Breakthrough Therapy Designations — April 2026
Breakthrough Therapy
Designations — April 2026
11 Breakthrough Therapy designations were granted in April 2026, spanning oncology, rare haematology, neurology, and infectious disease. 7 were granted by China’s NMPA and 3 by the US FDA. Three of the 11 are ADCs — all pairing Top I with a tumour antigen.
All 11 BTDs from April 2026 — by region and date
All data sourced from the April 2026 monthly report, Table 2, rows 3–13.
| Drug | Target | Drug Type | Indication | Developer | Region · Date |
|---|---|---|---|---|---|
| Pegylated bovine carboxyhemoglobin | — | Recombinant protein | Acute Ischemic Stroke | Prolong Pharmaceuticals LLC | US · Apr 30 |
| Gamgertamig | BCMA · CD3 | Bispecific T-cell Engager (BiTE) | Immunoglobulin Light-Chain Amyloidosis | Keymed Biomedical Technology (Chengdu) | China · Apr 28 |
| IBI-363 | PD-1 · IL2RA | Bispecific antibody; Antibody fusion proteins | MSS/pMMR/MSI-L Colorectal Carcinoma | Innovent Biologics (Suzhou) | China · Apr 28 |
| NHWD-870 | BRD4 | Small molecule drug | Advanced NUT Carcinoma | Hunan Hengya Pharmaceutical Technology; Ningbo Wenda Pharmaceutical Technology | China · Apr 28 |
| TERN-701 | Bcr-Abl | Small molecule drug | Philadelphia chromosome-positive CML in chronic phase | Terns Pharmaceuticals, Inc. | US · Apr 27 |
| Risvutatug Rezetecan | Top I · CD276 | Antibody drug conjugate (ADC) | Castration-Resistant Prostatic Cancer | Shanghai Hansoh Biomedical | China · Apr 21 |
| VGA-039 | PROS1 | Monoclonal antibody | Von Willebrand Diseases | Star Therapeutics, Inc. | US · Apr 21 |
| Trastuzumab Rezetecan | Top I · HER2 | Antibody drug conjugate (ADC) | HER2 Positive Breast Cancer | Suzhou Suncadia Biopharmaceuticals | China · Apr 9 |
| SHR-A2102 | nectin-4 · Top I | Antibody drug conjugate (ADC) | Metastatic Cervical Carcinoma; Recurrent Cervical Cancer | Shanghai Hengrui Pharmaceutical | China · Apr 8 |
| Pembrolizumab / Hyaluronidase | PD-1 · Hyaluronic acid | Monoclonal antibody; Enzyme | Locally Advanced NSCLC; Metastatic NSCLC | MSD R&D (China) | China · Apr 8 |
| Plixorafenib | BRAF | Small molecule drug | Glioblastoma Harboring BRAF V600 Mutation | Fore Biotherapeutics, Inc. | US · Apr 7 |
Four notable BTD drugs from April 2026
TERN-701 — Bcr-Abl in Philadelphia chromosome-positive CML
TERN-701 is a small molecule drug targeting Bcr-Abl, developed by Terns Pharmaceuticals, Inc. It received FDA Breakthrough Therapy designation on April 27, 2026 for Ph+ CML in chronic phase. With 4 clinical trials and 1 patent at designation stage, it enters a 178-drug Bcr-Abl competitive class where resistance to prior TKIs remains the primary unmet need.
Bcr-Abl · Small molecule · Ph+ CML · BTDRisvutatug Rezetecan — Top I/CD276 ADC in CRPC
Risvutatug Rezetecan is a Top I/CD276 ADC developed by Shanghai Hansoh Biomedical Co. Ltd. It received BTD from China’s NMPA on April 21, 2026 for Castration-Resistant Prostatic Cancer. CD276 (B7-H3) is an immune checkpoint overexpressed on CRPC tumour cells and vasculature. This is the second CD276/Top I ADC to receive BTD in a single month, alongside YL-201 in SCLC.
Top I · CD276 · ADC · CRPC · BTDPlixorafenib — BRAF inhibitor for BRAF V600 glioblastoma
Plixorafenib is a small molecule drug targeting BRAF, developed by Fore Biotherapeutics, Inc. It received FDA Breakthrough Therapy designation on April 7, 2026 for Glioblastoma Harboring BRAF V600 Mutation — a genetically defined GBM subpopulation with poor prognosis and limited targeted therapy options. BRAF V600 mutations occur in approximately 2–3% of GBM cases.
BRAF · Small molecule · GBM V600 · BTDGamgertamig — BCMA/CD3 BiTE in AL amyloidosis
Gamgertamig is a BCMA/CD3 Bispecific T-cell Engager (BiTE) developed by Keymed Biomedical Technology (Chengdu) Co., Ltd. It received BTD from China’s NMPA on April 28, 2026 for Immunoglobulin Light-Chain Amyloidosis — a rare plasma cell dyscrasia driven by clonal plasma cells overproducing misfolded immunoglobulin light chains. BCMA is expressed on malignant plasma cells, making it a validated target in this context.
BCMA · CD3 · BiTE · AL amyloidosis · BTD4 signals from the April 2026 BTD data
Strategic takeaways derived from the 11 BTDs in the April 2026 monthly report.
3 ADC BTDs in April 2026 — all Top I-paired, all from China
Three ADCs received Breakthrough Therapy designation in April 2026: Risvutatug Rezetecan (Top I/CD276, CRPC, Hansoh), Trastuzumab Rezetecan (Top I/HER2, HER2+ breast, Suncadia), and SHR-A2102 (nectin-4/Top I, cervical cancer, Hengrui). All three pair a Top I payload with a tumour antigen targeting antibody, and all three are from China-based developers — reflecting the depth of China’s ADC innovation pipeline across multiple oncology indications.
China NMPA grants 7 of 11 April 2026 BTDs — spanning ADC, BiTE, bispecific, and small molecule
China’s NMPA granted 7 of the 11 April 2026 BTDs: Gamgertamig (BiTE, amyloidosis), IBI-363 (bispecific, CRC), NHWD-870 (small molecule, NUT carcinoma), Risvutatug Rezetecan (ADC, CRPC), Trastuzumab Rezetecan (ADC, HER2+ breast), SHR-A2102 (ADC, cervical), and Pembrolizumab/Hyaluronidase (mAb, NSCLC). This modality diversity — spanning four distinct drug types — reflects the breadth of China’s innovative biopharma pipeline in 2026.
Plixorafenib: BRAF-targeted therapy entering the high-unmet-need GBM space
Plixorafenib’s BTD for BRAF V600 mutation glioblastoma reflects FDA’s recognition of a genetically defined GBM subpopulation that currently lacks approved targeted therapy. While BRAF inhibitors (vemurafenib, dabrafenib) are established in melanoma and some other BRAF V600-mutant tumours, their utility in GBM is limited by blood-brain barrier penetration and intrinsic CNS tumour resistance mechanisms. Plixorafenib’s CNS-optimised BRAF inhibition is the differentiated claim underpinning BTD.
IBI-363: PD-1/IL-2Rα bispecific targeting MSS CRC — a historically immunotherapy-resistant subtype
IBI-363, a PD-1/IL2RA bispecific antibody fusion protein from Innovent Biologics, received BTD from China’s NMPA on April 28, 2026 for MSS/pMMR/MSI-L Colorectal Carcinoma — a subtype comprising roughly 95% of metastatic CRC that has historically shown poor response to PD-1/PD-L1 checkpoint inhibitors. Combining PD-1 blockade with IL-2Rα (IL-2 receptor alpha) agonism as a strategy to reinvigorate T-cell responses in immunologically “cold” MSS CRC is mechanistically novel and the likely basis for the BTD signal.
Breakthrough therapy designations April 2026 — key questions
11 Breakthrough Therapy designations were granted in April 2026.
The 11 drugs were: Pegylated bovine carboxyhemoglobin (Acute Ischemic Stroke, US), Gamgertamig (BCMA/CD3, Immunoglobulin Light-Chain Amyloidosis, China), IBI-363 (PD-1/IL2RA, MSS CRC, China), NHWD-870 (BRD4, NUT Carcinoma, China), TERN-701 (Bcr-Abl, Ph+ CML, US), Risvutatug Rezetecan (Top I/CD276, CRPC, China), VGA-039 (PROS1, Von Willebrand Diseases, US), Trastuzumab Rezetecan (Top I/HER2, HER2+ breast, China), SHR-A2102 (nectin-4/Top I, cervical cancer, China), Pembrolizumab/Hyaluronidase (PD-1, NSCLC, China), and Plixorafenib (BRAF, glioblastoma, US).
7 of the 11 April 2026 Breakthrough Therapy designations were granted by China’s NMPA. The remaining 3 were granted by the US FDA (TERN-701, VGA-039, Plixorafenib) plus Pegylated bovine carboxyhemoglobin also in the US.
3 ADCs received Breakthrough Therapy designation in April 2026: Risvutatug Rezetecan (Top I/CD276, CRPC), Trastuzumab Rezetecan (Top I/HER2, HER2+ breast cancer), and SHR-A2102 (nectin-4/Top I, cervical cancer). All three are from China-based developers and all pair a Top I payload with a tumour antigen targeting antibody.
Plixorafenib, a BRAF-targeting small molecule drug developed by Fore Biotherapeutics, Inc., received Breakthrough Therapy designation in the United States on April 7, 2026 for Glioblastoma Harboring BRAF V600 Mutation.
Data on this page is sourced from the PatSnap April 2026 monthly pharmaceutical report, Table 2 (ERP designations). Represents a snapshot of available records as of April 2026.