Miltefosine is an anti-leishmanial agent. It is an alkyl phospholipids compound, was originally intended for breast cancer and other solid tumors. However, it could not be developed as an oral agent because of dose-limiting gastro-intestinal toxicity, and only a topical formulation is approved for skin metastasis. But Miltefosine showed excellent antileishmanial activity both in vitro and in experimental models. Miltefosine is effective in vitro against both promastigotes and amastigotes of various species of Leishmania and also other kinetoplastidae (Trypanosoma cruzi,T. brucei) and other protozoan parasites (Entamoeba histolytica, Acanthamoeba). Mechanism of action is unknown. It is likely to involve interaction with lipids (phospholipids and sterols), including membrane lipids, inhibition of cytochrome c oxidase (mitochondrial function), and apoptosis-like cell death. Miltefosine is approved for the treatment of Visceral leishmaniasis (due to Leishmania donovani), Cutaneous leishmaniasis (due to Leishmania braziliensis, Leishmania guyanensis, and Leishmania panamensis) and Mucosal leishmaniasis (due to Leishmania braziliensis).

Efinaconazole is triazole used as a 10% topical solution for the treatment of onychomycosis, a fungal infection of the nails. It was approved for use in Canada and the USA in 2014 and is marketed by Valeant Pharmaceuticals North America LLC under the name Jublia. Like other antifungal triazoles, efinaconazole inhibits the fungal cytochrome P450 enzyme lanosterol 14α demethylase (CYP51), thereby disrupting ergosterol synthesis and, consequently, membrane integrity and growth in fungi. CYP51 is evolutionarily conserved and, in mammals, mediates conversion of lanosterol to meiosis-activating sterols (MAS); MAS are intermediates in the biosynthesis of cholesterol and may have a signaling role in initiating meiosis and oocyte maturation. Azoles have higher affinity for fungal CYP51 compared to the mammalian enzyme and such selectivity contributes to the safety of this therapeutic class. Azoles have been reported to produce reproductive and developmental toxicity in both humans and laboratory animals. The mechanism is unknown but inhibition of mammalian CYP51 as well as other CYPs, e.g. CYP17, CYP19 and CYP26, have been postulated to play a role.

Dehydroepiandrosterone (INTRAROSA™, prasterone) is a major C19 steroid produced from cholesterol by the adrenal cortex. It is also produced in small quantities in the testis and the ovary. Dehydroepiandrosterone (INTRAROSA, prasterone) is structurally similar to, and is a precursor of, androstenedione, testosterone, estradiol, estrone and estrogen. It indicated for the treatment of moderate to severe dyspareunia, a symptom of vulvar and vaginal atrophy, due to menopause. The mechanism of action of dehydroepiandrosterone (INTRAROSA, prasterone) in postmenopausal women with vulvar and vaginal atrophy is not fully established.

Olodaterol is a beta2-adrenoceptor agonist discovered by Boehringer Ingelheim and approved for the treatment of Chronic Obstructive Pulmonary Disease. The compound exerts its pharmacological effects by binding and activation of beta2-adrenoceptors after inhalation. Activation of these receptors in the airways results in a stimulation of intracellular adenyl cyclase, an enzyme that mediates the synthesis of cyclic-3’, 5’ adenosine monophosphate (cAMP). Elevated levels of cAMP induce bronchodilation by relaxation of airway smooth muscle cells. Olodaterol effect lasts for 24 hours.

Olaparib is an oral inhibitor of poly (ADP-ribose) polymerase enzymes, including PARP1, PARP2, and PARP3 which are involved in normal cellular homeostasis, such as DNA transcription, cell cycle regulation, and DNA repair. Olaparib has shown activity in ovarian and breast tumors with known BRCA mutations and was the first FDA approved drug in this class. Lynparza (olaparib) is indicated for treatment of gBRCA-mutated advanced ovarian cancer. Its use together with other chemotherapy medicines can lead to increased effects on the blood resulting in reduction in the numbers of white blood cells and platelets, and anaemia.

Avibactam (formerly NXL104, AVE1330A) is a synthetic non-β-lactam, covalent, slowly reversible β-lactamase inhibitor that inhibits the activities of Ambler class A and C β-lactamases and some Ambler class D enzymes. The combination of ceftazidime with avibactam exhibited broad-spectrum activity against Ambler class A- and class C-producing Enterobacteriaceae. AVYCAZ is a combination of ceftazidime, a cephalosporin, and avibactam indicated for the treatment of patients with the following infections caused by designated susceptible microorganisms: Complicated Intra-abdominal Infections, used in combination with metronidazole and Complicated Urinary Tract Infections, including Pyelonephritis.

Eliglustat, marketed by Genzyme as CERDELGA, is a glucosylceramide synthase inhibitor indicated for the long-term treatment of type 1 Gaucher disease who are CYP2D6 extensive metabolizers, intermediate metabolizers, or poor metabolizers (PMs) as detected by an FDA-cleared test.

Pirfenidone is a synthetic antifibrotic agent indicated for the treatment of idiopathic pulmonary fibrosis as Esbriet. Pirfenidone inhibits fibroblast, epidermal, platelet-derived, and transforming beta-1 growth factors. It also inhibits DNA synthesis and the production of mRNA for collagen types I and III, resulting in a reduction in radiation-induced fibrosis. Pirfenidone has demonstrated activity in multiple fibrotic conditions however the exact mechanism of action of pirfenidone in the treatment of IPF has not been established.

Nintedanib is a receptor tyrosine kinase inhibitor with potential antiangiogenic and antineoplastic activities. It is the only kinase inhibitor drug approved to treat idiopathic pulmonary fibrosis. that targets multiple receptor tyrosine kinases (RTKs) and non-receptor tyrosine kinases (nRTKs). Nintedanib inhibits the following RTKs: platelet-derived growth factor receptor (PDGFR) α and β, fibroblast growth factor receptor (FGFR) 1-3, vascular endothelial growth factor receptor (VEGFR) 1-3, and Fms-like tyrosine kinase-3 (FLT3). Among them, FGFR, PDGFR, and VEGFR have been implicated in IPF pathogenesis. Nintedanib binds competitively to the adenosine triphosphate (ATP) binding pocket of these receptors and blocks the intracellular signaling which is crucial for the proliferation, migration, and transformation of fibroblasts representing essential mechanisms of the IPF pathology.

Tavaborole is a boron-based pharmaceutical agent indicated for the topical treatment of toenail onychomycosis, a fungal infection of the nail and nail bed due to Trichophyton rubrum or Trichophyton mentagrophytes infection. Tavaborole acts by inhibiting an aminoacyl-transfer ribonucleic acid (tRNA) synthetase (AARS) - Leucyl-tRNA synthetase. Leucyl-tRNA synthetase is an essential fungal enzyme required for protein synthesis and for the catalysis of ATP-dependent ligation of L-leucine to tRNA(Leu). Tavaborole’s low molecular weight (approximately half of most antifungals, such as terbinafine and efinaconazole) permits optimal nail plate penetration, superior to that of existing topical antifungal medications.