Pyridine is a six-membered heterocyclic compound containing one nitrogen heteroatom. Pyridine and piperidine are the most frequently occurring heterocyclic building blocks in drug molecules. According to incomplete statistics, there are currently more than 180 drugs containing pyridine or piperidine structure that have been marketed, nearly 1/5 of the drugs approved for marketing in recent years contain these two structures.
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Pyridine is a basic heterocyclic organic compound with the chemical formula C5H5N. It is structurally related to benzene, with one methine group (=CH−) replaced by a nitrogen atom. It is a highly flammable, weakly alkaline, water-miscible liquid with a distinctive, unpleasant fish-like smell.
Pyrimidine, also known as 1,3-diazobenzene, is a heterocyclic compound with the chemical formula C4H4N2. Pyrimidine is formed by substituting 2 nitrogen atoms for 2 carbons in the meta-position of benzene. It is a diazine and retains its aromaticity. Derivatives of pyrimidine widely exist in organic macromolecular nucleic acids, and many drugs also contain pyrimidine rings. In nucleic acids, three nucleobases are pyrimidine derivatives: cytosine, thymine and uracil. There are a variety of pyrimidine-containing drugs on the market, most of which are kinase inhibitors.
As the first approved small molecule splicing modifier drug, Risdiplam (Evrysdi) is granted approval by the European Commission to expand the EU marketing authorization. This extension now includes infants with a clinical diagnosis of SMA Type 1, Type 2 or Type 3 or with one to four SMN2 copies from birth to below two months. Type 1 spinal muscular atrophy (SMA) is a rare, progressive neuromuscular disease and a leading genetic factor of infant mortality, that is caused by low levels of functional survival of motor neuron (SMN) protein. Risdiplam modifies SMN2 pre-messenger RNA splicing and increases levels of functional SMN protein in both the central nervous system and peripheral organs.