Pharmaceutical Substances - Version 4.15

The version 4.15 of Pharmaceutical Substances was launched in September 2023.

The new release 4.15 covers 20 new APIs, amongst them several with novel mechanisms of action, and three updated drugs.

Approved as “first-of-its-kind" were Fezolinetant, an oral non-hormonal neurokinin 3 (NK3) receptor antagonist that reduces vasomotor symptoms during menopause, and Zavegepant, a calcitonin gene-related peptide (CGRP) receptor antagonist, as nasal spray against migraine.

Major, unmet medical needs are addressed by the sodium glucose cotransporter type 2 (SGLT2) inhibitors Sotagliflozin, for treatment of heart failure as well diabetes and Bexagliflozin as antidiabetic agent.

Key developments in oncology led to new kinase inhibitors such as Leniolisib, the first treatment for activated phosphoinositide 3-kinase delta syndrome and Quizartinib, the first small molecule FLT3 inhibitor for the treatment of acute myeloid leukemia (AML). The US FDA granted accelerated approval for Pirtobrutinib for relapsed or refractory mantel cell lymphoma (MCL) and Ritlecitinib as first treatment of severe adult alopecia areata. Elacestrant is an orally available selective estrogen receptor degrader (SERD) for the treatment of breast cancer.

Sparsentan, as the first single-molecule dual endothelin angiotensin receptor antagonist, received accelerated approval to reduce proteinuria.

Perfluorohexyloctane was approved by the FDA as an ophthalmic solution for the treatment of signs and symptoms dry eye disease (DED).

Daprodustat was approved as an oral treatment for anemia caused by chronic kidney disease in patients on dialysis. Other compounds in the new class of hypoxia-inducible factor prolyl hydrolase inhibitors are Roxadustat, Vadadustat, and Enarodustat.

Peptide drug Setmelanotide is the first drug approved for chronic weight management of patients with Bardet-Biedl Syndrome (BBS), a rare genetic disorder associated with early-onset childhood obesity. Trofenitide, another peptide drug, was approved for the treatment of Rett syndrome, a rare genetic neurodevelopmental disorder.

Rezafungin is an antifungal drug approved for injection for the treatment of candidemia and invasive candidiasis.

Omaveloxolone is an orally active, semi-synthetic triterpenoid drug that increases antioxidant activity for treatment of Friedreich`s ataxia.

Sodium phenylbutyrate was approved as a new therapy to treat certain patients living with urea cycle disorders (UCD).

This latest release also comprises updates for Celecoxib, Liraglutide, and Sitagliptin.

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