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Pralnacasan

Pralnacasan is an orally bioavailable pro-drug of a potent, non-peptide inhibitor of interleukin-1beta converting enzyme (ICE).

Inclisiran

[L28178] Inclisiran was later approved by the FDA on December 22, 2021, for the treatment of heterozygous familial hypercholesterolemia (HeFH) or clinical atherosclerotic cardiovascular disease in adults … [A225953,A225958] On December 11, 2020, the European Commission (EC) granted authorization for marketing inclisiran as the first and only approved siRNA for the treatment of adults with primary hypercholesterolemia

Bezisterim

AVX-001

AVX-001 is under investigation in clinical trial NCT05164393 (Phase I/iia Trial to Evaluate AVX001 Gel in Doses of 1% or 3% Compared With Vehicle Over Four Weeks of Field-directed Treatment Period in Adult

Flavin mononucleotide

[L54226] In addition to its systemic use as a means of riboflavin supplementation, a topical ophthalmic preparation of FMN was approved by the FDA in October 2025 for use as a photoenhancer in epithelium-on

Capivasertib

[A262021,A262026,A262031] On November 17th, 2023, capivasertib, under the brand name TRUQAP, was approved by the FDA for the treatment of adult patients HR-positive, HER2-negative locally advanced or

Dostarlimab

[L33340] This indication was granted full FDA approval on February 10, 2023.[L45161] Dostarlimab-gxly was granted second accelerated approval for the treatment of solid tumours in the same month. … In April 2021, dostarlimab was granted accelerated approval by the FDA - as GlaxoSmithKline's dostarlimab-gxly (Jemperli) - for the treatment of adult patients with recurrent or advanced mismatch repair

ISIS 325568

ISIS 325568 is a generation 2.2 antisense drug that targets the glucagon receptor (GCGR). It was under investigation in clinical trial NCT00519727 (Safety Study of ISIS 325568 in Healthy Volunteers) for lowering blood glucose and helping...