FDA Grants Breakthrough Therapy Status to Patritumab Deruxtecan for EGFR+ Metastatic NSCLC

Patients with EGFR-mutated metastatic non–small cell lung cancer may achieve benefit from treatment with patritumab deruxtecan, which was granted breakthrough therapy designation by the FDA.

Patritumab deruxtecan (U3-1402), a potential first-in-class HER3 directed antibody-drug conjugate, was granted breakthrough therapy designation by the FDA for the treatment of patients with metastatic or locally advanced, EGFR-mutant non–small cell lung cancer (NSCLC), according to a press release from developer Daiichi-Sankyo.1

The regulatory decision, which is designed to accelerate the development and regulatory review process of potential new therapies, was based on data from a dose escalation study and 2 expansion cohorts from a 3-cohort trial.

“The breakthrough therapy designation for patritumab deruxtecan acknowledges the need for new treatment approaches to overcome resistance and improve survival in patients with metastatic TKI-resistant, EGFR-mutated [NSCLC],” Ken Takeshita, MD, global head of R&D at Daiichi Sankyo, said in a press release. “We are proud that the FDA has once again recognized our innovative science and technology and we look forward to bringing this potential first-in-class HER3 directed antibody drug conjugate to patients with this specific type of lung cancer as quickly as possible.”

Data from a phase 1 trial (NCT03260491) assessing the efficacy of patritumab deruxtecan in a heavily pretreated population of patients with EGFR-mutant NSCLC that have become resistant to other TKIs were read out at the 2021 American Society of Clinical Oncology Annual Meeting: Lung Cancer.2 A 5.6 mg/kg dose of the treatment resulted in a confirmed objective response rate of 39% (95% CI, 26%-52%) in a population of patients who were previously treated with a TKI and platinum-based therapy (n = 57). Additionally, treatment with patritumab deruxtecan resulted in a disease control rate of 72% (95% CI, 59%-83%), as well as a median progression-free survival of 8.2 months (95% CI, 4.0–not evaluable).

Notably, this is the first breakthrough therapy designation for patritumab deruxtecan.


  1. Patritumab deruxtecan granted U.S. FDA breakthrough therapy designation in patients with metastatic EGFR-mutated non-small cell lung cancer. News release. Daiichi-Sankyo. December 23, 2021. Accessed January 3, 2022. https://bit.ly/3qECNWu
  2. Jänne PA, Baik CS, Su W-C, et al. Efficacy and safety of patritumab deruxtecan (HER3-DXd) in EGFR inhibitor-resistant, EGFR-mutated (EGFRm) non-small cell lung cancer (NSCLC). J Clin Oncol. 2021;39(suppl 15):9007. doi:10.1200/JCO.2021.39.15_suppl.9007