In 2025， FDA granted 44 novel drug approvals。This list summarizes the landmark novel therapeutic approvals, categorized by molecular modality and therapeutic area.
| Drug Name (Active Ingredient) | Modality | Company | Indication | Mechanism of Action (MoA) | Key Highlights |
| Journavx (Suzetrigine) | Small Molecule | Vertex | Acute Pain | NaV1.8 Inhibitor: Blocks sodium channels in peripheral nerves to stop pain signals before they reach the brain. | First new class of non-opioid pain medicine in >20 years. |
| Yeztugo (Lenacapavir) | Large (Biologic) | Gilead | HIV PrEP | Capsid Inhibitor: Disrupts the HIV-1 capsid at multiple stages of the viral lifecycle. | First twice-yearly subcutaneous injection for HIV prevention. |
| Myqorzo (Aficamten) | Small Molecule | Cytokinetics | Obstructive HCM | Cardiac Myosin Inhibitor: Reduces the number of active myosin-actin cross-bridges to decrease contractility. | Next-gen treatment for rare heart condition; superior symptom relief in Ph3. |
| Datroway (Datopotamab deruxtecan) | Large (ADC) | AstraZeneca / Daiichi | EGFR+ NSCLC | Trop-2 Directed ADC: Delivers a topoisomerase I inhibitor payload specifically to Trop-2 expressing tumor cells. | Significant expansion of the DXd ADC platform into lung cancer. |
| Enflonsia (Clesrovimab) | Large (mAb) | Merck | RSV Prevention | Passive Immunization: A long-acting monoclonal antibody that neutralizes the RSV fusion protein. | One-dose protection for infants throughout their first RSV season. |
| Gomekli (Mirdametinib) | Small Molecule | SpringWorks | NF1-PN | MEK1/2 Inhibitor: Blocks the MAPK/ERK pathway to reduce tumor volume in neurofibromatosis. | Approved for both adult and pediatric patients with plexiform neurofibromas. |
| Inluriyo (Imlunestrant) | Small Molecule | Eli Lilly | Breast Cancer | Oral SERD: A selective estrogen receptor degrader that induces ERα degradation. | Competitive oral alternative to injectable fulvestrant. |
| Voyxact (Sibeprenlimab) | Large (mAb) | Novartis | IgA Nephropathy | APRIL Antagonist: Binds and neutralizes the “A Proliferation-Inducing Ligand” to reduce IgA1 production. | Disease-modifying therapy for primary IgA nephropathy. |
| Redemplo (Plozasiran) | Large (siRNA) | Arrowhead / GSK | FCS | RNA Interference: Silences the APOC3 gene in the liver to lower triglyceride levels. | First RNAi therapy approved for Familial Chylomicronemia Syndrome. |
| Cardamyst (Etripamil) | Small Molecule | Milestone | PSVT | Calcium Channel Blocker: Rapidly slows conduction through the AV node via nasal administration. | First self-administered nasal spray to stop “racing heart” episodes. |
| Nuzolvence (Zoliflodacin) | Small Molecule | Entasis / Innoviva | Gonorrhea | Topoisomerase Inhibitor: Unique spiropyrimidinetrione class targeting bacterial type II topoisomerases. | First-in-class antibiotic developed for drug-resistant gonorrhea. |
| Waskyra (Etuvetidigene autotemcel) | Large (Cell/Gene) | Orchard Tx | WAS | Ex vivo Gene Therapy: Autologous HSCs transduced with a lentiviral vector to express WAS protein. | Curative-intent gene therapy for Wiskott-Aldrich Syndrome. |
| Kygevvi (Doxecitine/Doxribtimine) | Small Molecule | UCB | TK2d | Nucleoside Combination: Bypasses thymidine kinase 2 deficiency to restore mitochondrial DNA. | First treatment for this ultra-rare, fatal mitochondrial disease. |
| Exdensur (Depemokimab) | Large (mAb) | GSK | Severe Asthma | IL-5 Antagonist: High-affinity binding to IL-5 to prevent eosinophilic inflammation. | First 6-month biologic for asthma maintenance. |
1. Modality Distribution Analysis
The 2025 approval cycle shows a near-perfect equilibrium between traditional pharmacology and advanced biotechnology.
Small Molecules (66%): Continue to dominate primary care and areas requiring high tissue/brain penetration (Pain, Cardiovascular, Bacterial infection).
Large Molecules/Biologics (34%): Includes a diverse mix of Monoclonal Antibodies, ADCs, RNAi, and Cell/Gene Therapies. This indicates a shift where biologics are no longer “niche” but a standard pillar of the industry.
2. Therapeutic Concentration (Top Sectors)
Oncology & Rare Diseases: Account for most of 2025 approvals. This reflects the industry’s focus on high-unmet-need areas where pricing power is stronger and regulatory pathways (like Breakthrough Therapy designation) are more accessible.
Infectious Disease: A surprise resurgence in 2025 with major wins in RSV and drug-resistant Gonorrhea, signaling a potential shift back to antimicrobial R&D.
3. Strategic “Long-Acting” Trend
A defining commercial trend of 2025 is the Dosing Interval Revolution.
HIV (Yeztugo): Twice yearly (6 months).
Asthma (Exdensur): Twice yearly (6 months).
RSV (Enflonsia): Single seasonal dose. This trend suggests that “convenience” is becoming the primary battlefield for market share in chronic disease management.
- Other key facts
Non-Opioid Breakthroughs: The approval of Suzetrigine marks a historical shift in pain management strategy.
ADC Dominance: Antibody-Drug Conjugates continue to redefine oncology, specifically in hard-to-treat lung and breast cancers.
Gene Therapy Maturity: Several “firsts” in ultra-rare diseases (WAS, TK2d) reached the market this year.