FDA approvals

FDA Novel Drug Approvals for 2025: Precision Therapies, Rare Diseases, and China’s Growing Footprint

As of August 12, 2025, the FDA has approved 25 novel drugs, emphasizing precision medicine, rare disease advances, and a notable increase in China-origin therapies gaining U.S. approval. This analysis covers key drugs, modalities, and the evolving China biotech footprint.

SDW Editor

— 11 min read

Updated August 12, 2025

The U.S. FDA has approved 25 novel drugs in 2025 to date, spanning oncology, infectious diseases, neurology, hematology, nephrology, ophthalmology, and pain management. This year’s approvals underscore three key themes:

  • Expanding precision medicine in oncology
  • First-in-class mechanisms for non-opioid pain control
  • Breakthroughs in rare disease therapeutics

The approvals, detailed on the FDA’s Novel Drug Approvals 2025 page, also highlight a growing presence of China-origin assets securing global positions — most notably Dizal Pharmaceutical’s sunvozertinib for EGFR exon20 insertion NSCLC.

Modality Distribution

FDA Novel Drug Approvals, Jan 1 – Aug 12, 2025

  • Small Molecules – 16
  • Monoclonal Antibodies – 4
  • Antibody-Drug Conjugates (ADCs) – 2
  • siRNA – 1
  • Bispecific Antibody – 1
  • Combination Therapy – 1

Small molecules dominating, alongside a steady rise in biologics and novel modalities.

Key Achievements in 2025

  • Precision Oncology — Six approvals target defined genetic subtypes:
    • ROS1+ NSCLC (taletrectinib)
    • KRAS-mutant ovarian cancer (avutometinib/defactinib)
    • c-Met overexpressing NSCLC (telisotuzumab vedotin)
    • EGFR exon20ins NSCLC (sunvozertinib, China-origin)
    • HER2-negative breast cancer (datopotamab deruxtecan)
    • HER2-mutant NSCLC (zongertinib)
  • Non-Opioid Analgesic Breakthrough — Vertex’s suzetrigine (Journavx), the first NaV1.8 inhibitor approved for acute pain, marks a novel approach in pain management; however, label expansion to chronic pain faces FDA scrutiny.
  • Rare Disease Advancements —
    • Mirdametinib for adult neurofibromatosis type 1 plexiform neurofibromas
    • Fitusiran (siRNA) for hemophilia A/B
    • Garadacimab for hereditary angioedema prevention
    • Modeyso (dordaviprone) for H3 K27M–mutant diffuse midline glioma
    • BRINSUPRI (brensocatib) for non-cystic fibrosis bronchiectasis, the first approved therapy for this chronic lung disease
  • Combatting Antimicrobial Resistance — Gepotidacin introduces a first-in-class oral antibiotic targeting resistant uropathogens.
  • Pediatric and Preventive Focus — RSV prevention (clesrovimab) and siRNA-based prophylaxis (fitusiran) expand options for infants and children.

Notable 2025 Approvals

  • Journavx (suzetrigine) — First-in-class NaV1.8 blocker, redefining acute pain management.
  • Gomekli (mirdametinib) — First NF1-PN therapy with strong overall response rates in adults and pediatrics.
  • Blujepa (gepotidacin) — Novel antibiotic active against resistant urinary tract infections.
  • Qfitlia (fitusiran) — First siRNA therapy for hemophilia, enabling simplified prophylaxis.
  • Imaavy (nipocalimab) — Anti-FcRn monoclonal antibody improving myasthenia gravis symptoms.
  • Ibtrozi (taletrectinib) — CNS-penetrant ROS1/TRK inhibitor for NSCLC.
  • Enflonsia (clesrovimab) — Single-dose RSV prophylaxis reducing infant hospitalizations.
  • Zegfrovy (sunvozertinib) — China’s Dizal Pharmaceutical wins first U.S. approval for an EGFR exon20 insertion oral targeted therapy.
  • Modeyso (Dordaviprone) - First FDA approval of a systemic therapy for H3 K27M-mutant diffuse midline glioma (DMG, formerly DIPG), offering new hope in this devastating pediatric brain tumor.
  • Hernexeos (zongertinib) — First oral targeted therapy for HER2-mutant NSCLC, showing 75% ORR in phase 1 with durable responses and improved tolerability over EGFR-targeting TKIs.
  • BRINSUPRI (brensocatib) — First and only DPP1 inhibitor approved for non-cystic fibrosis bronchiectasis, reducing exacerbations by 21% (10 mg) and 20% (25 mg) in the phase 3 ASPEN trial, addressing neutrophilic inflammation.

China-Linked Highlights

  • Zegfrovy (sunvozertinib) — First U.S. approval for Dizal Pharmaceutical; marks a milestone for Chinese biotech innovation in lung cancer precision therapy.
  • Penpulimab — Akeso’s PD-1 antibody gained U.S. approval in nasopharyngeal carcinoma, joining about 10 PD-1/PD-L1 inhibitors already on the market. The FDA nod positions Penpulimab in direct competition with Coherus BioSciences and Junshi Biosciences’ China-origin PD-1 Loqtorzi, the first FDA-approved PD-1 for nasopharyngeal carcinoma in 2023, highlighting China’s growing footprint in immuno-oncology.
  • Taletrectinib — Developed by China’s AnHeart Therapeutics in collaboration with Innovent Biologics, originally discovered by Daiichi Sankyo, and now with Novation holding global commercial rights, this CNS-penetrant ROS1/TRK inhibitor secured FDA approval for ROS1+ NSCLC. It illustrates the increasingly complex and global nature of drug development involving Chinese biotech players.

Full 2025 Approval List (Jan 1 – Aug 12)

A comprehensive table including drug name, active ingredient, approval date, indication, modality, mechanism of action, IUPAC name, SMILES, sponsor, and clinical significance (where applicable) is available here (24 entries). The list will be updated with new approvals before year-end.

  1. Drug Name (Brand Name): Datroway
    Generic Name: datopotamab deruxtecan-dlnk
    Structure:
    Approval Date: 1/17/2025
    Indication: HER2-negative, HR-positive metastatic breast cancer
    Modality: ADC (antibody-drug conjugate)
    MOA/Target: TROP2-directed ADC
    IUPAC Name: N/A (biologic)
    SMILES: N/A (biologic)
    Sponsor: Daiichi Sankyo/AstraZeneca
  2. Drug Name (Brand Name): Grafapex
    Generic Name: treosulfan
    Structure:


Approval Date: 1/21/2025
Indication: Stem cell transplant preparative regimen
Modality: Small molecule
MOA/Target: Alkylating agent
IUPAC Name: [(2S,3S)-2,3-dihydroxy-4-methylsulfonyloxybutyl] methanesulfonate
SMILES: COCS(=O)(=O)OCC(O)C(O)C(O)COS(=O)(=O)OC
Sponsor: Medac

  1. Drug Name (Brand Name): Journavx
    Generic Name: suzetrigine
    Structure:


Approval Date: 1/30/2025
Indication: Moderate-severe acute pain
Modality: Small molecule
MOA/Target: Voltage-gated sodium channel blocker
IUPAC Name: 4-[[(2R,3S,4S,5R)-3-(3,4-difluoro-2-methoxyphenyl)-4,5-dimethyl-5-(trifluoromethyl)oxolane-2-carbonyl]amino]pyridine-2-carboxamide
SMILES:C[C@H]1[C@H]([C@@H](O[C@@]1(C)C(F)(F)F)C(=O)NC2=CC(=NC=C2)C(=O)N)C3=C(C(=C(C=C3)F)F)OC
Sponsor: Vertex

Significance: First-in-class non-opioid analgesic for moderate-to-severe acute pain, addressing the opioid crisis via NaV1.8 inhibition. Efforts to expand its label to chronic pain face FDA hurdles, and a follow-on candidate, VX-993, was discontinued after failing a phase 2 acute pain trial.

  1. Drug Name (Brand Name): Gomekli
    Generic Name: mirdametinib
    Structure:


Approval Date: 2/11/2025
Indication: NF1 with plexiform neurofibromas
Modality: Small molecule
MOA/Target: MEK1/2 inhibitor
IUPAC Name: N-[(2R)-2,3-dihydroxypropoxy]-3,4-difluoro-2-(2-fluoro-4-iodoanilino)benzamide
SMILES: C1=CC(=C(C=C1I)F)NC2=C(C=CC(=C2F)F)C(=O)NOC[C@@H](CO)O
Sponsor: SpringWorks

Significance: First approved therapy for adult NF1-PN, with 41% ORR in adults and 52% in pediatrics (ReNeu trial), addressing a rare condition with significant morbidity.

  1. Drug Name (Brand Name): Romvimza
    Generic Name: vimseltinib
    Structure:


Approval Date: 2/14/2025
Indication: Symptomatic tenosynovial giant cell tumor
Modality: Small molecule
MOA/Target: CSF1R inhibitor
IUPAC Name: 3-methyl-5-[6-methyl-5-[2-(1-methylpyrazol-4-yl)pyridin-4-yl]oxypyridin-2-yl]-2-(propan-2-ylamino)pyrimidin-4-one
SMILES: CC1=C(C=CC(=N1)C2=CN=C(N(C2=O)C)NC(C)C)OC3=CC(=NC=C3)C4=CN(N=C4)C
Sponsor: Deciphera

  1. Drug Name (Brand Name): Blujepa
    Generic Name: gepotidacin
    Structure:


Approval Date: 3/25/2025
Indication: Uncomplicated UTI
Modality: Small molecule
MOA/Target: Bacterial topoisomerase II inhibitor
IUPAC Name: (2R)-2-({4-[(3,4-Dihydro-2H-pyrano[2,3-c]pyridin-6-ylmethyl)amino]-1-piperidinyl}methyl)-1,2-dihydro-3H,8H-2a,5,8a-triazaacenaphthylene-3,8-dione
SMILES: C1CC2=CC(=NC=C2OC1)CNC3CCN(CC3)C[C@@H]4CN5C(=O)C=CC6=C5N4C(=O)C=N6
Sponsor: GSK

Significance: First-in-class antibiotic, effective against resistant uropathogens (EAGLE-2/3 trials), addressing antimicrobial resistance.

  1. Drug Name (Brand Name): Qfitlia
    Generic Name: fitusiran
    Structure:
    Approval Date: 3/28/2025
    Indication: Hemophilia A or B
    Modality: siRNA
    MOA/Target: siRNA targeting antithrombin
    IUPAC Name: N/A (oligonucleotide)
    SMILES: N/A (oligonucleotide)
    Sponsor: Sanofi

Significance: First siRNA for hemophilia, reducing annualized bleeding rate by 71-73% in phase 3 trials, simplifying prophylaxis with bimonthly dosing.

  1. Drug Name (Brand Name): Vanrafia
    Generic Name: atrasentan
    Structure:


Approval Date: 4/2/2025
Indication: IgA nephropathy
Modality: Small molecule
MOA/Target: Endothelin A receptor antagonist
IUPAC Name: (2R,3R,4S)-4-(1,3-benzodioxol-5-yl)-1-[2-(dibutylamino)-2-oxoethyl]-2-(4-methoxyphenyl)pyrrolidine-3-carboxylic acid
SMILES:CCCCN(CCCC)C(=O)CN1C[C@@H]([C@H]([C@@H]1C2=CC=C(C=C2)OC)C(=O)O)C3=CC4=C(C=C3)OCO4
Sponsor: Chinook/Roche

  1. Drug Name (Brand Name): penpulimab-kcqx
    Generic Name: penpulimab-kcqx
    Structure:
    Approval Date: 4/23/2025
    Indication: Nasopharyngeal carcinoma
    Modality: Monoclonal antibody
    MOA/Target: Anti-PD-1 antibody
    IUPAC Name: N/A (biologic)
    SMILES: N/A (biologic)
    Sponsor: Akeso
  2. Drug Name (Brand Name): Imaavy
    Generic Name: nipocalimab-aahu
    Structure:
    Approval Date: 4/29/2025
    Indication: Generalized myasthenia gravis
    Modality: Monoclonal antibody
    MOA/Target: Anti-FcRn antibody
    IUPAC Name: N/A (biologic)
    SMILES: N/A (biologic)
    Sponsor: J&J

Significance: Improves MG-ADL score by 4.70 points (VIVACITY-MG3 trial), enhancing muscle function and quality of life for myasthenia gravis patients.

  1. Drug Name (Brand Name): Avmapki Fakzynja Co-Pack
    Generic Name: avutometinib / defactinib
    Structure:


Approval Date: 5/8/2025
Indication: KRAS-mutant ovarian cancer
Modality: Combination (small molecules)
MOA/Target: MEK inhibitor + FAK inhibitor
IUPAC Name:

    • Avutometinib: N-(1-methyl-5-(2-(5-(trifluoromethyl)-1H-imidazol-2-yl)pyridin-4-yloxy)pyridin-2-yl)-2-(3-(trifluoromethyl)phenyl)acetamide
    • Defactinib: N-methyl-4-({4-[({3-[methyl(methylsulfonyl)amino]pyridin-2-yl}methyl)amino]-5-(trifluoromethyl)pyrimidin-2-yl}amino)benzamide

             SMILES:

    • Avutometinib: Cc1c(c(c(c(n1)NC(=O)Cc2cc(cc(c2)C(F)(F)F)C(F)(F)F)Oc3ccnc(c3)c4[nH]c(c(n4)C(F)(F)F)C)C
    • Defactinib: CNC(=O)c1ccc(cc1)Nc2ncc(c(n2)NCc3c(c(c(cn3)N(C)S(=O)(=O)C)C(F)(F)F)C

              Sponsor: Verastem

  1. Drug Name (Brand Name): Emrelis
    Generic Name: telisotuzumab vedotin-tllv
    Structure:
    Approval Date: 5/14/2025
    Indication: NSCLC with c-Met overexpression
    Modality: ADC (antibody-drug conjugate)
    MOA/Target: c-Met-directed ADC
    IUPAC Name: N/A (ADC/biologic)
    SMILES: N/A (ADC/biologic)
    Sponsor: AbbVie/Seagen
  2. Drug Name (Brand Name): Tryptyr
    Generic Name: acoltremon
    Structure:


Approval Date: 5/28/2025
Indication: Dry eye disease
Modality: Small molecule
MOA/Target: Neurotrophic factor receptor modulator
IUPAC Name: (1R,2S,5R)-N-(4-methoxyphenyl)-5-methyl-2-propan-2-ylcyclohexane-1-carboxamide
SMILES: C[C@@H]1CC[C@H]([C@@H](C1)C(=O)NC2=CC=C(C=C2)OC)C(C)C 
Sponsor: Novaliq

  1. Drug Name (Brand Name): Enflonsia
    Generic Name: clesrovimab-cfor
    Structure:
    Approval Date: 6/9/2025
    Indication: RSV prevention in infants
    Modality: Monoclonal antibody
    MOA/Target: Anti-RSV antibody
    IUPAC Name: N/A (biologic)
    SMILES: N/A (biologic)
    Sponsor: Janssen

Significance: Reduces RSV-associated hospitalizations by 84.3% (CLEVER trial), offering single-dose prophylaxis for infants

  1. Drug Name (Brand Name): Ibtrozi
    Generic Name: taletrectinib
    Structure:


Approval Date: 6/11/2025
Indication: ROS1+ NSCLC
Modality: Small molecule
MOA/Target: ROS1 inhibitor
IUPAC Name: N-(5-((4-(1H-indol-3-yl)pyrimidin-2-yl)amino)-2-fluorophenyl)-4-(1-methyl-1H-pyrazol-4-yl)thiazol-2-amine
SMILES: Cn1cc(c(n1)C)Sc2ncc(s2)Nc3cc(c(c(c3)F)Nc4nccc(n4)c5c[nH]c6c5cccc6)
Sponsor: AnHeart

Significance: Next-generation ROS1 inhibitor with high CNS penetration, achieving 71% ORR and 14.1-month DOR (TRUST-I/II trials) for NSCLC.

  1. Drug Name (Brand Name): Andembry
    Generic Name: garadacimab-gxii
    Structure:
    Approval Date: 6/16/2025
    Indication: Prevent hereditary angioedema attacks
    Modality: Monoclonal antibody
    MOA/Target: Anti-FXII monoclonal antibody
    IUPAC Name: N/A (biologic)
    SMILES: N/A (biologic)
    Sponsor: CSL Behring
  2. Drug Name (Brand Name): Lynozyfic
    Generic Name: linvoseltamab-gcpt
    Structure:
    Approval Date: 7/2/2025
    Indication: Multiple myeloma (after ≥4 prior therapies)
    Modality: Bispecific antibody
    MOA/Target: CD38-targeted bispecific antibody
    IUPAC Name: N/A (biologic)
    SMILES: N/A (biologic)
    Sponsor: Regeneron
  3. Drug Name (Brand Name): Zegfrovy
    Generic Name: sunvozertinib
    Structure:


Approval Date: 7/2/2025
Indication: NSCLC, EGFR exon 20 mutation
Modality: Small molecule
MOA/Target: EGFR exon 20 inhibitor
IUPAC Name: N-[5-[[4-[5-chloro-4-fluoro-2-(2-hydroxypropan-2-yl)anilino]pyrimidin-2-yl]amino]-2-[(3R)-3-(dimethylamino)pyrrolidin-1-yl]-4-methoxyphenyl]prop-2-enamide
SMILES: CC(C)(C1=CC(=C(C=C1NC2=NC(=NC=C2)NC3=C(C=C(C(=C3)NC(=O)C=C)N4CCC@HN(C)C)OC)Cl)F)O
Sponsor: Dizal Pharmaceutical

Significance: First and only FDA-approved oral targeted therapy for EGFR exon20ins NSCLC, developed by China’s Dizal Pharmaceutical.

  1. Drug Name (Brand Name): Ekterly
    Generic Name: sebetralstat
    Structure:


Approval Date: 7/3/2025
Indication: Acute hereditary angioedema attacks
Modality: Small molecule
MOA/Target: Plasma kallikrein inhibitor
IUPAC Name: N-[(3-fluoro-4-methoxypyridin-2-yl)methyl]-3-(methoxymethyl)-1-[[4-[(2-oxopyridin-1-yl)methyl]phenyl]methyl]pyrazole-4-carboxamide
SMILES: COCC1=NN(C=C1C(=O)NCC2=NC=CC(=C2F)OC)CC3=CC=C(C=C3)CN4C=CC=CC4=O
Sponsor: KalVista

  1. Drug Name (Brand Name): Anzupgo
    Generic Name: delgocitinib
    Structure:


Approval Date: 7/23/2025
Indication: Chronic hand eczema
Modality: Small molecule
MOA/Target: Pan-JAK inhibitor
IUPAC Name: 3-[(3S,4R)-3-methyl-7-(7H-pyrrolo[2,3-d]pyrimidin-4-yl)-1,7-diazaspiro[3.4]octan-1-yl]-3-oxopropanenitrile 
SMILES: C[C@H]1CN([C@]12CCN(C2)C3=NC=NC4=C3C=CN4)C(=O)CC#N 
Sponsor: Japan Tobacco

  1. Drug Name (Brand Name): Sephience
    Generic Name: sepiapterin
    Structure:


Approval Date: 7/28/2025
Indication: Hyperphenylalaninemia in PKU
Modality: Small molecule
MOA/Target: Tetrahydrobiopterin precursor
IUPAC Name: 2-amino-6-[(2S)-2-hydroxypropanoyl]-7,8-dihydro-3H-pteridin-4-one
SMILES: C[C@@H](C(=O)C1=NC2=C(NC1)N=C(NC2=O)N)O
Sponsor: PTC Therapeutics

  1. Drug Name (Brand Name): Vizz
    Generic Name: aceclidine
    Structure:


Approval Date: 7/31/2025
Indication: Presbyopia
Modality: Small molecule
MOA/Target: Muscarinic receptor agonist
IUPAC Name: 1-azabicyclo[2.2.2]octan-3-yl acetate
SMILES: CC(=O)OC1CN2CCC1CC2
Sponsor: Visus Therapeutics

  1. Drug Name (Brand Name): Modeyso
    Generic Name: dordaviprone
    Structure:


Approval Date: 8/6/2025
Indication:  H3 K27M–mutant diffuse midline glioma
Modality: Small molecule
MOA/Target: Antagonist of dopamine receptor D2/3 (DRD2/3) and agonist of mitochondrial caseinolytic protease P (ClpP).
IUPAC Name: 11-benzyl-7-[(2-methylphenyl)methyl]-2,5,7,11-tetrazatricyclo[7.4.0.02,6]trideca-1(9),5-dien-8-one
SMILES: CC1=CC=CC=C1CN2C(=O)C3=C(CCN(C3)CC4=CC=CC=C4)N5C2=NCC5
Sponsor: Jazz Pharmaceuticals (acquired Chimerix in April 2025)

Significance:  First systemic therapy FDA-approved for H3 K27M-mutant DMG, a previously untreatable aggressive midline brain tumor predominantly affecting children and young adults.

  1. Drug Name (Brand Name): Hernexeos
    Generic Name: zongertinib
    Structure:


Approval Date: 8/8/2025
Indication: NSCLC, HER2 mutant/metastatic
Modality: Small molecule
MOA/Target: HER2 (ERBB2) kinase inhibitor
IUPAC Name: N-[1-[4-[3-methyl-4-(1-methylbenzimidazol-5-yl)oxyanilino]pyrimido[5,4-d]pyrimidin-6-yl]piperidin-4-yl]prop-2-enamide
SMILES: CC1=C(C=CC(=C1)NC2=NC=NC3=CN=C(N=C32)N4CCC(CC4)NC(=O)C=C)OC5=CC6=C(C=C5)N(C=N6)C
Sponsor: Boehringer Ingelheim

Significance: Marks Boehringer Ingelheim’s first U.S. oncology drug approval and introduces the first oral HER2-selective TKI for NSCLC—offering a convenient, tolerable alternative to Enhertu, the HER2-targeting ADC from AstraZeneca and Daiichi-Sankyo.

  1. Drug Name (Brand Name): BRINSUPRI
    Generic Name: brensocatib
    Structure:

Approval Date: 8/12/2025
Indication: Non-cystic fibrosis bronchiectasis (NCFB)
Modality: Small molecule
MOA/Target: Reversible inhibitor of dipeptidyl peptidase 1 (DPP1)
IUPAC Name: (2S)-N-[(1S)-1-cyano-2-[4-(3-methyl-2-oxo-1,3-benzoxazol-5-yl)phenyl]ethyl]-1,4-oxazepane-2-carboxamide
SMILES: CN1C2=C(C=CC(=C2)C3=CC=C(C=C3)CC@@HNC(=O)[C@@H]4CNCCCO4)OC1=O
Sponsor: Insmed
Significance: First FDA-approved therapy for non-cystic fibrosis bronchiectasis and first DPP1 inhibitor for neutrophil-mediated diseases, reducing exacerbations by 21% (10 mg) and 20% (25 mg) in the phase 3 ASPEN trial, addressing neutrophilic inflammation, a root cause of NCFB progression (NEJM, 2025; DOI: 10.1056/NEJMoa2411664).

Note: Expected late-2025 approvals include aficamten (PDUFA: Sept 26) and tolebrutinib (PDUFA: Sept 28).

Read more