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Acoramidis
go.drugbank.com/drugs/DB17999ApprovedInvestigationalAcoramidis is a small molecule stabilizer of transthyretin (TTR) for use in patients with TTR amyloidosis. … [A264768] Although they share a mechanism of action, acoramidis is more selective for TTR and is a more potent stabilizer when compared to tafamidis.
Dextrothyroxine
go.drugbank.com/drugs/DB00509ApprovedWithdrawnThyroxine is peripherally deiodinated to form triiodothyronine which exerts a broad spectrum of stimulatory effects on cell metabolism. … Thyroxine is synthesized via the iodination of tyrosines (monoiodotyrosine) and the coupling of iodotyrosines (diiodotyrosine) in the thyroglobulin.
Difelikefalin
go.drugbank.com/drugs/DB11938ApprovedInvestigational[A237615,A237620] The clinical burden of uremic pruritus in this patient population is being increasingly recognized as contributing to a significant reduction in patient quality of life, poor outcomes … [A237610] These revelations led to the study of KOR agonists as a potential treatment option in patients suffering from pruritic conditions.
Leniolisib
go.drugbank.com/drugs/DB16217ApprovedInvestigational[L45758] APDS is a primary immunodeficiency caused by mutations in genes encoding the PI3Kδ, thereby increasing the activity of PI3Kδ, causing immune dysfunction, and elevating susceptibility to infections … Leniolisib is a potent and selective inhibitor of phosphoinositide 3-kinase δ (PI3Kδ).
Talimogene laherparepvec
go.drugbank.com/drugs/DB13896ApprovedInvestigationalTalimogene laherparepvec is an oncolytic treatment used in local treatment of unresectable cutaneous, subcutaneous, and nodal lesions in patients with recurrent melanoma. … It is a genetically administered herpes simplex virus 1 (HSV-1) that expresses human cytokine granulocyte-macrophage colony stimulating factor (GM-CSF) with antitumor and immune-stimulating activities.
Defactinib
go.drugbank.com/drugs/DB12282ApprovedInvestigational[L53208,A273943] Defactinib was approved by the US FDA in May 2025 in a co-package alongside [avutometinib] (called Avmapki Fakzynja) for the treatment of recurrent KRAS-mutated low-grade serous ovarian … [L53198,L53208] FAK is important for the integrin-mediated activation of several downstream signal transduction pathways, including those involving RAS/MEK/ERK and PI3K/Akt, and its upregulation is a key
Synonyms: N-methyl-4-[[4-[[3-[methyl(methylsulfonyl)amino]pyrazin-2-yl]methylamino]-5-(trifluoromethyl)pyrimidin-2-yl]amino]benzamidePancrelipase amylase
go.drugbank.com/drugs/DB11065ApprovedInvestigationalPancrelipase, in general, is composed of a mixture of pancreatic enzymes which include amylases, lipases, and proteases. These enzymes are extracted from porcine pancreatic glands. … [T177] The pancrelipase amylase is a single polypeptide chain containing two SH groups and four disulfide bridges.
Mixtures: KREON MIKRO 5000 IU MINIMIKROSFER,Synonyms: Amylase AAP5280
go.drugbank.com/drugs/DB05112ExperimentalAP5280 is a novel N-(2-hydroxypropyl)methacrylamide (HPMA) copolymer-bound platinum (Pt) therapeutic designed to increase the therapeutic index relative to conventional, small-molecule platinum agents.
Vilobelimab
go.drugbank.com/drugs/DB16416ApprovedInvestigational[L45839] In April 2023, the FDA issued an emergency use authorization (EUA) for vilobelimab for the treatment of COVID-19 in hospitalized adults requiring mechanical ventilation or artificial life support … Vilobelimab is a chimeric monoclonal immunoglobulin G4 (IgG4) antibody that binds to the soluble form of human C5a with high affinity.
Allogeneic processed thymus tissue
go.drugbank.com/drugs/DB16736ApprovedAllogeneic processed thymus tissue is used for immune reconstitution in immune disorders where the patients are severely immunocompromised due to the absence of a functional thymus, such as congenital … [A239814] On October 8, 2021, FDA approved allogeneic processed thymus tissue-agdc, under the market name RETHYMIC, as a one-time regenerative tissue-based therapy for immune reconstitution in pediatric