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.

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.

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.

Lusutrombopag (trade name Mulpleta) is an orally bioavailable, small molecule thrombopoietin (TPO) receptor agonist being developed by Shionogi for chronic liver disease (CLD) patients with thrombocytopenia prior to elective invasive surgery. Lusutrombopag acts selectively on the human TPO receptor and activates signal transduction pathways that promote the proliferation and differentiation of bone marrow cells into megakaryocytes, thereby increasing platelet levels. In September 2015, Lusutrombopag received its first global approval in Japan for the improvement of CLD-associated thrombocytopenia in patients scheduled to undergo elective invasive procedures. Oral Lusutrombopag is rapidly absorbed, with a median time to maximum serum concentration (Tmax) of 3.8–4.0 h in healthy subjects administered single doses of oral Lusutrombopag 1, 2 or 4 mg, and 6 h in CLD patients with thrombocytopenia administered oral Lusutrombopag 3 mg once daily for 7 days. The major metabolic pathway for Lusutrombopag appears to be omega- and beta-oxidation. Lusutrombopag is a substrate of breast cancer resistance protein and P-glycoprotein, according to in vitro data.

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.

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.

Encorafenib, also known as BRAFTOVI or LGX818, is an orally available mutated BRaf V600E inhibitor with potential antineoplastic activity, which was developed by Novartis. LGX818 possesses selective anti-proliferative and apoptotic activity in cells expressing BRAFV600E. In the A375 (BRAFV600E) human melanoma cell line LGX818 suppresses phospho-ERK (EC50 = 3 nM) leading to potent inhibition of proliferation (EC50 = 4 nM). No significant activity was observed against a panel of 100 kinases (IC50 > 900 nM) and LGX818 did not inhibit proliferation of > 400 cell lines expressing wild-type BRAF. On June 27, 2018, the Food and Drug Administration approved encorafenib and Binimetinib (BRAFTOVI and MEKTOVI, Array BioPharma Inc.) in combination for patients with unresectable or metastatic melanoma with a BRAF V600E or V600K mutation, as detected by an FDA-approved test. Encorafenib and binimetinib target two different kinases in the RAS/RAF/MEK/ERK pathway. Compared with either drug alone, co-administration of encorafenib and binimetinib result in greater anti-proliferative activity in vitro in BRAF mutation-positive cell lines and greater anti-tumor activity with respect to tumor growth inhibition in BRAF V600E mutant human melanoma xenograft studies in mice. In addition to the above, the combination of encorafenib and binimetinib acted to delay the emergence of resistance in BRAF V600E mutant human melanoma xenografts in mice compared with the administration of either drug alone. Encorafenib is in phase III for Metastatic Colorectal Cancer and in phase II for Relapsed or Refractory Multiple Myeloma.

Duvelisib (IPI-145), is an orally available, small-molecule, selective dual inhibitor of phosphatidylinositol 3 kinase (PI3K) δ and γ isoforms originated by Intellikine (owned by Takeda) and developed by Infinity Pharmaceuticals. Orally administered duvelisib was rapidly absorbed, with a dose-proportional increase in exposure. The compound produced a half-life of approximately 7-12 hours, following 14 days of dosing. Duvelisib exerts profound effects on adaptive and innate immunity by inhibiting B and T cell proliferation, blocking neutrophil migration, and inhibiting basophil activation. Duvelisib blockade of PI3K-δ and PI3K-γ potentially lead to significant therapeutic effects in multiple inflammatory, autoimmune, and hematologic diseases. The molecule is in phase III development as a combination therapy for patients with haematological malignancies such as chronic lymphocytic leukemia and follicular lymphoma.

Baricitinib (trade name Olumiant) is an investigational drug for rheumatoid arthritis (RA), being developed by Incyte and Eli Lilly. Baricitinib is a selective JAK1 and JAK2 inhibitor with IC50 of 5.9 nM and 5.7 nM in cell-free assays. In February 2017 Baricitinib was approved for use in the European Union as a second-line therapy for moderate to severe active rheumatoid arthritis in adults, either alone or in combination with methotrexate. On 31 May 2018 FDA approved Barictinib for the treatment of adult patients with moderately to severely active rheumatoid arthritis who have had an inadequate response to one or more TNF antagonist therapies.

Stiripentol is an anticonvulsant drug used in the treatment of epilepsy. It recently proved to increase the GABAergic transmission in vitro in an experimental model of immature rat. Clinical studies were based on the fact that STP also acts as an inhibitor of CYP3A4, CYP1A2, and CYP2C19 in vivo in epileptic patients. Side effects are largely due to the increase in plasma concentrations of other anticonvulsants and can be reduced by lowering the dose of those drugs. Nausea and vomiting are particularly noted when used in combination with sodium valproate. It appears to increase the potency of phenobarbital, primidone, phenytoin, carbamazepine, clobazam and diazepam.