As Christmas is approaching, 2024 marked a significant year for drug development, with the FDA approving numerous novel therapies across various therapeutic areas. Rare diseases experienced a transformative year in 2024, with drugs accounting for 23.8% of approvals, highlighting a strong commitment to addressing unmet medical needs. Oncology drugs also made significant breakthroughs, representing 19% of approvals and ranking second, while hematological diseases showed promising potential for further exploration.
Based on the FDA’s new drug approvals in 2024, the below table will introduce the 21 most impactful small-molecule drugs.
| # | Drug Name | Indication | Company |
| 1 | Rezdiffra (resmetirom) | Nonalcoholic steatohepatitis (NASH) | Madrigal |
| 2 | Tryvio (aprocitentan) | Hypertension | Idorsia |
| 3 | Duvyzat (givinostat) | Duchenne muscular dystrophy | Italfarmaco Spa |
| 4 | Zelsuvmi (berdazimer) | Molluscum contagiosum | Novan Inc. |
| 5 | Zevtera (ceftobiprole medocaril sodium) | Various bacterial infections | Roche |
| 6 | Ohtuvayre (ensifentrine) | Chronic obstructive pulmonary disease (COPD) | Verona Pharma |
| 7 | Voranigo (vorasidenib) | Certain types of brain tumors | Servier Pharmaceuticals |
| 8 | Leqselvi (deuruxolitinib) | Alopecia areata | Sun Pharmaceuticals |
| 9 | Flyrcado (flurpiridaz F 18) | Cardiac imaging | GE Healthcare |
| 10 | Ojemda (tovorafenib) | Pediatric low-grade glioma | Viracta Therapeutics Inc. |
| 11 | Iqirvo (elafibranor) | Primary biliary cholangitis | Genfit SA |
| 12 | Aqneursa (levacetylleucine) | Niemann-Pick disease type C | IntraBio |
| 13 | Cobenfy (xanomeline and trospium chloride) | Schizophrenia | BMS |
| 14 | Vafseo (vadadustat) | Anemia due to chronic kidney disease | P&G |
| 15 | Sofdra (sofpironium) | Primary axillary hyperhidrosis | Bodor Laboratories Inc. |
| 16 | Itovebi (inavolisib) | Breast cancer | Genentech |
| 17 | Miplyffa (arimoclomol) | Niemann-Pick disease type C | Zevra Therapeutics |
| 18 | Xolremdi (mavorixafor) | WHIM syndrome | Sanofi |
| 19 | Livdelzi (seladelpar) | Primary biliary cholangitis | Gilead Sciences |
| 20 | Voydeya (danicopan) | Paroxysmal nocturnal hemoglobinuria | Achillion Pharmaceuticals Inc. |
| 21 | Lazcluze (lazertinib) | Non-small cell lung cancer | Johnson & Johnson |
These 21 drugs represent a significant advancement in the field of drug development and offer hope for patients with a wide range of diseases. As the pharmaceutical industry continues to innovate, we can expect to see more groundbreaking drug approvals in the coming years.