March 2026 Expedited Review Pathway Drugs
March 2026 Expedited
Review Pathway Drugs
In March 2026, there were 104 drugs under Expedited Review pathways worldwide, including 55 Orphan Drug, 10 Breakthrough Therapy, 11 Fast Track, 14 Priority Review, and more across all major regulatory agencies.
104 ERP designations in March 2026 — Orphan Drug leads at 53%
Breakdown across 12 expedited review pathway types, spanning FDA, EMA, NMPA, PMDA, and other agencies worldwide.
In March 2026, there were 104 drugs under Expedited Review pathways worldwide, including 55 Orphan Drug, 2 Rare Pediatric Disease, 10 Breakthrough Therapy, 11 Fast Track, 14 Priority Review, 2 Conditional Marketing Approval, 2 Regenerative Medicine Advanced Therapy, 3 PRIME, 2 Accelerated Approval, 1 Advanced Therapy Medicinal Products, 1 Promising Innovative Medicine, and 1 Qualified Infectious Disease Product.
Orphan Drug designation dominated the month, reflecting the continued pipeline focus on rare and ultra-rare diseases. The 10 Breakthrough Therapy designations — spanning oncology, rare metabolic disease, infectious disease, and cardiovascular — signal FDA and NMPA prioritisation of programs with preliminary evidence of substantial improvement over existing therapies.
10 Breakthrough Therapy designations in March 2026
Across oncology, rare disease, infectious disease, and metabolic indications — spanning FDA (US) and NMPA (China) regulatory agencies.
| Drug | Target | Indication | Developer | Region · Date |
| GFH-375 | KRAS G12D | KRAS G12D mutation pancreatic cancer | Genfleet Therapeutics (Shanghai) | China · Mar 31 |
| Amivantamab-VMJM/Hyaluronidase | c-Met · EGFR | Squamous Cell Carcinoma of Head and Neck | Janssen R&D LLC | China · Mar 31 |
| Quabodepistat | DprE1 | Multidrug resistant pulmonary tuberculosis | Otsuka Beijing Research Institute | China · Mar 30 |
| HSK-39004 | PDE4 · PDE3 | Pulmonary Disease, Chronic Obstructive | Sichuan Haisco Pharmaceutical | China · Mar 27 |
| Venglustat | UGCG | Gaucher Disease, Type 3 | Sanofi | US · Mar 18 |
| Zilurgisertib | ALK2 | Myositis Ossificans | Incyte Biosciences Shanghai | China · Mar 18 |
| YL-201 | CD276 · Top I | Extensive stage Small Cell Lung Cancer | Suzhou Medilink Therapeutics | China · Mar 10 |
| Sovleplenib | Syk | Warm autoimmune hemolytic anemia | HUTCHMED (China) Ltd. | China · Mar 10 |
| Sutacimig | TLT1 · factor VIIa | Thrombasthenia | HEMAB THERAPEUTICS INC. | US · Mar 5 |
| HSK-31679 | THR-β | Metabolic Dysfunction Associated Steatohepatitis | Haisco Pharmaceutical Group | China · Mar 4 |
Notable ERP drugs from March 2026
Selected for first-in-class mechanism, rare disease focus, or novel modality from the March 2026 ERP table.
GFH-375 — KRAS G12D in pancreatic cancer
GFH-375 is a small molecule drug targeting KRAS G12D, developed by Genfleet Therapeutics (Shanghai). It received Breakthrough Therapy designation from China’s NMPA in March 2026 for KRAS G12D mutation pancreatic cancer — one of the most difficult-to-treat solid tumour indications with limited approved targeted options.
KRAS G12D · Small molecule · Pancreatic cancerVenglustat — Gaucher Disease Type 3
Venglustat is a small molecule drug targeting UGCG, developed by Sanofi. Its Breakthrough Therapy designation for Gaucher Disease Type 3 reflects serious unmet need in a rare neuronopathic lysosomal storage disorder where existing enzyme replacement therapy cannot address the neurological component of the disease.
UGCG · Small molecule · Rare neuronopathicYL-201 — CD276/Top I ADC in SCLC
YL-201 is an antibody drug conjugate (ADC) targeting CD276 and Top I, developed by Suzhou Medilink Therapeutics. It received Breakthrough Therapy designation in China for extensive stage Small Cell Lung Cancer — a high-unmet-need indication where ADC approaches targeting CD276 represent an emerging strategy beyond platinum-based chemotherapy.
CD276 · Top I · ADC · SCLCMarnetegragene autotemcel — LAD-1 gene therapy
Marnetegragene autotemcel is a CD18-targeting hematopoietic stem cell gene therapy developed by Rocket Pharmaceuticals, receiving Accelerated Approval from the FDA on March 26, 2026 for Leukocyte Adhesion Deficiency Type 1 — an ultra-rare primary immunodeficiency with no previously approved curative therapy.
CD18 · Gene therapy · HSCT · LAD-14 signals from the March 2026 ERP data
Strategic takeaways for biopharma intelligence teams derived from the 104 ERP designations in March 2026.
China accounts for 7 of 10 BTDs
7 of the 10 Breakthrough Therapy designations in March 2026 were granted by China’s NMPA, covering KRAS G12D, EGFR/c-Met, DprE1, PDE4/PDE3, ALK2, CD276/Top I, Syk, and THR-β. This concentration reflects China’s accelerating regulatory sophistication and the maturing of its oncology and rare disease pipeline.
Orphan Drug at 53% — rare disease pipeline dominates
55 of the 104 March 2026 ERP designations were Orphan Drug, reflecting continued industry investment in rare and ultra-rare disease programs. This concentration spans gene therapy, AAV-based approaches, enzyme replacement alternatives, and novel small molecules across lysosomal storage disorders, retinal dystrophies, and rare oncology indications.
ADC modality prominent across multiple ERP pathways
Multiple ADC programs appeared across the March 2026 ERP table, including YL-201 (CD276/Top I, BTD, SCLC), Risvutatug Rezetecan (Top I/CD276, Orphan Drug, SCLC), ADCE-D01 (Top I/MRC2, Orphan Drug, Soft Tissue Sarcoma), HDM-2017 (CDH17/Top I, Orphan Drug, multiple), and Fam-trastuzumab deruxtecan (HER2/Top I, Priority Review and CMA). The payload diversity signals continued ADC platform expansion.
Gene therapy and cell therapy active in Orphan & RMAT pathways
Gene therapy programs in March 2026 ERPs include Marnetegragene autotemcel (CD18, AAC, LAD-1), Sonpiretigene Isteparvovec (Opsin receptors, AAV, retinal dystrophy), PBGENE-DMD (dystrophin, AAV, Duchenne MD), AAV9-LMNA (LMNA, progeria), ST-002 (merlin, NF2), and MZ-1866 (TCF4, AAV, Pitt-Hopkins Syndrome). RMAT designations were granted to KSD-101 and NG-01.
March 2026 ERP designations — key questions answered
In March 2026, there were 104 drugs under Expedited Review pathways worldwide.
Orphan Drug designation led with 55 designations in March 2026, accounting for more than half of all 104 ERP designations that month.
10 Breakthrough Therapy designations were granted in March 2026, including drugs targeting KRAS G12D, UGCG, ALK2, EGFR/c-Met, Syk, DprE1, PDE4/PDE3, THR-β, CD276/Top I, and TLT1/factor VIIa.
14 Priority Review designations were granted in March 2026.
11 Fast Track designations were granted in March 2026.
In addition to Orphan Drug (55), Breakthrough Therapy (10), Fast Track (11), and Priority Review (14), March 2026 ERP designations included: 2 Conditional Marketing Approval, 2 Regenerative Medicine Advanced Therapy (RMAT), 3 PRIME, 2 Accelerated Approval, 1 Advanced Therapy Medicinal Products (ATMP), 1 Promising Innovative Medicine (PIM), 2 Rare Pediatric Disease, and 1 Qualified Infectious Disease Product (QIDP).
Data on this page is sourced from the PatSnap March 2026 monthly pharmaceutical report and Synapse database. Represents a snapshot of available records as of March 2026.