Levels of M23 formed in nonclinical species were generally lower than in humans, resulting in relatively low exposure margins in some cases.”
✓ VerifiedFDA multi-discipline review, p. 52Source ↗

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

Orladeyo

berotralstat · Plasma kallikrein
Foamy macrophages/histiocytes in spleen, thymus and intestine (phospholipidosis)

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

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 →