NON-SMALL cell lung cancer (NSCLC) with epidermal growth factor receptor exon 20 insertion (EGFRExon20ins) is a very rare type of lung cancer that affects mostly young individuals and non-smokers. Until now, there was no targeted treatment for this aggressive cancer, and consequently, the disease has a poor prognosis. To fulfil this unmet need, researchers aimed to study a novel drug target, mobocertinib, that could treat this rare lung cancer.
Mobocertinib was tested in a Phase I/II multicentre, open-label study involving 86 patients with NSCLC EGFRExon20ins that had been previously treated with platinum-based chemotherapy. Patients were given a daily 160 mg dose of mobocertinib until their cancer worsened or they had intolerable side effects. Results from this trial showed that patients had a median progression-free survival of 7.3 months, and the primary endpoint was met by reaching a 26% confirmed objective response rate.
Overall, the study had positive outcomes in proving the effectiveness of mobocertinib to treat NSCLC EGFRExon20ins. Importantly, the drug had a tolerable safety profile, with the most common adverse events being diarrhoea, rash, and fatigue. Recently, the Medicines and Healthcare products Regulatory Agency (MHRA) has approved mobocertinib in the UK to treat eligible patients with rare NSCLC EGFRExon20ins. The approval process was fast-tracked by Project Orbis, an initiative that aims to approve cancer drugs faster in order to help patients receive treatment as soon as possible.
Angela Terry, chair of EGFR Positive UK, shared her thoughts on the decision to approve mobocertinib for NSCLC EGFRExon20ins: “It is fantastic news that EGFR Exon20 insertion NSCLC patients will now have the opportunity to benefit from an oral, targeted treatment that offers the hope of potentially improved outcomes. We also hope that having targeted treatments available will mean that patients are tested more effectively at diagnosis for this specific mutation — something that has historically been quite variable across the country.”
The next steps for mobocertinib include ensuring that all patients who are eligible for treatment with mobocertinib will have access to the drug; the National Institute for Health and Care Excellence (NICE) will also assess their recommendation regarding mobocertinib later this year. In the meantime, the approval of mobocertinib marks a significant milestone in the treatment of NSCLC EGFRExon20ins and will potentially improve outcomes for people with this rare lung cancer.