EGFR+ lung cancer, a non-smoking-related type of lung cancer caused by nonhereditary gene mutations, specifically the EGFR exon 20 insertion mutation, has limited treatment options beyond chemotherapy. In 2021, a tyrosine kinase inhibitor called Mobocertinib (Exkivity) was approved for the treatment of this mutation. However, the manufacturer withdrew the drug from use in the United States in late 2023, and the United Kingdom is set to follow suit soon. Campaigners are concerned about the lack of treatment options for patients with this mutation, potentially reducing lifespan and increasing mortality rates. This mutation accounts for up to 10% of EGFR+ lung cancer cases and typically has a poorer prognosis compared to other mutations.
Despite most lung cancer cases being linked to smoking, non-small cell lung cancers such as EGFR+ lung cancers, caused by mutations of the epidermal growth factor receptor, are not smoking-related. These cancers often present differently with atypical symptoms such as shoulder pain or musculoskeletal symptoms rather than the typical symptoms associated with smoking-related lung cancers. EGFR exon 20 insertion mutation is the third most common cause of EGFR+ lung cancer, affecting up to 10% of cases and presenting a challenge in terms of treatment due to limited options available. Mobocertinib, the recently approved tyrosine kinase inhibitor for this mutation, showed promise in early clinical trials in terms of tolerability, slowing cancer progression, and increasing survival time.
Unfortunately, Mobocertinib was voluntarily withdrawn from use in the United States in October 2023 due to the drug failing to show a significant effect on progression-free survival in phase 3 clinical trials. Despite no significant safety concerns, the drug did not meet the desired primary endpoint and confirmatory data requirements for approval. This withdrawal raises concerns among patients and advocates, as Mobocertinib was the only licensed treatment specifically designed for the EGFR exon 20 insertion mutation in the United Kingdom. Patients relying on this medication now face a potential treatment gap that may impact their prognosis and quality of life.
The withdrawal of Mobocertinib from use in the U.K. will leave patients with limited treatment options, particularly those with the EGFR exon 20 insertion mutation. The drug’s withdrawal is not due to safety concerns but rather its failure to meet efficacy endpoints in recent clinical trials. This decision has implications for patients’ access to effective treatments and may lead to reduced lifespans and increased mortality rates. Alternative treatments, such as aminvantamab, are available privately but not funded by the National Health Service (NHS), highlighting the disparities in access to potentially life-saving medications for patients in the U.K.
Campaigners and healthcare professionals are advocating for measures to ensure continued access to effective treatments for patients with the EGFR exon 20 insertion mutation. They emphasize the importance of expediting approval processes for alternative medications like aminvantamab to address the treatment gap left by Mobocertinib’s withdrawal. Patients already on Mobocertinib may have access to compassionate use programs to continue their treatment if they are deriving clinical benefit from the medication. Ensuring access to effective treatments for patients with rare mutations like EGFR exon 20 insertion is crucial for improving outcomes and quality of life for individuals facing this challenging diagnosis.
In conclusion, the withdrawal of Mobocertinib from use in the U.K. poses challenges for patients with the EGFR exon 20 insertion mutation, who may now face limited treatment options. Campaigners, healthcare professionals, and advocates are calling for alternative medications like aminvantamab to be made available to NHS patients to address this gap in treatment. Ensuring access to effective treatments for rare mutations like EGFR exon 20 insertion is crucial for improving outcomes and quality of life for individuals with EGFR+ lung cancer. Collaboration between stakeholders, healthcare authorities, and pharmaceutical companies is essential to address these issues and provide optimal care for patients with rare mutations.