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Xanomeline
go.drugbank.com/drugs/DB15357ApprovedInvestigationalSchizophrenia is a complex disease involving a number of different neurotransmitters, including serotonin, dopamine, and acetylcholine. … [L51713] It is approved as part of a combination product alongside [trospium], a muscarinic antagonist that acts primarily on peripheral muscarinic receptors in order to mitigate the risk and severity
Voclosporin
go.drugbank.com/drugs/DB11693ApprovedInvestigational[L31218] This [cyclosporine] A analog was approved by the FDA on January 22, 2021 following promising results in clinical trials. … LN is a significant cause of renal failure, morbidity, and death in patients with SLE.
Metreleptin
go.drugbank.com/drugs/DB09046ApprovedInvestigationalLipodystrophies include a range of disorders characterized by the reduction, absence, or altered distribution of adipose tissue. … Metreleptin is produced in _E. coli_ and differs from native human leptin by the addition of a methionine residue at its amino terminus.
Conestat alfa
go.drugbank.com/drugs/DB09228ApprovedC1 Esterase Inhibitor (Recombinant) is a recombinant analogue of endogenous complement component-1 esterase inhibitor (rhC1INH), purified from the milk of transgenic rabbits. … Marketed as the product Ruconest (FDA), this drug is indicated for the treatment of acute attacks of hereditary angioedema (HAE) due to C1 esterase inhibitor deficiency in adults.
Daratumumab
go.drugbank.com/drugs/DB09331ApprovedInvestigational[A199002] Daratumumab was granted FDA approval on 16 November 2015. … In July 2024, it was further approved in combination with bortezomib, lenalidomide, and dexamethasone (D-VRd) for induction and consolidation therapy in newly diagnosed, transplant-eligible patients.
Odronextamab
go.drugbank.com/drugs/DB16684ApprovedInvestigational[L43887,L43892] A phase I clinical study (NCT02290951) showed that in heavily pre-treated patients with B-cell non-Hodgkin lymphoma, odronextamab elicited a durable response with a manageable safety … [A254187] In December 2020, the FDA placed a partial clinical hold and requested the drug developer, Regeneron Pharmaceuticals, to amend clinical protocols to reduce the incidence of CRS.
Anifrolumab
go.drugbank.com/drugs/DB11976ApprovedInvestigational[A237044] Anifrolumab was granted FDA approval on 30 July 2021.[L34929] … Anifrolumab, or MEDI-546, is a type 1 interferon receptor (IFNAR) inhibiting IgG1κ monoclonal antibody indicated in the treatment of adults with moderate to severe systemic lupus erythematosus.
Velmanase alfa
go.drugbank.com/drugs/DB12374ApprovedInvestigationalAlpha-mannosidosis is a rare autosomal recessive lysosomal storage disorder. … 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.
SPC2968
go.drugbank.com/drugs/DB06351InvestigationalIt was under investigation in clinical trial NCT02564614 (A Study of Hypoxia-inducible Factor 1a (HIF1A) Messenger Ribonucleic Acid (mRNA) Antagonist (RO7070179), to Demonstrate Proof-of-mechanism in Adult … SPC2968 is a synthetic antisense oligodeoxynucleotide targeting hypoxia-inducible factor-1alpha (HIF-1alpha).
VTX-801
go.drugbank.com/drugs/DB17646InvestigationalVTX-801 is a replication-deficient recombinant adeno-associated viral vector (rAAV) consisting of an AAV liver tropic capsid containing a single-stranded DNA genome carrying a shortened version of the