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Efgartigimod alfa
go.drugbank.com/drugs/DB15270ApprovedInvestigational[A243759,A243784] Efgartigimod alfa is a first-in-class[L39501] antagonist of the neonatal Fc receptor (FcRn) used in the treatment of MG. … and a reduction in electrical nerve impulses.
Tividenofusp alfa
go.drugbank.com/drugs/DB17628ApprovedInvestigationalTividenofusp alfa is a first-in-class, brain-penetrant enzyme replacement therapy (ERT) indicated for the treatment of Mucopolysaccharidosis type II (MPS II), also known as Hunter syndrome [L56097, L56099 … Hunter syndrome is a rare, X-linked lysosomal storage disorder caused by a deficiency in the enzyme iduronate-2-sulfatase (IDS), which leads to the toxic accumulation of glycosaminoglycans (GAGs) throughout
Melphalan
go.drugbank.com/drugs/DB01042ApprovedInvestigational[L40928] It was first synthesized in the early 1950s by substituting L-phenylalanine for the methyl group on nitrogen mustard. … [A261150, A261155] Melphalan is used in the treatment of multiple myeloma and ovarian carcinoma.[L40928] It is also used for high-conditioning before hematopoietic stem cell transplant.
Synonyms: p-N,N-bis(2-chloroethyl)amino-L-phenylalanine, p-N-Bis(2-chloroethyl)amino-L-phenylalanineVelmanase alfa
go.drugbank.com/drugs/DB12374ApprovedInvestigational[A244509] In February 2023, the FDA also approved velmanase alfa for the same indication.[L45295] Velmanase alfa is currently not approved in Canada. … Patients with alpha-mannosidosis have a genetic mutation that causes a deficiency in the lysosomal enzyme alpha-mannosidase, which is an enzyme responsible for breaking down complex sugars in the body.
Eladocagene exuparvovec
go.drugbank.com/drugs/DB16780ApprovedInvestigational[A253687] It was subsequently approved by the FDA in November 2024.[L51898,L51893] … [L43642] Eladocagene exuparvovec received EMA approval in July 2022.
Macitentan
go.drugbank.com/drugs/DB08932ApprovedInvestigationalMacitentan is a dual endothelin receptor antagonist used in the treatment of pulmonary arterial hypertension (PAH).[L35890] It was first approved by the FDA in 2013. … A combination product (Opsynvi) comprising macitentan and [tadalafil] was approved in Canada in October 2021 for the treatment of PAH.[L39105] It was subsequently approved by the FDA in March 2024.
Homatropine
go.drugbank.com/drugs/DB11181ApprovedIt is present in antitussives, under the trade name Hycodan, in combination with hydrocodone (dihydrocodeinone) bitartrate indicated for the symptomatic relief of cough as oral tablets or solutions. … Homatropine is included in subtherapeutic amounts as homatropine methylbromide to discourage deliberate overdosage.
Voxelotor
go.drugbank.com/drugs/DB14975Approved[T734] Voxelotor was granted accelerated FDA approval on November 25 2019, as it is likely to be a promising treatment for the 100,000 individuals in the U.S. suffering from the disease, in addition … This is a genetically inherited condition most prevalent in the Middle East, Africa, and certain parts of India.
Lecanemab
go.drugbank.com/drugs/DB14580ApprovedInvestigational[A253947,A253957] In clinical trials, it significantly reduced brain Aβ plaques compared to placebo. … Lecanemab is a recombinant humanized immunoglobulin gamma 1 (IgG1) monoclonal antibody directed against aggregated soluble and insoluble forms of amyloid beta (Aβ), which are implicated in the pathophysiology
Medrogestone
go.drugbank.com/drugs/DB09124ApprovedWithdrawnIt was conceived as an alternative for an orally effective contraceptive option.[A31526] It was developed by Ayerst, approved in Canada in 1969 and its current status is cancelled post-marketing. … [L1118] It was never approved by the FDA.