Pharmaceutical Substances - Version 4.16

The version 4.16 of Pharmaceutical Substances was launched in May 2024.

2023 was again a very productive year for the pharmaceutical industry with for example 55 new drug FDA approvals, the second highest number after 2018. Among them, we counted 38 new chemical entities, continuing the high level seen in the last 5 years. This is reflected in the new version 4.16 of Pharmaceutical Substances which contains 19 new APIs and 5 updates.

Several new kinase inhibitors for the treatment of different cancers are described. Capivasertib, a first in class pan-AKT inhibitor, is used for the treatment of hormone receptor-positive, HER2-negative locally advanced or metastatic breast cancer. The VEGFR inhibitor Fruquintinib is used to treat refractory, metastatic colorectal cancer. Additional drugs described are the JAK1/JAK2 inhibitor Momelotinib for the treatment of myelofibrosis, a type of bone marrow cancer that causes scar tissue buildup in the bone marrow, and Repotrectinib, an inhibitor of the proto-oncogene tyrosine-protein kinase ROS1, for the treatment of ROS1-positive non-small-cell lung cancer (NSCLC).

The FDA also approved the first gamma-secretase inhibitor Nirogacestat for the treatment of desmoid tumors, noncancerous growths in the connective tissue. The macrocyclic peptide Motixafortide acts as CXCR4 inhibitor and is used to treat multiple myeloma. In addition, the cyanine dye Indocyanine green is described which is not only applied as a cancer diagnostic but also as a photosensitizer to selectively kill cancer cells in photothermal and photodynamic therapy.

New treatment options for several rare diseases became available in the recent months, among them most notably the selective retinoic acid receptor gamma (RARγ) agonist Palovarotene for the reduction of the volume of new heterotopic ossification (bone formation outside the skeleton) for patients with fibrodysplasia ossificans progressiva, a disorder in which bones gradually form in muscle tissue and connective tissue. Novel peptide therapeutics made it to the market in this space with Vosoritide, a modified C-type natriuretic peptide (CNP) for the treatment of achondroplasia, and Zilucoplan, a macrocyclic peptide inhibitor of complement component 5 (C5 inhibitor) for the treatment of generalized myasthenia gravis, a long-term neuromuscular junction disease. Fosdenopterin was approved to reduce the risk of death due to a rare genetic disease known as molybdenum cofactor deficiency type A and the nucleoside Cedazuridine as an inhibitor of cytidine deaminase that is used as a pharmacoenhancer of decitabine to increase the oral bioavailability of this DNA methylase inhibitor treatment of myelodysplastic syndromes.

The ectoparasiticide Lotilaner is an another first-in-class medication used to treat Demodex blepharitis, a chronic eyelid inflammation caused by Demodex folliculorum, a microscopic mite. Zuranolone, a positive allosteric modulator of the GABAA receptor, is the first oral drug to treat postpartum depression, a complex mix of physical, emotional, and behavioral changes that can occur around childbirth. Furthermore, the 5-HT1A agonist Gepirone provides a novel option for the treatment of depression and the CGRP receptor antagonist Rimegepant for the acute treatment of migraine.

The sphingosine-1-phosphate receptor modulator Etrasimod was approved for the treatment of ulcerative colitis (Colitis ulcerosa). Moreover, the version 4.16 contains the P2X3-receptor antagonist Gefaxipant for the treatment of chronic cough and Vamorolone, a new synthetic corticosteroid for the treatment of Duchenne muscular dystrophy with an improved side-effect profile.

The updates most importantly cover two of the most talked about new drugs on the market, the selective GLP-1R agonist Semaglutide and the GLP-1R/GIPR dual agonist Tirzepatide, both used for the treatment of obesity and type 2 diabetes. Further updates are on two cancer drugs, the KRASG12C inhibitor Sotorasib and 5-Aminolevulinic acid, a substrate in the porphyrin synthesis pathway, which is used both in cancer diagnostics and treatment. The last update is on Pregabalin, an anticonvulsive for the treatment of neuropathic pain.


Prof. Axel Kleemann, Prof. Bernhard Kutscher, Dr. Dietmar Reichert, Dr. Martin Bossart

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