A Bear Bile Acid Goes Bust: Taurursodiol + Phenylbutyrate

Recently, we summarized the novel small molecule drug approvals of 2023 (34/55). The remaining 21 molecular entities approved were large molecules like proteins and oligonucleotides, accounting for nearly 38% of the total approvals. Notably, seven of these large molecules have earned the prestigious Breakthrough Therapy designation, relative to the two small molecules that share this recognition. A summary of approvals for large molecule drugs, accompanied by links to FDA labels and additional information pertaining to the drug's discovery and originators, appears here.

Our latest roundup of small molecules in the news includes two potential upcoming approvals: rezafungin Rezafungin (CD-101) is a once-weekly IV antifungal agent and next-generation echinocandin (glucan synthase inhibitor) that demonstrated non-inferiority vs. standard of care caspofungin (IV QD) (NCT03667690). An FDA AdCom voted 14 to 1 in [...]

In April 2024, there were promising examples of drug repurposing, significant clinical and regulatory milestones for therapies aimed at rare and pediatric diseases, the initiation of a rolling NDA submission for suzetrigine, substantial billion-dollar acquisitions, and positive trial results were reported for both a D1/D5 receptor agonist and a PARP1 inhibitor. However, the industry also had setbacks as demonstrated by the failed clinical trials for an MNK inhibitor and an NMDA receptor modulator. In case you missed anything, here’s a recap of the most notable news highlights from April 2024!

Here’s a rundown of August’s key clinical developments, including FDA approvals, potential upcoming approvals, notable trial readouts, new NDA submissions, and pipeline realignments.

The FDA approved five novel small molecule drugs in neurology in 2021. Addressing a range of conditions from ADHD to relapsing MS, the 2021 neurology drug approvals are: atogepant (Qulipta): calcitonin gene-related peptide (CGRP) receptor antagonist ponsimod (Ponvory): selective sphingosine-1-phosphate (S1P) receptor 1 (S1PR1) modulator [...]