首创新药时代：2025年FDA新药全景透视

Core Insights - The FDA's CDER approved 44 new drugs by 2025, with over half being "first-in-class" drugs, indicating a shift from "incremental" to "disruptive" treatment paradigms in drug development [1][3][24] - The rise of small molecules continues, but their development is evolving from broad inhibition to precision modulation, enhancing efficacy while reducing side effects [6][7][9][10] First-in-Class Drugs - The approval of "first-in-class" drugs is driven by unmet clinical needs, particularly in rare and complex chronic diseases, where existing treatments are inadequate [3][4] - Brinsupri (brensocatib) is a notable example, targeting Dipeptidyl Peptidase 1 (DPP1) to address non-cystic fibrosis bronchiectasis, providing a new mechanism of action that significantly improves patient outcomes [3][20] - Eight drugs received breakthrough therapy designation, reflecting their innovative mechanisms and potential for substantial clinical improvement [5] Small Molecule Innovations - Small molecules accounted for 66% of new drug approvals, maintaining their central role in drug development while shifting towards precision approaches [6][10] - Aficamten (Myqorzo) exemplifies this trend as a selective allosteric modulator for obstructive hypertrophic cardiomyopathy, demonstrating improved safety and efficacy [7] - Covalent drugs, such as Zegfrovy (sunvozertinib), show promise in overcoming resistance issues by forming stable bonds with targets, enhancing therapeutic effectiveness [8] Peptide and Nucleic Acid Therapies - Peptide and nucleic acid therapies are emerging as significant players, with multiple innovative drugs approved in 2025, marking a shift towards RNA-targeted treatments [10][11] - Qfitlia (fitusiran) offers a revolutionary preventive solution for hemophilia by significantly reducing annual bleeding rates [10][12] - Dawnzera (donidalorsen) provides a long-acting preventive treatment for hereditary angioedema, demonstrating substantial reductions in attack frequency [11] Antibody-Drug Conjugates (ADCs) - ADCs are evolving beyond cancer treatment, with Datroway (datopotamab deruxtecan) showcasing innovative mechanisms and expanding indications [14][15] - Datroway's "bystander effect" allows it to target heterogeneous tumor populations effectively, improving treatment outcomes in breast cancer and non-small cell lung cancer [15][16] Future Directions - The landscape of drug development is shifting towards a broader understanding of drug value, emphasizing patient outcomes and treatment paradigms over traditional sales metrics [19][23] - The integration of advanced technologies like AI and gene editing into drug development processes is expected to yield therapies that fundamentally alter disease progression [24]