Diquafosol, a dinucleotide Up4U, is an agonist for purinergic P2Y2 receptor. Diquafosol stimulated water and mucin secretion by acting on P2Y2 receptors on the conjunctival epithelial and goblet cell membrane and elevating intracellular calcium ion concentrations. The Japanese Ministry of Health, Labour and Welfare granted approval for DIQUAS Ophthalmic Solution 3% (diquafosol tetrasodium) for the treatment of dry eye.

Cytarabine ocfosfate (commercial name: Starasid) is a prodrug having stearyl group attached to phosphoric acid at 5' position of arabinose moiety of cytosine arabinoside (Ara-C). This drug is given orally. The mode of action is in the inhibition of DNA synthesis after conversion to Ara-CTP as in Ara-C. The drug is metabolized in the liver, producing the intermediate metabolite, C-C3PCA which is converted to Ara-C gradually. This property results in the maintenance of relatively long time the blood Ara-C levels. This was proved to be active clinically against acute leukemia and MDS.

Laquinimod is a new orally available carboxamide derivative, which is currently developed for relapsing remitting (RR) and chronic progressive (CP) forms of multiple sclerosis (MS; RRMS or CPMS) as well as neurodegenerative diseases. The mechanism of action of laquinimod is not fully elucidated because the molecular target is not known. Treatment with laquinimod led to a significant and persistent increase in brain-derived neuroprotective factor (BDNF) serum levels compared to placebo treatment. In human studies, a decrease of pro-inflammatory and an increase of anti-inflammatory cytokines have been measured. After commercial launch the unexpected severe cardiac adverse events (AEs) such as serositis, pericarditis, and myocardial infarction were detected.

Erdosteine is an antioxidant compound developed by Edmond Pharma and approved in Europe for the treatment of chronic bronchitis and COPD. Erdosteine has two thiol groups and is believed to act as a free radicals scavenger (through the formation of the active metabolite I, N-thiodiglycolylhomocysteine). Also the drug effect may be due to the inhibition of the activity of elastase enzyme and its interaction with mucosa. The drug got Orphan Drug designation by FDA for the treatment of bronchiectasis.

Sitaxentan (TBC11251, trade name Thelin) is a potent and selective Endothelin A receptor antagonist. Sitaxentan was under development by Encysive Pharmaceuticals (now Pfizer) for use in the treatment of pulmonary hypertension, congestive heart failure and asthma. It was launched in the major markets of the European Union (EU) under name Thelin for the treatment of pulmonary arterial hypertension. In December 2010, Pfizer discontinued clinical trials of sitaxentan worldwide and initiated voluntary product withdrawal from markets where it is approved due to life-threatening idiosyncratic risk of liver injury.

Propamidine, an aromatic diamidine compound, is widely used as an antimicrobial agent. Propamidine isethionate, the salt of propamidine with isethionic acid, is used in the treatment of Acanthamoeba infection. Diseases caused by Acanthamoeba include keratitis and granulomatous amoebic encephalitis.

Nafamostat mesilate (NM), a synthetic serine protease inhibitor, has anticoagulant and anti-inflammatory properties. Nafamostat is approved and marketed in Japan. It relieves symptoms such as pain due to inflammation of the spleen. It improves visceral disorders and bleeding tendency caused by blood clotting tendency in the vessels. It prevents coagulation in the blood circuit during hemodialysis. It is usually used to improve acute symptoms of pancreatitis (acute pancreatitis, acute exacerbation phase of chronic pancreatitis, post-operative acute pancreatitis, acute pancreatitis after pancreatography, traumatic pancreatitis) and to prevent disseminated intravascular coagulation (DIC) and clotting of perfusing blood in extracorporeal blood circuit. Nafamostat mesilate significantly inhibits the release of platelet beta-thromboglobulin (beta TG) at 60 and 120 min. Nafamostat mesilate (NM) prevents any significant release of neutrophil elastase; at 120 min, plasma elastase-alpha 1-antitrypsin complex is 0.16 mg/mL in the NM group and 1.24 mg/mL in the control group. Nafamostat mesilate completely inhibits formation of complexes of C1 inhibitor with kallikrein and FXIIa.

Suramin is an antiprotozoal and anthelmintic compound. It is indicated for the treatment of African trypanosomiasis (African sleeping sickness; trypanosome fever) and Onchocerciasis (river blindness). Additionally, suramin exhibits antineoplastic action. It was discovered that suramin produced dramatic, but transient, improvement of core symptoms of autism spectrum disorder.

Beraprost is a stable, orally active prostacyclin analogue. Beraprost acts by binding to prostacyclin membrane receptors ultimately inhibiting the release of Ca2+ from intracellular storage sites. This reduction in the influx of Ca2+ has been postulated to cause relaxation of the smooth muscle cells and vasodilation. Beraprost is indicated for the treatment of pulmonary hypertension and improvement of ulcers, pain & feeling of coldness associated with chronic arterial occlusion. In addition beraprost displays thyroid hormone receptor antagonistic properties.

Tilorone (trade names Amixin, Lavomax and others) is the first recognized synthetic, small molecular weight compound that is an orally active interferon inducer. Tilorone induces the formation of interferons (alpha, beta, gamma) by intestinal epithelial cells, hepatocytes, T-lymphocytes, and granulocytes. After ingestion, the maximum production of interferon is determined in the sequence of the intestine - liver - blood after 4-24 hours. Activates the stem cells of the bone marrow, stimulates humoral immunity, increases the production of IgM, IgA, IgG, affects the antibody formation, reduces the degree of immunosuppression, restores the ratio of T-helperers / T-suppressors. The mechanism of antiviral action is associated with the inhibition of translation of virus-specific proteins in infected cells, thereby suppressing the replication of the virus. Effective against influenza viruses and viruses that cause ARVI, hepato- and herpesviruses, incl. CMV and others. The mechanism of antiviral action is associated with the inhibition of translation of virus-specific proteins in infected cells, thereby suppressing the replication of the virus.