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.

Triclabendazole, (brand name Avomec, Egaten, etc) is a member of the benzimidazole family of anthelmintics used to treat liver flukes, specifically fascioliasis and paragonimiasis. Triclabendazole used routinely since 1983 in veterinary practice for the treatment of fascioliasis. It was not used in humans until the 1989 epidemic of fascioliasis near the Caspian Sea when Iranian authorities approved the use of the veterinary formulation to treat the infection. Fasciolicidal not only against the adult worms present in the biliary ducts, but also against the immature larval stages of Fasciola migrating through the hepatic parenchyma. Triclabendazole is shown to penetrate into liver flukes by transtegumentary absorption followed by inhibition of the parasite's motility, probably related to the destruction of the microtubular structure, resulting in the death of the parasite; the immobilizing effect is paralleled by changes in the parasite's resting tegumental membrane potential, strongly inhibiting the release of proteolytic enzymes, a process that appears critical to the survival of the parasite. Side effects are generally few, but can include abdominal pain and headaches. Biliary colic may occur due to dying worms. While no harms have been found with use during pregnancy, triclabendazole has not been well studied in this population. Triclabendazole is on the World Health Organization's List of Essential Medicines, the most effective and safe medicines needed in a health system. It is not commercially available in the United States.

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.

Lofexidine is newly FDA approved in the United States under the brand name LUCEMYRA for the treatment of opioid withdrawal symptoms in adults. Lofexidine acts as an agonist to α2 adrenergic receptors. These receptors inhibit adenylyl cyclase activity, leading to the inhibition of the second messenger, cyclic adenosine monophosphate (cAMP). The inhibition of cAMP leads to potassium efflux through calcium-activated channels, blocking calcium ions from entering the nerve terminal, resulting in suppression of neural firing, inhibition of norepinephrine release. Lofexidine replaces the opioid-driven inhibition of cAMP production and moderating the symptoms of opioid withdrawal.

Tafenoquine is anti-malaria drug originated in Walter reed army institute of research and developed by GSK and 60 Degrees Pharmaceuticals. In 2018 United States Food and Drug Administration (FDA) approved single dose tafenoquine for the radical cure (prevention of relapse) of Plasmodium vivax malaria. Tafenoquine, an 8-aminoquinoline antimalarial, is active against all the stages of Plasmodium species that include the hypnozoite (dormant stage) in the liver. Studies in vitro with the erythrocytic forms of Plasmodium falciparum suggest that tafenoquine may exert its effect by inhibiting hematin polymerization and inducing apoptotic like death of the parasite. In addition to its effect on the parasite, tafenoquine causes red blood cell shrinkage in vitro. Tafenoquine is active against pre-erythrocytic (liver) and erythrocytic (asexual) forms as well as gametocytes of Plasmodium species that include P. falciparum and P. vivax. The activity of tafenoquine against the pre-erythrocytic liver stages of the parasite, prevents the development of the erythrocytic forms of the parasite.

Cannabidiol is the major nonpsychoactive ingredient in cannabis. Cannabidiol demonstrates a range of effects that may be therapeutically useful, including anti-seizure, antioxidant, neuroprotective, anti-inflammatory, analgesic, anti-tumor, anti-psychotic, and anti-anxiety properties. Exact mechanism of action of cannabidiol is not known, but may include effects on the orphan G-protein-coupled receptor GPR55; the transient receptor potential of vanilloid type-1 channel; the 5-HT1a receptor; and the α3 glycine receptors. GW Pharmaceuticals successfully developed the world’s first prescription medicine derived from the cannabis plant, Sativex® (buccal spray containing delta-9-tetrahydrocannabinol and cannabidiol) now approved in over 29 countries outside of the United States for the treatment of spasticity due to Multiple Sclerosis. GW Pharmaceuticals is developing Epidiolex® (a liquid formulation of pure plant-derived cannabidiol) for certain rare and severe early-onset, drug-resistant epilepsy syndromes.

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.

Binimetinib (MEK162) is an oral small-molecule with potential antineoplastic activity. It is a selective mitogen-activated protein kinase 1 and 2 (MEK1/2) inhibitor, a key protein kinase in the RAS/RAF/MEK/ERK pathway, which regulates several key cellular activities including proliferation, differentiation, migration, survival and angiogenesis. Inappropriate activation of this pathway has been shown to occur in many cancers, in particular through mutations in BRAF, KRAS and NRAS. MEK162 at 6 mg/kg, BID combined with BEZ235 (dual PI3K/mTOR inhibitor) resulted in a significant reduction of tumor growth in immunodeficient mice injected with MCF7 cells. There are three ongoing Phase 3 trials with binimetinib in advanced cancer patients: NEMO (NRAS-mutant melanoma), COLUMBUS (encorafenib in combination with binimetinib in BRAF-mutant melanoma) and BEACON CRC (encorafenib, binimetinib and cetuximab in BRAF-mutant colorectal cancer).

Revefenacin (trade name Yupelri is a long-acting muscarinic antagonist developed by Mylan Ireland ltd for the treatment of chronic obstructive pulmonary disease (COPD). It has similar affinity to the subtypes of muscarinic receptors M1 to M5. In the airways, it exhibits pharmacological effects through inhibition of M3 receptor at the smooth muscle leading to bronchodilation. The competitive and reversible nature of antagonism was shown with human and animal origin receptors and isolated organ preparations. In preclinical in vitro as well as in vivo models, prevention of methacholine- and acetylcholine-induced bronchoconstrictive effects was dose-dependent and lasted longer than 24 hours.

Tezacaftor (VX-661) is an investigational compound developed by Vertex Pharmaceuticals to treat cystic fibrosis (CF). It is an oral corrector of the CF transmembrane regulator (CFTR) and is similar to lumacaftor, another N-aryl-1-(2,2-difluoro-1,3-benzodioxol-5-yl)cyclopropanecarboxamide derivative developed by Vertex. Cystic fibrosis is caused by defects in CFTR gene, which encodes an epithelial chloride channel. The most common mutant Δ508CFTR is a misfolded protein that does not reach the cell membrane. VX-661 corrects trafficking of Δ508CFTR and partially restores chloride channel activity. In vitro, a combination of VX-661 and ivacaftor, an FDA approved in 2012 CFTR potentiator which increases the time the CFTR channel is open, allowing chloride ions to flow through the CFTR proteins on the surface of epithelial cells, resulted in greater CFTR activity compared with VX-661 alone. In February 2012, a phase 2, double-blind, placebo-controlled study of VX-661 was initiated in CF patients who were homozygous or heterozygous for the F508del mutation. There is an ongoing Vertex Phase 3 development program of VX-661 in combination with ivacaftor which includes four studies on CF patients 1) with two copies of the F508del mutation, 2) one copy of the F508del mutation and a second mutation that results in residual CFTR function, 3) one copy of the F508del mutation and a second mutation that results in residual CFTR function gating defect in the CFTR protein and 4) one copy of the F508del mutation and a second mutation that results in minimal CFTR function.