Breakthrough Therapy Designations — March 2026
Breakthrough Therapy
Designations — March 2026
10 Breakthrough Therapy designations were granted in March 2026, spanning oncology, rare disease, infectious disease, respiratory disease, haematology, and metabolic indications. 7 were granted in China (NMPA), 3 in the United States (FDA).
10 BTDs in March 2026 — 7 in China, 3 in the US
All data sourced from the March 2026 monthly pharmaceutical report, Table 1.
In March 2026, 10 Breakthrough Therapy designations were granted, spanning a diverse range of therapeutic areas from oncology and rare disease to infectious disease and metabolic dysfunction. China’s NMPA granted 7 of the 10 designations, with targets including KRAS G12D, EGFR/c-Met, DprE1, PDE4/PDE3, ALK2, CD276/Top I, Syk, and THR-β. The FDA granted 3 designations: Venglustat (UGCG, Gaucher Disease Type 3), Sutacimig (TLT1/factor VIIa, Thrombasthenia), and HSK-31679 (THR-β, MASH).
| Drug | Target | Drug Type | Indication | Developer | Region · Date |
|---|---|---|---|---|---|
| Amivantamab-VMJM/Hyaluronidase | c-Met · EGFR | Bispecific antibody · Enzyme | Squamous Cell Carcinoma of Head and Neck | Janssen R&D LLC | China · Mar 31 |
| GFH-375 | KRAS G12D | Small molecule drug | KRAS G12D mutation pancreatic cancer | Genfleet Therapeutics (Shanghai) | China · Mar 31 |
| Quabodepistat | DprE1 | Small molecule drug | Multidrug resistant pulmonary tuberculosis | Otsuka Beijing Research Institute | China · Mar 30 |
| HSK-39004 | PDE4 · PDE3 | Small molecule drug | Pulmonary Disease, Chronic Obstructive | Sichuan Haisco Pharmaceutical | China · Mar 27 |
| Venglustat | UGCG | Small molecule drug | Gaucher Disease, Type 3 | Sanofi | US · Mar 18 |
| Zilurgisertib | ALK2 | Small molecule drug | Myositis Ossificans | Incyte Biosciences Shanghai | China · Mar 18 |
| YL-201 | CD276 · Top I | Antibody drug conjugate (ADC) | Extensive stage Small Cell Lung Cancer | Suzhou Medilink Therapeutics | China · Mar 10 |
| Sovleplenib | Syk | Small molecule drug | Warm autoimmune hemolytic anemia | HUTCHMED (China) Ltd. | China · Mar 10 |
| Sutacimig | TLT1 · factor VIIa | Bispecific antibody | Thrombasthenia | HEMAB THERAPEUTICS INC. | US · Mar 5 |
| HSK-31679 | THR-β | Small molecule drug | Metabolic Dysfunction Associated Steatohepatitis | Haisco Pharmaceutical Group | China · Mar 4 |
Four notable BTD drugs from March 2026
Selected for first-in-class target, rare disease focus, or novel modality — all data from the March 2026 monthly report.
GFH-375 — KRAS G12D in pancreatic cancer
GFH-375 is a small molecule drug targeting KRAS G12D, developed by Genfleet Therapeutics (Shanghai), Inc. It received Breakthrough Therapy designation from China’s NMPA on March 31, 2026 for KRAS G12D mutation pancreatic cancer. KRAS G12D is one of the most prevalent and historically undruggable oncogenic mutations in solid tumours.
KRAS G12D · Small molecule · Pancreatic cancerVenglustat — Gaucher Disease Type 3
Venglustat is a small molecule drug targeting UGCG, developed by Sanofi. It received FDA Breakthrough Therapy designation on March 18, 2026 for Gaucher Disease, Type 3 — a rare neuronopathic lysosomal storage disorder where enzyme replacement therapy cannot address the neurological component of the disease.
UGCG · Small molecule · Rare neuronopathicYL-201 — CD276/Top I ADC in extensive stage SCLC
YL-201 is an antibody drug conjugate (ADC) targeting CD276 and Top I, developed by Suzhou Medilink Therapeutics Ltd. It received Breakthrough Therapy designation from China’s NMPA on March 10, 2026 for extensive stage Small Cell Lung Cancer, a high-unmet-need indication with limited options beyond first-line chemotherapy.
CD276 · Top I · ADC · Extensive SCLCSutacimig — TLT1/factor VIIa bispecific in Thrombasthenia
Sutacimig is a bispecific antibody targeting TLT1 and factor VIIa, developed by HEMAB THERAPEUTICS INC. It received FDA Breakthrough Therapy designation on March 5, 2026 for Thrombasthenia — a rare inherited platelet disorder with severe bleeding manifestations and limited treatment options beyond platelet transfusion and recombinant factor VIIa.
TLT1 · factor VIIa · Bispecific · Rare haematology4 signals from the March 2026 BTD data
Strategic takeaways derived from the 10 Breakthrough Therapy designations granted in March 2026.
China leads with 7 of 10 March 2026 BTDs
China’s NMPA granted 7 of the 10 March 2026 Breakthrough Therapy designations: Amivantamab (EGFR/c-Met, HNSCC), GFH-375 (KRAS G12D, pancreatic cancer), Quabodepistat (DprE1, MDR-TB), HSK-39004 (PDE4/PDE3, COPD), Zilurgisertib (ALK2, Myositis Ossificans), YL-201 (CD276/Top I, SCLC), and Sovleplenib (Syk, warm AIHA). This reflects the deepening of China’s innovative biopharma pipeline across oncology, rare disease, and infectious disease.
Novel target diversity: KRAS G12D, ALK2, DprE1, TLT1
The March 2026 BTD cohort covers several targets with limited prior approved therapies: KRAS G12D (GFH-375, pancreatic cancer), ALK2 (Zilurgisertib, Myositis Ossificans), DprE1 (Quabodepistat, MDR-TB), and TLT1/factor VIIa (Sutacimig, Thrombasthenia). Each represents a target area where BTD status signals clinical evidence of meaningful differentiation in a high-unmet-need setting.
Rare disease prominent: 3 of 10 BTDs in ultra-rare indications
3 of the 10 March 2026 BTDs target ultra-rare indications: Venglustat (Gaucher Disease Type 3, Sanofi), Zilurgisertib (Myositis Ossificans, Incyte Biosciences Shanghai), and Sutacimig (Thrombasthenia, HEMAB THERAPEUTICS). All three involve conditions with high unmet need and limited approved alternatives, consistent with the BTD framework’s emphasis on serious conditions with preliminary evidence of substantial improvement.
Modality mix: small molecules dominate, ADC and bispecific present
Of the 10 March 2026 BTDs, 7 are small molecule drugs (GFH-375, Quabodepistat, HSK-39004, Venglustat, Zilurgisertib, Sovleplenib, HSK-31679), 1 is an ADC (YL-201, CD276/Top I), and 2 are bispecific antibodies (Amivantamab-VMJM/Hyaluronidase with enzyme, Sutacimig). The small molecule dominance reflects the continued regulatory confidence in orally bioavailable agents for novel oncology and rare disease targets.
Breakthrough Therapy designations March 2026 — key questions
10 Breakthrough Therapy designations were granted in March 2026.
The 10 drugs that received Breakthrough Therapy designation in March 2026 were: GFH-375 (KRAS G12D, pancreatic cancer), Amivantamab-VMJM/Hyaluronidase (c-Met/EGFR, head and neck squamous cell carcinoma), Quabodepistat (DprE1, multidrug resistant tuberculosis), HSK-39004 (PDE4/PDE3, COPD), Venglustat (UGCG, Gaucher Disease Type 3), Zilurgisertib (ALK2, Myositis Ossificans), YL-201 (CD276/Top I, extensive stage SCLC), Sovleplenib (Syk, warm autoimmune hemolytic anemia), Sutacimig (TLT1/factor VIIa, Thrombasthenia), and HSK-31679 (THR-β, MASH).
7 of the 10 Breakthrough Therapy designations in March 2026 were granted in China by the NMPA. The remaining 3 were granted in the United States by the FDA.
March 2026 BTDs spanned oncology (KRAS G12D pancreatic cancer, head and neck squamous cell carcinoma, SCLC), rare disease (Gaucher Disease Type 3, Myositis Ossificans, Thrombasthenia), infectious disease (multidrug resistant tuberculosis), respiratory disease (COPD), haematology (warm autoimmune hemolytic anemia), and metabolic disease (MASH).
Breakthrough Therapy designation is granted by the FDA (or equivalent agencies in other regions) to expedite the development and review of drugs for serious conditions where preliminary clinical evidence indicates substantial improvement over existing therapies. It is not an approval — the drug still requires completion of clinical trials and a full regulatory review before approval can be granted.
Companies receiving BTDs in March 2026 include: Genfleet Therapeutics (Shanghai) for GFH-375, Janssen R&D LLC for Amivantamab-VMJM/Hyaluronidase, Otsuka Beijing Research Institute for Quabodepistat, Sichuan Haisco Pharmaceutical for HSK-39004, Sanofi for Venglustat, Incyte Biosciences Shanghai for Zilurgisertib, Suzhou Medilink Therapeutics for YL-201, HUTCHMED (China) Ltd. for Sovleplenib, HEMAB THERAPEUTICS INC. for Sutacimig, and Haisco Pharmaceutical Group for HSK-31679.
Data on this page is sourced from the PatSnap March 2026 monthly pharmaceutical report. All Breakthrough Therapy designation data is from Table 1 of that report. Represents a snapshot of available records as of March 2026.