This week’s Oncology update highlights major oncology regulatory milestones, with multiple Breakthrough Therapy and Orphan Drug designations, global approvals, and late-stage clinical data across solid tumors. Momentum is especially strong in targeted therapies for lung, colorectal, pancreatic, breast, and rare molecularly defined cancers.
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🧬 Sevabertinib first-line BTD in US and China [1] [US/China • 06 Jan 2026]
Context: Preliminary evidence from SOHO-01 cohort F in untreated HER2-mutant NSCLC; follows US accelerated approval in previously treated disease in Nov 2025.
Key point: FDA and China CDE granted Breakthrough Therapy Designation for first-line sevabertinib in locally advanced or metastatic HER2-mutant NSCLC.
Implication: May influence prescriber choice and payer reviews pending full data.
🧪 Zoldonrasib BTD for KRAS G12D-mutant NSCLC [2] [US • 08 Jan 2026]
Context: Based on Phase 1 monotherapy cohort of RMC-9805-001 showing antitumor activity and acceptable safety (endpoint not specified).
Key point: FDA granted Breakthrough Therapy Designation to zoldonrasib for previously treated KRAS G12D-mutant NSCLC.
Implication: May influence prescriber choice and payer reviews pending full data.
💉 sac-TMT + pembrolizumab BTD in PD-L1-positive NSCLC [3] [China • 05 Jan 2026]
Context: OptiTROP-Lung05 Phase 3 met primary PFS endpoint with a positive OS trend per company.
Key point: China CDE granted BTD for sacituzumab tirumotecan plus pembrolizumab in first-line PD-L1 TPS ≥1% NSCLC without EGFR/ALK alterations.
Implication: May influence prescriber choice and payer reviews pending full data.
🧫 FDA “Study May Proceed” for (Z)-endoxifen in metastatic breast cancer [4] [US • 06 Jan 2026]
Context: (Z)-Endoxifen is a SERM/D with ER-targeted effects and PKC inhibition; not approved for any indication.
Key point: FDA cleared initiation of Atossa’s metastatic ER+/HER2- breast cancer study under a new IND.
Implication: Signals pipeline investment and modality expansion.
🛰️ Orphan Drug Designation for CPI-008 imaging agent in pancreatic cancer [5] [US/EU • 07 Jan 2026]
Context: Integrin-targeted, zwitterionic imaging drug for intraoperative margin detection; investigator-initiated Phase 2 imaging across cancers (details not specified).
Key point: FDA and EMA granted Orphan Drug Designation for CPI-008 for pancreatic cancer surgery.
Implication: Could inform practice and payer discussions; interpretation depends on study design and confounding control.
🧷 Orphan Drug Designation for ARB1002 ADC in pancreatic cancer [6] [US • 07 Jan 2026]
Context: Anti-CDH17 ADC completing IND-enabling work; Phase 1 entry planned in 2026 (n not stated).
Key point: FDA granted Orphan Drug Designation to ARB1002 for pancreatic cancer.
Implication: Signals pipeline investment and modality expansion.
📈 BREAKWATER Cohort 3, encorafenib+cetuximab+FOLFIRI in BRAF V600E mCRC [7] [10 Jan 2026]
Context: Phase 3 randomized cohort in previously untreated mCRC; safety consistent with known profiles; OS descriptive at ~10 months.
Key point: ORR 64.4% with encorafenib+cetuximab+FOLFIRI vs 39.2% with FOLFIRI±bevacizumab, BICR, p=0.001.
Implication: May influence prescriber choice and payer reviews pending full data.
🔩 Fast Track for Enolen implants in localized prostate cancer [8] [US • 08 Jan 2026]
Context: Enzalutamide-eluting implants for localized delivery; Phase 1 ongoing to assess safety and preliminary efficacy.
Key point: FDA granted Fast Track designation for Enolen in low to intermediate risk localized prostate cancer.
Implication: Signals pipeline investment and modality expansion.
💠 Needle-free delivery selected for melanoma DNA immunotherapy [9] [US • 09 Jan 2026]
Context: Scancell’s iSCIB1+ delivered via PharmaJet Stratis in SCOPE Phase 2; presentation cited PFS 74% at 16 months vs 50% at 11.5 months for SOC.
Key point: PharmaJet’s Stratis needle-free system selected for late-stage development of iSCIB1+ following Phase 2 update.
Implication: Could streamline initiation and adherence via remote prescribing and logistics.
🧠 Repotrectinib approved in China for NTRK fusion-positive solid tumors [10] [China • 06 Jan 2026]
Context: Approval covers TRK TKI-naive and TKI-pretreated patients; based on TRIDENT-1 Phase 1/2 efficacy and safety.
Key point: China NMPA approved AUGTYRO (repotrectinib) for adult patients with NTRK fusion-positive solid tumors.
Implication: Introduces competition that may affect pricing and formulary access.
Why it Matters
- First-line NSCLC acceleration across targets: HER2 and TROP2 programs gain expedited pathways in US and China.
- KRAS G12D targeting advances: BTD recognizes a new option for a high-need NSCLC subset without approved targeted therapies.
- mCRC regimen flexibility: BREAKWATER supports encorafenib combinations beyond mFOLFOX6, broadening potential use.
- Pancreatic cancer diversification: Orphan designations span intraoperative imaging and ADC modalities.
- China precision oncology access: Repotrectinib expands TRK options across prior-therapy status.
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FAQ
What did Bayer’s sevabertinib receive and for whom?
FDA and China CDE granted BTD for first-line sevabertinib in locally advanced or metastatic HER2-mutant NSCLC, supported by SOHO-01 cohort F and following US accelerated approval in Nov 2025 for previously treated disease [1].
Which KRAS program received BTD in NSCLC?
Revolution Medicines’ zoldonrasib, a RAS(ON) G12D-selective inhibitor, received FDA BTD for previously treated KRAS G12D-mutant NSCLC based on Phase 1 monotherapy data showing activity and tolerability [2].
What is sac-TMT’s new China designation and setting?
China CDE granted BTD for sacituzumab tirumotecan plus pembrolizumab as first-line therapy for PD-L1 TPS ≥1% NSCLC without EGFR/ALK alterations, after OptiTROP-Lung05 met PFS per company report [3].
How did Pfizer’s BREAKWATER Cohort 3 perform?
In untreated BRAF V600E mCRC, encorafenib+cetuximab+FOLFIRI achieved ORR 64.4% vs 39.2% for control, BICR, p=0.001; safety was consistent, with OS descriptive at about 10 months of follow-up [7].
What new approval did Zai Lab announce in China?
NMPA approved repotrectinib (AUGTYRO) for adult patients with NTRK fusion-positive solid tumors across TRK TKI-naive and TKI-pretreated populations, based on TRIDENT-1 [10].
Which pancreatic cancer programs got Orphan Drug Designation?
Curadel’s CPI-008 imaging agent received US and EU ODD for surgical margin detection [5], and Arbele’s ARB1002 anti-CDH17 ADC received US FDA ODD [6].
Entities
Sevabertinib (Bayer; Hyrnuo) • HER2-mutant NSCLC • SOHO-01 • Zoldonrasib (Revolution Medicines; RMC-9805) • KRAS G12D • Sacituzumab tirumotecan (sac-TMT; SKB264/MK-2870; Kelun-Biotech; Merck/MSD) • Pembrolizumab (Keytruda) • OptiTROP-Lung05 • (Z)-Endoxifen (Atossa) • CPI-008 (Curadel; cRGD-ZW800-1) • ARB1002 (Arbele; anti-CDH17 ADC) • BRAFTOVI (encorafenib; Pfizer) • Cetuximab (Erbitux) • FOLFIRI • BREAKWATER • Enolen (Alessa; enzalutamide-eluting implants) • PharmaJet Stratis • iSCIB1+ (Scancell) • Repotrectinib (AUGTYRO; Zai Lab/BMS) • NTRK fusion-positive tumors.
References
- https://www.bayer.com/media/en-us/bayer-receives-breakthrough-therapy-designation-in-the-us-and-china-for-sevabertinib-as-a-first-line-treatment-for-patients-with-her2-mutant-non-small-cell-lung-cancer/
- https://ir.revmed.com/news-releases/news-release-details/revolution-medicines-announces-fda-breakthrough-therapy-1
- https://www.prnewswire.com/news-releases/kelun-biotech-announces-breakthrough-therapy-designation-granted-in-china-for-sacituzumab-tirumotecan-sac-tmt-in-combination-with-immunotherapy-pembrolizumab-for-first-line-treatment-of-pd-l1-positive-nsclc-302652491.html
- https://investors.atossatherapeutics.com/2026-01-06-Atossa-Therapeutics-Receives-FDA-Study-May-Proceed-Letter-for-Z-Endoxifen-Investigational-New-Drug-Application-for-Metastatic-Breast-Cancer
- https://www.curadelpharma.com/2026/01/07/fda-and-ema-grant-orphan-drug-designation-for-curadels-cpi-008-a-targeted-zwitterionic-imaging-drug-for-pancreatic-cancer/
- https://www.prnewswire.com/news-releases/arbele-receives-fda-orphan-drug-designation-for-arb1002-an-anti-cdh17-antibodydrug-conjugate-for-the-treatment-of-pancreatic-cancer-302654913.html
- https://www.pfizer.com/news/press-release/press-release-detail/pfizers-braftovir-regimen-additional-chemotherapy-backbone
- https://alessatherapeutics.com/news/alessa-therapeutics-announces-fda-fast-track-designation-for-enolen-a-first-of-its-kind-treatment-for-localized-prostate-cancer/
- https://pharmajet.com/pharmajets-needle-free-injection-system-selected-to-administer-dna-immunotherapy-for-advanced-melanoma-in-late-stage-clinical-development-following-positive-phase-2-data/
- https://ir.zailaboratory.com/news-releases/news-release-details/zai-lab-announces-national-medical-products-administration-nmpa