AstraZeneca, Daiichi Sankyo get USFDA Breakthrough Therapy status for lung cancer drug Enhertu

UK: AstraZeneca and Daiichi Sankyo Company, Limited has been granted Breakthrough Therapy Designation (BTD) in the US for the treatment of patients with metastatic non-small cell lung cancer (NSCLC) whose tumours have a HER2 mutation and with disease progression on or after platinum-based therapy.

NSCLC is the most common type of lung cancer, and the prognosis is particularly poor for patients with metastatic disease as only about 6-10% will be alive five years after diagnosis. Approximately 2-4% of patients with NSCLC have a HER2 mutation.

The Food and Drug Administration (FDA)'s BTD is designed to accelerate the development and regulatory review of potential new medicines that are intended to treat a serious condition and address a significant unmet medical need. The new medicine needs to have shown encouraging early clinical results that demonstrate substantial improvement on a clinically significant endpoint over available medicines.

José Baselga, Executive Vice President, R&D Oncology, said: "Today's news is very welcome as we continue to evaluate the potential of Enhertu to help patients with this devastating type of lung cancer. Targeted treatments and immunotherapies are demonstrating tremendous advancements, but there remains an unmet medical need for patients with HER2 mutations who are not benefiting from such therapies or for those whose cancer continues to progress."

Gilles Gallant, Senior Vice President, Global Head, Oncology Development, Oncology R&D, Daiichi Sankyo, said: "We are encouraged by the promising evidence of activity seen with Enhertu in patients with advanced lung cancer and a HER2 mutation. We look forward to working closely with the FDA on the potential for Enhertu to become the first HER2-directed therapy approved for non-small cell lung cancer."

The FDA granted BTD based on data from the ongoing Phase II DESTINY-Lung01 trial currently testing Enhertu, a HER2-directed antibody-drug conjugate (ADC), in patients with HER2-mutant (HER2m) metastatic NSCLC, and data from the Phase I trial published in Cancer Discovery. An interim analysis from DESTINY-Lung01 will be presented during the 2020 American Society of Clinical Oncology ASCO20 Virtual Scientific Program, 29 to 31 May 2020.

The overall safety and tolerability profile of Enhertu in the ongoing DESTINY-Lung01 trial is consistent with that seen in the Phase I trial. The most common adverse events to date (n=42) are gastrointestinal and haematological including nausea, alopecia, anaemia, decreased appetite and decreased neutrophil count. There have been five cases of drug-related interstitial lung disease (ILD) and pneumonitis in patients with HER2m NSCLC, all of which were Grade 2. There have been no ILD-related deaths.

This is the third BTD granted for Enhertu in the US. Last week, Enhertu received BTD in patients with HER2-positive unresectable or metastatic gastric or gastroesophageal junction adenocarcinoma who have received two or more prior regimens including trastuzumab. Enhertu received BTD in 2017 for HER2-positive metastatic breast cancer and received approval in December 2019.

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