U.S. Department of Health & Human Services Divider Arrow National Institutes of Health Divider Arrow NCATS

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Showing 11 - 20 of 13230 results

Sodium artesunate, an artemisinin derivative, is used in malaria treatment. Artesunate, has been licensed in Thailand for the treatment of falciparum malaria since 1990. It is a potent antimalarial drug that can reduce parasitaemia by 90% within 24 h of administration. Sodium artesunate was first isolated in China, it is a water soluble antimalaria used clinically in China.
Allopregnanolone is a neurosteroid metabolite of progesterone. It is an allosteric modulator of inhibitory γ-aminobutyric acid (GABA-A) receptors on neural stem cells and other cell types in the brain. Allopregnanolone has effects similar to those of other positive allosteric modulators of the GABA action at GABAA receptor such as the benzodiazepines, including anxiolytic, sedative, and anticonvulsant activity. A solution of allopregnanolone, SAGE-547 is an intravenous allosteric modulator of both synaptic and extrasynaptic γ-aminobutyric acid type A (GABAA)receptors. It's believed that allopregnanolone is effective as an anticonvulsant when prolonged seizure activity has become resistant to benzodiazepine treatment. Under the names brexanolone and SAGE-547, allopregnanolone is under development by SAGE Therapeutics as an intravenously administered drug for the treatment of super-refractory status epilepticus, postpartum depression, and essential tremor. Allopregnanolone is in phase III trials for the treatment of super-refractory status epilepticus (SRSE) and postpartum depression.

Class (Stereo):
CHEMICAL (ABSOLUTE)

Cenobamate (also known as YKP3089) is a small molecule sodium channel blocker in development for the treatment of partial-onset seizures in adult patients. In mice and rats, Cenobamate displayed an anticonvulsant activity in the maximal electroshock test and prevented seizures induced by chemical convulsants such as pentylenetetrazol and picrotoxin. In addition, Cenobamate was reported to be effective in two models of focal seizure, the hippocampal kindled rat and the mouse 6 Hz psychomotor seizure models. Two completed adequate and well-controlled clinical studies demonstrated a significant reduction in focal seizures with Cenobamate in patients with epilepsy, and a long-term open-label phase 3 safety clinical trial is currently ongoing. Cenobamate is considered a new generation antiepileptic therapy and clinical trials have shown that it may be more effective and safer than existing drugs. If licensed, Cenobamate will offer a new adjunctive treatment option for patients with partial focal epilepsy.
Bremelanotide (formerly PT-141) was developed for the treatment of female sexual dysfunction, hemorrhagic shock, and reperfusion injury. Bremelanotide, a synthetic peptide analog of α-melanocyte-stimulating hormone (α-MSH) is an agonist at melanocortin receptors including the MC3R and MC4R, which are expressed primarily in the central nervous system. Bremelanotide originally was tested for intranasal administration in treating female sexual dysfunction but this application was temporarily discontinued in 2008 after concerns were raised over adverse side effects of increased blood pressure. It appears that development for hemorrhagic shock and reperfusion injury has been discontinued. Palatin Technologies licensed North American development and commercialization rights of bremelanotide to Amag in January 2017. In June 2018, the US Food and Drug Administration (FDA) accepted AMAG Pharmaceuticals’ new drug application for bremelanotide for treatment of hypoactive sexual desire disorder in premenopausal women. If approved, bremelanotide will be available as a self-administered, disposable subcutaneous auto-injector used in anticipation of a sexual encounter.

Class (Stereo):
CHEMICAL (ABSOLUTE)

Afamelanotide (SCENESSE) is a synthetic α-melanocyte stimulating hormone analog and first-in-class melanocortin-1 receptor agonist that is approved in the EU for the prevention of phototoxicity in adults with erythropoietic protoporphyria. Afamelanotide differs from endogenous α-melanocyte stimulating hormone at the fourth and seventh amino acid residues, increasing its resistance to immediate degradation and increasing its binding time to melanocortin-1 receptor. Afamelanotide is mimic the pharmacological activity of α-melanocyte stimulating hormone by binding to the melanocortin-1 receptor on melanocytes and activating the synthesis of eumelanin. Eumelanin provides photoprotection through mechanisms including, but not limited to, the absorption and scattering of visible and UV light and antioxidant activity. Afamelanotide increases eumelanin density in healthy volunteers and patients with erythropoietic protoporphyria. In healthy, fair-skinned volunteers, a significant increase in melanin density and skin darkening in both sun-exposed and non-sun-exposed sites was seen with subcutaneous injections of afamelanotide. The most common afamelanotide adverse events included headache and nausea. Common adverse effects include back pain, upper respiratory tract infections, decreased appetite, migraine, and dizziness.
Netarsudil ophthalmic solution (Rhopressa) is a Rho kinase inhibitor for the treatment of open-angle glaucoma or ocular hypertension. As of December 18, 2017 the FDA approved Aerie Pharmaceutical's Rhopressa (netarsudil ophthalmic solution) 0.02% for the indication of reducing elevated intraocular pressure in patients with open-angle glaucoma or ocular hypertension. Acting as both a rho kinase inhibitor and a norepinephrine transport inhibitor, Netarsudil is a novel glaucoma medication in that it specifically targets the conventional trabecular pathway of aqueous humour outflow to act as an inhibitor to the rho kinase and norepinephrine transporters found there as opposed to affecting protaglandin F2-alpha analog like mechanisms in the unconventional uveoscleral pathway that many other glaucoma medications demonstrate.
(+)-alpha-Dihydrotetrabenazine (HTBZ) is an active component of tetrabenazine. Tetrabenazine is a mixture of closely-related compounds (isomers) and is readily metabolized in the human body to HTBZ and related isomers. Tetrabenazine is a drug for the symptomatic treatment of hyperkinetic movement disorder and is marketed under the trade names Nitoman in Canada and Xenazine in New Zealand and some parts of Europe, and is also available in the USA as an orphan drug. (+)-alpha-Dihydrotetrabenazine and related benzo[a]quinolizines have been labeled with tritium and carbon-11 radioisotopes and used for in vitro and in vivo studies of the VMAT2 in animal and human brain. Adeptio Pharmaceuticals is developing alpha-dihydrotetrabenazine (HTBZ) for the treatment of neurological disorders. It acts by inhibiting vesicular monoamine transporter 2 (VMAT2), thereby blocking the transport of dopamine into axon terminals or into storage vesicles.

Class (Stereo):
CHEMICAL (RACEMIC)



Secnidazole (trade names Flagentyl, Sindose, Solosec) is a nitroimidazole derivative used to in the treatment of amoebiasis and bacterial vaginosis. Secnidazole and other 5-nitroimidazole drugs enter micro-organisms by passive diffusion and undergo activation by reduction of the 5-nitro group. In anaerobic micro-organisms, such as Trichomonas, Giardia and Entamoeba spp., this intracellular reduction occurs via the pyruvate ferredoxin oxidoreductase complex and results in a concentration gradient across the cell membrane which, in tum, enhances transport of the parent drug into the cell. Because the electron affinity of the 5-nitroimidazoles is greater than that of reduced ferredoxin, the drug interrupts the normal electron flow. Aerobic micro-organisms have a more positive redox potential (i.e. are more efficient electron acceptors) than secnidazole and other 5-nitroimidazoles, which explains the selective toxicity of these drugs against anaerobic microorganisms. DNA is the intracellular target of the Secnidazole and other 5-nitroimidazoles. Secnidazole and other 5-nitroimidazoles possess selective activity against many anaerobic Gram-positive and Gram-negative bacteria and protozoa. In general, secnidazole and metronidazole were approximately equipotent in activity against Bacteroides fragilis, Trichomonas vaginalis, and Entamoeba histolytica, in in vitro studies. Secnidazole is rapidly and completely absorbed after oral administration. Plasma drug concentrations are linear over the therapeutic dose range of 0.5 to 2g. The tolerability profile of secnidazole does not differ markedly from other 5-nitroimidazoles. The most commonly reported adverse events in clinical trials involved the gastrointestinal tract (nausea, vomiting, glossitis, anorexia, epigastric pain and a metallic taste) and occurred in 2 to 10% of patients. A headache and dizziness were experienced by about 2% of patients. The drug was equally well tolerated in adults and children, and no adverse event required therapeutic intervention or treatment withdrawal.

Class (Stereo):
CHEMICAL (ABSOLUTE)



Obeticholic acid (also known as INT-747), is a potent, orally bioavailable farnesoid X receptor (FXR) agonist. The key role of the farnesoid X receptor (FXR) as a regulator of bile and cholesterol metabolism in the liver, with preclinical data from numerous studies providing strong rationale for the advancement of FXR agonists as hepatoprotective therapeutics in chronic liver disease. Obeticholic acid is marketed under the trade name Ocaliva. Ocaliva is specifically indicated for the treatment of primary biliary cholangitis (PBC) in combination with ursodeoxycholic acid (UDCA) in adults with an inadequate response to UDCA, or as monotherapy in adults unable to tolerate UDCA.

Class (Stereo):
CHEMICAL (ABSOLUTE)


Conditions:

Brivaracetam (UCB 34714, trade name Briviact), the 4-n-propyl analog of levetiracetam, is a racetam derivative with anticonvulsant properties. Briviact is indicated as adjunctive therapy in the treatment of partial-onset seizures in patients 16 years of age and older with epilepsy. Brivaracetam is believed to act by binding to the ubiquitous synaptic vesicle glycoprotein 2A (SV2A), like levetiracetam, but with 20-fold greater affinity. There is some evidence that racetams including levetiracetam and brivaracetam access the luminal side of recycling synaptic vesicles during vesicular endocytosis. They may reduce excitatory neurotransmitter release and enhance synaptic depression during trains of high-frequency activity, such as is believed to occur during epileptic activity.

Showing 11 - 20 of 13230 results