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Phenobarbital
go.drugbank.com/drugs/DB01174ApprovedInvestigationalA barbituric acid derivative that acts as a nonselective central nervous system depressant.
Categories: P-glycoprotein substrates with a Narrow Therapeutic Index, Cytochrome P-450 CYP2C18 Substrates with a Narrow Therapeutic IndexMirdametinib
go.drugbank.com/drugs/DB07101ApprovedInvestigationalMirdametinib is a mitogen-activated protein kinase (MAP2K, MEK, MAPKK) inhibitor. On February 11, 2025, mirdametinib was approved by the FDA for the treatment of neurofibromatosis type 1 (NF1). … [L52485] NF1 is an autosomal-dominant genetic condition caused by loss-of-function variants in the _NF1_ tumour suppressor gene,[A265065,A265070] leading to neurofibromin dysfunction and aberrant activation
MPI-674
go.drugbank.com/drugs/DB05749ExperimentalMPI-674 is an aromatase inhibitor (AI) with a well-established, multi-year chronic safety and tolerability profile. … AIs are a class of drugs that reduce the amount of estrogen circulating in the body by binding to and inhibiting the enzyme aromatase, which is responsible for converting certain hormones to estrogen.
Risankizumab
go.drugbank.com/drugs/DB14762ApprovedInvestigationalRisankizumab is a fully humanized IgG1 monoclonal antibody (mAb) directed against interleukin 23 (IL-23).
Etuvetidigene Autotemcel
go.drugbank.com/drugs/DB17593ApprovedInvestigational[L54693] WAS is a rare X-linked disorder caused by a mutation in the gene encoding the WAS protein, leading to a compromised immune system. … Etuvetidigene autotemcel is an autologous gene therapy which includes hematopoietic stem cells (HSCs) that have been genetically modified ex vivo.
Gedatolisib
go.drugbank.com/drugs/DB11896ApprovedInvestigational[L63927] Its approval also addresses a population defined by the absence of a biomarker, as patients with PIK3CA wild-type disease were not eligible for the PI3Kα-selective options. … [L63796,L63927] The PI3K/AKT/mTOR pathway is a long-standing target in the breast cancer setting, with previously approved agents acting on a single node of it (i.e. PI3Kα, AKT, or mTORC1).
Toremifene
go.drugbank.com/drugs/DB00539ApprovedInvestigationalToremifene is a selective estrogen receptor modulator (SERM) and a nonsteroidal antiestrogen used to treat estrogen receptor positive breast cancer.
Cedazuridine
go.drugbank.com/drugs/DB15694ApprovedInvestigational[A215082, A215092, A215097] Hypomethylating agents such as [decitabine] and [azacitidine] are used to treat MDS through inducing DNA hypomethylation and apoptosis of cancerous cells. … Myelodysplastic syndromes (MDS) are a group of hematopoietic neoplasms that give rise to variable cytopenias progressing to secondary acute myeloid leukemia (sAML), which is invariably fatal if untreated
Riltozinameran
go.drugbank.com/drugs/DB17095ApprovedInvestigationalThe Omicron variant of SARS-CoV-2 is a variant of concern that was first reported in November 2021. … [L43917] Riltozinameran is an mRNA vaccine encoding the S glycoprotein of SARS-CoV-2 Omicron variant lineage BA.1.
Migalastat
go.drugbank.com/drugs/DB05018ApprovedInvestigationalFabry disease is a rare, progressive genetic disorder characterized by a defective GLA gene that causes a deficiency in the enzyme alpha-Galactosidase A (alpha-Gal A). … unmet medical need and where a drug is shown to have certain effects that are reasonably likely to predict a clinical benefit to patients.
Categories: Alpha-Galactosidase A (alpha-Gal A) Pharmacological Chaperones