Drug intelligence / Profile preview

hydroxychloroquine

Development stage
Approved
Lead developer
Sanofi
Modality
Small Molecules
Administration
Oral, Inhalation
01

Overview

Hydroxychloroquine is a 4-aminoquinoline derivative originally developed as an antimalarial agent but now widely utilized as a disease-modifying antirheumatic drug (DMARD) for autoimmune conditions such as systemic lupus erythematosus and rheumatoid arthritis. Its mechanism of action involves the inhibition of endosomal Toll-like receptors (specifically TLR7 and TLR9) and the stabilization of lysosomal membranes by increasing lysosomal pH. Beyond its established roles in rheumatology, hydroxychloroquine is investigated as an autophagy inhibitor in oncology to overcome chemotherapy resistance in cancers like breast cancer and small cell lung cancer. It has also shown potential protective effects in age-related macular degeneration (AMD) by reducing drusen formation and is being explored in aerosolized formulations for respiratory infections.

Brand names
PlaquenilQuineprox
Other names
hydroxychloroquine sulfateHCQHCQS
02

Targets

PPT1 (Palmitoyl-protein thioesterase 1)PTGS2 (Prostaglandin-Endoperoxide Synthase 2)TLR7 (Toll-like receptor 7)TLR8 (Toll-like receptor 8)ACE2 (Angiotensin-converting enzyme 2)TLR9 (Toll-like receptor 9)β-Hematin

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