Elbasvir is an inhibitor of the Hepatitis C Virus (HCV) Non-Structural protein 5A (NS5A). Elbasvir was approved by the FDA in January 2016 for the treatment of hepatitis C. It was developed by Merck and completed Phase III trials, used in combination with the NS3/4A protease inhibitor grazoprevir under the trade name Zepatier. Zepatier is indicated for treatment of chronic HCV genotype 1 or 4 infection in adults.

Lifitegrast (under brand name Xiidra) was approved as an ophthalmic solution for the treatment of the signs and symptoms of dry eye disease. Lifitegrast binds to the integrin lymphocyte function-associated antigen-1 (LFA-1); a cell surface protein found on leukocytes and blocks the interaction of LFA-1 with its cognate ligand intercellular adhesion molecule-1 (ICAM-1). This LFA-1/ICAM-1 interaction is a key step in the inflammatory cascade that contributes to dry eye disease. Besides lifitegrast participates in phase II clinical trials for prevention of the signs and symptoms of allergic conjunctivitis.

Crisaborole is a topically administered, boron-containing, anti-inflammatory compound that inhibits the phosphodiesterase-4 (PDE4) activity and thereby suppresses the cytokine release of TNFalpha, IL-12, IL-23 and other cytokines. PDE4 is an an enzyme that converts the intracellular second messenger 3'5'-cyclic adenosine monophosphate (cAMP) into the active metabolite adenosine monophosphate (AMP). By inhibiting PDE4 and thus increasing levels of cAMP, crisaborole controls inflammation. The use of boron chemistry enabled synthesis of a low-molecular-weight compound (251 daltons), thereby facilitating effective penetration of crisaborole through human skin. Crisaborole is in clinical development for the topical treatment of psoriasis and being pursued for the topical treatment of atopic dermatitis. Preliminary studies in children and adults demonstrated favorable efficacy and safety profiles. Crisaborole may represent an anti-inflammatory option that safely minimizes the symptoms and severity of AD and that can be used for both acute and long-term management.

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.

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.

Brexpiprazole is a novel D2 dopamine and serotonin 1A partial agonist, called serotonin-dopamine activity modulator (SDAM), and a potent antagonist of serotonin 2A receptors, noradrenergic alpha 1B and 2C receptors. Brexpiprazole is approved for the treatment of schizophrenia, and as an adjunctive treatment for major depressive disorder (MDD). Although it failed Phase II clinical trials for ADHD, it has been designed to provide improved efficacy and tolerability (e.g., less akathisia, restlessness and/or insomnia) over established adjunctive treatments for major depressive disorder (MDD).Brexpiprazole is sold under the brand name Rexulti. Although the mechanism of action of brexpiprazole in the treatment of MDD and schizophrenia is unclear, the efficacy of brexpiprazole may be attributed to partial agonist activity at serotonin 1A and dopamine D2 receptors, and antagonist activity at serotonin 2A receptors.

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.

MOVANTIK (naloxegol) is a peripherally-acting mu-opioid receptor antagonist indicated for the treatment of opioid-induced constipation (OIC) in adult patients with chronic noncancer pain. It is being investigated for the treatment of constipation as a side effect of prescription opioid pain medicines.

Idelalisib is a first-in-class selective inhibitor of adenosine-5'-triphosphate (ATP) binding to PI3Kdelta kinase, resulting in inhibition of the P13K signalling pathway in malignant B cells. The compound is approved for the treatment of several types of blood cancer. Idelalisib is intended to be used in combination with rituximab as second or subsequent line therapy for the treatment of chronic lymphocytic leukaemia. The drug may cause fatal and/or severe diarrhea or colitis, hepatotoxicity, pneumonitis and intestinal perforation.