Review of 2021 FDA-Approved Drugs for Genetic Conditions

Peptidic GLP-1R agonists have received significant media coverage over the past year for their astounding efficacy in several indications. GLP-1R agonists now appear to be efficacious in reducing heart disease risk and in treating certain chronic kidney diseases, expanding their likely future use. This review by Oliver Philps provides an overview of GLP-1 agonists and where they fit in the treatment of diabetes, how the peptide drugs evolved over time to today’s weight loss medications, and what to expect next...

Among the small molecule highlights in January’s news were a $100M+ Series A for a PARP1 + PI3Kα-focused company, clinical data with an SLC inhibitor for PKU, a 5-HT2C superagonist for seizures, and a NK1,3 dual antagonist for women’s health. A novel anti-obesity agent from phenotypic screening also made headlines, and the close of a major acquisition may bring relief to the industry. You can read about these notable scientific highlights and more below.

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.

KIN-3248 (Kinnate Biopharma) is a next-generation, irreversible, pan-fibroblast growth factor receptor inhibitor (FGFRi) in a first-in-human (FIH) dose-escalation study (NCT05242822) in adults with advanced tumors harboring FGFR2 and FGFR3 gene alterations. The program is an excellent case study for rational covalent drug design to address kinase mutations while maintaining sufficient PK properties for oral daily dosing in humans.

This October roundup features key clinical updates, including FDA approvals, significant trial results for major drug candidates, the initiation of new clinical trials, and updates on halted studies.