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

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Showing 51 - 60 of 1726 results


Class (Stereo):
CHEMICAL (ACHIRAL)


GBT440 (previously GTx011) is a potent and direct drug for sickle cell treatment. In sickle cell anemia, abnormal hemoglobin molecules are formed, which causes problems for the flow of blood and oxygen through the body. GBT440 can selectively bind to hemoglobin, thereby increasing its affinity for oxygen. By inhibiting hemoglobin polymerization, it also prevents deformation of the red blood cells. GBT440, renamed Voxelotor, is thought to help prevent sickle cells blocking blood vessels, and therefore reduces pain (sickle cell crisis) experienced by patients. GBT440 is well absorbed following intravenous and oral administration, and quickly partitions into the red blood cell with a small part re‐distributed into the plasma. GBT440 was well tolerated in a randomized, placebo‐controlled, double blind, parallel group phase I/II study in healthy volunteers and sickle cell disease patients. Headache is the most reported adverse event related to the use of this drug, and no serious adverse events are known. A phase 3 clinical trial examining the efficacy and safety of the drug (compared to placebo) is planned to be completed in 2019. Voxelotor was also studied as a potential therapy for treatment of low oxygen levels in the blood of idiopathic pulmonary fibrosis patients, but this program was discontinued because of a lack of clinical benefits.

Class (Stereo):
CHEMICAL (ACHIRAL)


LASMIDITAN is a serotonin (5-HT) receptor agonist without vasoconstrictor activity, which selectively binds to the 5-HT(1F) receptor subtype. It is under development for the treatment of migraine.

Class (Stereo):
CHEMICAL (ABSOLUTE)

LEFAMULIN is a pleuromutilin antibiotic under development for the treatment of community-acquired bacterial pneumonia, as well as acute bacterial skin and skin structure infections. It inhibits bacterial protein synthesis by binding to the peptidyl transferase center of the 50S ribosome, resulting in the cessation of bacterial growth.
Pexidartinib (PLX3397) is a small-molecule receptor tyrosine kinase (RTK) inhibitor with potential antineoplastic activity. Pexidartinib binds to and inhibits phosphorylation of stem cell factor receptor (KIT), colony-stimulating factor-1 receptor (CSF1R) and FMS-like tyrosine kinase 3 (FLT3), which may result in the inhibition of tumor cell proliferation and down-modulation of macrophages, osteoclasts and mast cells involved in the osteolytic metastatic disease. FDA has granted Breakthrough Therapy Designation to pexidartinib (PLX3397) for the treatment of tenosynovial giant cell tumor (TGCT) where surgical removal of the tumor would be associated with potentially worsening functional limitation or severe morbidity. In addition to Breakthrough Therapy Designation, pexidartinib (PLX3397) has been granted Orphan Drug Designation by FDA for the treatment of pigmented villonodular synovitis (PVNS) and giant cell tumor of the tendon sheath (GCT-TS). It also has received Orphan Designation from the European Commission for the treatment of TGCT.
Pitolisant (INN) or tiprolisant (USAN) is a histamine receptor inverse agonist/antagonist selective for the H3 subtype. It has stimulant and nootropic effects in animal studies and may have several medical applications, having been researched for the treatment of narcolepsy, for which it has been granted orphan drug status in the EU and US. It is currently in clinical trials for schizophrenia and Parkinson’s disease. Pitolisant hydrochloride was approved by European Medicine Agency (EMA) on Mar 31, 2016. It was developed and marketed as Wakix® by Bioprojet in EU. Wakix® is available as the tablet for oral use, containing 4.5 mg and 18 mg of Pitolisant hydrochloride. The initial dose of 9 mg (two 4.5 mg, tablets) per day, and it should be used at the lowest effective dose, depending on individual patient response and tolerance, according to an up-titration scheme, without exceeding the dose of 36 mg/day. Pitolisant was the first clinically used H3 receptor inverse agonist.

Class (Stereo):
CHEMICAL (ACHIRAL)


Conditions:

Prucalopride is a novel enterokinetic compound and is the first representative of the benzofuran class. Prucalopride is a potent, selective and specific serotonin 5-HT4 receptor (5-HT4-R) agonist. Prucalopride (Resolor®), a highly selective serotonin 5-HT4 receptor agonist, is indicated in the European Economic Area for the treatment of adults with chronic idiopathic constipation (CIC) in whom laxatives have failed to provide adequate relief.

Class (Stereo):
CHEMICAL (ABSOLUTE)


Conditions:

Migalastat (Galafold)-a small molecule drug developed by Amicus Therapeutics that restores the activity of specific mutant forms of α-galactosidase-has been approved for the treatment of Fabry disease in the EU in patients with amenable mutations. Migalastat attaches to certain unstable forms of alpha-galactosidase A, stabilising the enzyme. This allows the enzyme to be transported into areas of the cell where it can break down GL-3. Under the trade name Galafold (formerly known as Amigal), Migalastat is used to treat patients aged 16 years or over with Fabry disease. Because the number of patients with Fabry disease is low, the disease is considered ‘rare’, and the US Food and Drug Administration (FDA) assigned Galafold orphan drug status in 2004, and the European Committee for Medicinal Products for Human Use (CHMP) followed in 2006.