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Triheptanoin
go.drugbank.com/drugs/DB11677ApprovedInvestigational[A214817] Triheptanoin was granted FDA approval on 30 June 2020.[L14612] … Triheptanoin is a source of heptanoate fatty acids, which can be metabolized without the enzymes of long chain fatty acid oxidation.
Afamelanotide
go.drugbank.com/drugs/DB04931ApprovedFDA approval in October 2019. … [L9092] Despite its relatively recent approval, afamelanotide has been available for use as an orphan drug in both the US and EU since 2008.[L9122,L9125]
Niraparib
go.drugbank.com/drugs/DB11793ApprovedInvestigationalNiraparib is an orally active poly (ADP-ribose) polymerase (PARP) inhibitor. By blocking the enzymes responsible for DNA repair, niraparib induces cytotoxicity in cancer cells. … [A253248] First approved by the FDA on March 27, 2017,[A253912] niraparib is used to treat epithelial ovarian, fallopian tube, or primary peritoneal cancer.
Dabrafenib
go.drugbank.com/drugs/DB08912ApprovedInvestigational[L41955] In May 2018, Tafinlar (dabrafenib), in combination with Mekinist ([DB08911]), was approved to treat anaplastic thyroid cancer caused by an abnormal BRAF V600E gene.[L41955]
Ofatumumab
go.drugbank.com/drugs/DB06650ApprovedInvestigational[A6921] Ofatumumab was first approved by the FDA in 2009. … [L12753] The FDA subsequently approved ofatumumab for the treatment of RMS on August 20, 2020.
Voretigene neparvovec
go.drugbank.com/drugs/DB13932ApprovedInvestigational[A31478] VN-rzyl was developed by Spark Therapeutics Inc. and FDA approved on December 19, 2017.[L1095] … Voretigene Neparvovec-rzyl (VN-rzyl) is an adeno-associated virus vector-based gene therapy.[L1094] An adeno-associated virus is a small virus that infects humans and other primates.
Metreleptin
go.drugbank.com/drugs/DB09046ApprovedInvestigational[L41315] In February 2014, metreleptin was approved by the FDA for the treatment of complications of leptin deficiency, as an adjunct to diet, in patients with congenital generalized or acquired generalized … Metreleptin, a recombinant analog of the human hormone leptin, is an orphan drug used to treat complications of leptin deficiency in people with lipodystrophy.
Amikacin
go.drugbank.com/drugs/DB00479ApprovedInvestigationalVet approvedof patients with the disease who do not respond to traditional treatment. … [L4680] Several forms of amikacin are used currently, including an intravenous (IV) or intramuscular (IM) injection.
Synonyms: D-STREPTAMINE, O-3-AMINO-3-DEOXY-.ALPHA.-D-GLUCOPYRANOSYL-(1->6)-O-(6-AMINO-6-DEOXY-.ALPHA.-D-GLUCOPYRANOSYL-(1->4))-N(SUP 1)-(4-AMINO-2-HYDROXY-1-OXOBUTYL)-2-DEOXY-, (S)-, -D-GLUCOPYRANOSYL-(1->4)-O-(6-AMINO-6-DEOXY-.ALPHA.-D-GLUCOPYRANOSYL-(1->6))-N(SUP 3)-(4-AMINO-L-2-HYDROXYBUTYRYL)-2-DEOXY-L-STREPTAMINETemozolomide
go.drugbank.com/drugs/DB00853ApprovedInvestigational[L32033] Temozolomide was granted FDA approval on August 11, 1999, as an oral capsule and subsequently on February 27, 2009, as an intravenous injection. … [A229853, A229923] Temozomolide as an adjunct to radiotherapy followed by maintenance dosing remains the standard of care for both Glioblastoma and refractory anaplastic astrocytoma.
Synonyms: 8-carbamoyl-3-methylimidazo(5,1-d)-1,2,3,5-tetrazin-4(3H)-one, 3,4-dihydro-3-methyl-4-oxoimidazo(5,1-d)-as-tetrazine-8-carboxamideMetamfetamine
go.drugbank.com/drugs/DB01577ApprovedIllicitInvestigationalWithdrawnIt is a scheduled drug in most countries due to its high potential for addiction and abuse. … The FDA withdrew its approval for the use of all parenteral drug products containing methamphetamine hydrochloride, a metamfetamine salt.[L43942]
Synonyms: d-deoxyephedrine, d-desoxyephedrine