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Cysteamine
go.drugbank.com/drugs/DB00847ApprovedInvestigational[A218721] Several preparations of cysteamine exist for the treatment of cystinosis manifestations, some in capsule form, and others in ophthalmic solution form. … A defect in cystinosin function is followed by cystine accumulation throughout the body, especially the eyes and kidneys.
Fenofibric acid
go.drugbank.com/drugs/DB13873ApprovedInvestigationalFenofibric acid is a lipid-lowering agent that is used in severe hypertriglyceridemia, primary hyperlipidemia, and mixed dyslipidemia. … It works to decrease elevated low-density lipoprotein cholesterol, total cholesterol, triglycerides, apolipoprotein B, while increasing high-density lipoprotein cholesterol.
Clofazimine
go.drugbank.com/drugs/DB00845ApprovedInvestigationalWithdrawnIt was originally described in 1957 and was the prototypical riminophenazine dye - a bright-red dye that, in its clinical use, results in long-lasting discoloration of the skin and bodily fluids. … [A203144] Although it carries _in vitro_ activity against other mycobacterium, such as _Mycobacterium tuberculosis_, it is generally considered an ineffective treatment in comparison to classic tuberculosis
Linerixibat
go.drugbank.com/drugs/DB11729ApprovedInvestigationalLinerixibat is a first-in-class and small-molecule inhibitor of the ileal bile acid transporter (IBAT). … [A275481, A275482] On March 19, 2026, linerixibat was approved by the FDA for the treatment of cholestatic pruritus in patients with primary biliary cholangitis (PBC).
Ethylhexyl methoxycrylene
go.drugbank.com/drugs/DB11226ApprovedIt currently enjoys regional approvals in Canada, the EU, and the US [L2847]. … It is used as a solvent for crystalline UV filters and is easily emulsified and suitable for use in various lotions, creams, sprays, sticks, and gel formulations [F104].
Polatuzumab vedotin
go.drugbank.com/drugs/DB12240ApprovedInvestigationalPolatuzumab vedotin is a CD79b-directed antibody-drug conjugate that delivers monomethyl auristatin E (MMAE), an anti-mitotic agent, to cancer cells. … [L6658] Polatuzumab vedotin was granted accelerated FDA approval on June 10, 2019 [A254936] and was approved by Health Canada on July 9, 2020.[L44141]
Baclofen
go.drugbank.com/drugs/DB00181ApprovedInvestigationalAlthough originally designed in 1962 to treat epilepsy, baclofen was not effective in treating this condition but instead was shown to reduce spasticity in selected patients. … [A245338] Baclofen was reintroduced in 1971 as a treatment for spasticity and was later approved by the FDA in 1977.
Motixafortide
go.drugbank.com/drugs/DB14939ApprovedInvestigational[A261476] Motixafortide was approved by the FDA in September 2023, in combination with filgrastim, for use in stem cell mobilization prior to autologous stem cell transplant in patients with multiple … [L48116] When administered alongside [filgrastim], another agent used to aid in stem cell collection, motixafortide enabled the collection of an adequate number of stem cells in ~92% of patients within
Orforglipron
go.drugbank.com/drugs/DB18964ApprovedInvestigationalOrforglipron is a first-in-class oral, non-peptide glucagon-like peptide-1 (GLP-1) receptor agonist. … [L56123] On April 1, 2026, the US FDA granted approval to orforglipron for use alongside diet and exercise to aid in weight reduction in certain patient populations.
Inavolisib
go.drugbank.com/drugs/DB15275ApprovedInvestigational[L51748,L51738] In February 2025, it was approved by Health Canada for the same indication. [L52605] … _PIK3CA_ is one of the most frequently mutated oncogenes, with the resulting mutated p110α protein it encodes playing a central role in tumor cell proliferation.