FDA Oncology Update January 2026: New Horizons in Precision Medicine

The landscape of oncology has seen a flurry of activity as 2026 begins, with the FDA granting critical designations to several novel therapies targeting rare and difficult-to-treat malignancies. From pioneering RAS inhibitors to innovative cell therapies for rare blood cancers, the first few weeks of January have underscored a continued shift toward precision medicine and off-the-shelf solutions for high-unmet-need populations.

Here are the most significant FDA updates from January 2026, highlighting the drugs poised to reshape the standard of care.

FDA Grants Breakthrough Status to Sevabertinib in 1L HER2+ NSCLC

On January 6, the FDA granted breakthrough therapy designation (BTD) to sevabertinib (Hyrnuo, formerly BAY 2927088) for the first-line treatment of patients with unresectable or metastatic non–small cell lung cancer (NSCLC) harboring activating HER2 (ERBB2) mutations.

FDA Grants Orphan Drug Designation to CK0804 in Myelofibrosis

Also on January 6, CK0804, an investigational allogeneic regulatory T-cell therapy, received orphan drug designation (ODD) for the treatment of myelofibrosis.

FDA and EMA Grant Orphan Drug Status to Novel Imaging Drug for Pancreatic Cancer

On January 7, the FDA and European Medicines Agency awarded ODD to CPI-008 (cRGD-ZW800-1), a novel integrin-targeted, zwitterionic imaging agent, for margin detection of pancreatic cancer during surgery.

FDA Clears IND of Off-the-Shelf In Vivo CAR-T Therapy for R/R Myeloma

On January 7, the FDA granted clearance to the investigational new drug application (NDA) for KLN-1010, a novel off-the-shelf in vivo gene therapy, for the treatment of patients with relapsed/refractory (R/R) multiple myeloma (MM). This decision follows a recent presentation of promising first-in-human data at the 67th American Society of Hematology Annual Meeting and Exposition in December 2025.

FDA Orphan Drug Status Granted to Anti-CDH17 ADC in Pancreatic Cancer

Also on January 7, the FDA granted ODD to ARB1002, an investigational anti-CDH17 antibody-drug conjugate, for the treatment of pancreatic cancer.

FDA Fast-Tracks PARG Inhibitor for mBRCA Platinum-Resistant Ovarian Cancer

On January 8, the investigational small molecule inhibitor ETX-19477 earned fast track designation (FTD), accelerating its clinical development for treatment of adult patients with BRCA-mutated, platinum-resistant, high-grade serous ovarian cancer.

Zoldonrasib Earns FDA Breakthrough Therapy Status in KRAS G12D-Mutant NSCLC

Also on January 8, the FDA granted BTD to zoldonrasib (RMC-9805), a RAS(ON) G12D-selective inhibitor, in KRAS G12D-mutated, locally advanced or metastatic NSCLC previously treated with anti–PD-1/PD-L1 therapy and platinum-based chemotherapy.

FDA Orphan Drug Designation Given to NP-G2-044 in Pancreatic Cancer

On January 12, NP-G2-044 (Prilukae), a novel oral fascin inhibitor earned ODD for the treatment of pancreatic cancer. This designation covers the use of the agent as monotherapy and in combination with anti–PD-1 immune checkpoint inhibitors.

FDA Grants Orphan Drug Designation for Gotistobart in Squamous NSCLC

On January 12, the FDA granted ODD to gotistobart (BNT316/ONC-392) for the treatment of patients with squamous NSCLC. The announcement highlights the investigational anti–CTLA-4 monoclonal antibody’s potential in treating an aggressive subset of lung cancer that has historically seen limited therapeutic breakthroughs following the failure of first-line immunotherapy.

For a Second Time, FDA Denies Approval of Tab-Cel in EBV+ PTLD

Also on January 12, the FDA issued a complete response letter for the biologics license application (BLA) of the T-cell immunotherapy tabelecleucel (tab-cel; Ebvallo) seeking accelerated approval for treatment of patients 2 years and older with relapsed or refractory Epstein-Barr virus–positive posttransplant lymphoproliferative disease who have received at least 1 prior therapy, including an anti-CD20–containing regimen. This marks the FDA’s second denial of the agent, a year after the first on January 15, 2025.

FDA Grants Breakthrough Status to ICT01 Plus Venetoclax/Azacitidine in AML

On January 13, the FDA granted BTD to ICT01 (IPN60340), an investigational anti-BTN3A antibody, in combination with venetoclax (Venclexta) and azacitidine for first-line treatment of acute myeloid leukemia.

Ropeginterferon Alfa’s Supplemental BLA Moves on as PDUFA Date Issued

Alson on January 13, the FDA accepted the supplemental BLA for ropeginterferon alfa-2b-njft (Besremi), a treatment for adult patients with essential thrombocythemia. The BLA was issued a Prescription Drug User Fee Act (PDUFA) date of August 30, 2026.

FDA Grants Priority Review to Gedatolisib for Advanced Breast Cancer

On January 20, the FDA accepted for filing a NDA for gedatolisib, an investigational PI3K and mTOR inhibitor, for the treatment of certain patients with advanced breast cancer. The regulatory agency granted priority review for the application and assigned a PDUFA target action date of July 17, 2026.

FDA Grants Breakthrough Therapy Designation to Sofetabart Mipitecan for Platinum-Resistant Ovarian Cancer

Also on January 20, the FDA granted BTD to sofetabart mipitecan (LY4170156) for the treatment of patients with platinum-resistant epithelial ovarian, fallopian tube, or primary peritoneal cancer who have previously received bevacizumab (Avastin) and mirvetuximab soravtansine-gynx (Elahere), if eligible.

FDA Grants Breakthrough Therapy Designation to Sofi-Cel for R/R T-ALL/LBL

On January 21, the FDA granted BTD to soficabtagene geleucel (sofi-cel; WU-CART-007) for the treatment of R/R T cell acute lymphoblastic leukemia (T-ALL) and T cell lymphoblastic lymphoma (T-LBL).

BNT113 Garners Fast-Track Designation in PD-L1+ HPV16+ HNSCC

On January 21, the FDA granted FTD to the investigational mRNA immunotherapy BNT113 for the treatment of patients with HPV16+ head and neck squamous cell carcinoma expressing PD-L1.

Zavabresib Granted Orphan Drug Designation for Myelofibrosis

On January 21, the FDA has granted the BET small molecule inhibitor, zavabresib (OPN-285) ODD for the treatment of myelofibrosis.

FDA OKs Guardant360 CDx for Encorafenib Combo in BRAF V600E+ Metastatic CRC

On January 22, the FDA granted approval to Guardant360 CDx, a companion diagnostic to help identify patients with BRAF V600E-mutant metastatic colorectal cancer who may benefit from treatment with encorafenib (Braftovi) plus cetuximab (Erbitux) and chemotherapy.

FDA Grants Breakthrough Designation for Bezuclastinib in GIST

On January 26, the FDA granted BTD for bezuclastinib in combination with sunitinib (Sutent) for the treatment of patients with gastrointestinal stromal tumors (GIST).

FDA Grants Fast Track Designation to IBI3003 for R/R Multiple Myeloma

On January 27, the FDA granted FTD to IBI3003, a first-in-class trispecific antibody, for the treatment of patients with R/R MM with progression after at least 4 prior lines of therapy, including a proteasome inhibitor, an immunomodulatory drug, and an anti-CD38 monoclonal antibody.

FDA Approves Daratumumab Quadruplet for Transplant-Ineligible Myeloma

Also on January 27, the FDA approved daratumumab and hyaluronidase-fihj (Darzalex Faspro) in combination with bortezomib (Velcade), lenalidomide (Revlimid), and dexamethasone (D-VRd) for adults with newly diagnosed MM who are ineligible for autologous stem cell transplant.

CTD402 Gains FDA Orphan Drug Designation in T-ALL and T-LBL

On January 27, the FDA granted ODD to CTD402, a novel allogeneic CAR T therapy, for treatment of R/R T-ALL and T-LBL.

FDA Clears Larger Vial Size of Nelarabine Injection in T-ALL and T-LBL

On January 28, the FDA granted approval to a larger vial size of nelarabine injection (SH-111) for use in treatment of adult and pediatric patients with T-ALL and T-LBL. Nelarabine injection is a nucleoside metabolic inhibitor indicated for treatment of adult and pediatric patients aged 1 year and older with R/R disease following treatment with at least 2 chemotherapy regimens. This approval marks the agent’s second FDA approval after its initial approval in 2023 for a 250-mg/50-mL–sized vial.

FDA Grants Fast Track Designation to GPRC5D Bispecific LBL-034 for R/R Multiple Myeloma

On January 29, the FDA granted FTD to LBL-034, an investigational GPRC5D/CD3 bispecific T-cell engager for patients with R/R MM.