The National Institute for Health and Care Excellence (NICE) has published a positive recommendation for EXKIVITY (mobocertinib) for treating EGFR Exon 20 insertion mutation-positive advanced non-small-cell lung cancer after platinum-based chemotherapy within a Final Appraisal Document (FAD).
Key highlights:
- NICE has published a positive recommendation for EXKIVITY (mobocertinib) for treating EGFR Exon 20 insertion mutation-positive advanced non-small-cell lung cancer after platinum-based chemotherapy within a Final Appraisal Document (FAD).
- This decision from NICE represents a step-change in the clinical management of this extremely rare and aggressive cancer and provides eligible patients with a long-awaited oral, targeted therapy.
- It follows the conditional marketing authorisation granted in Great Britain by the Medicines and Healthcare products Regulatory Agency (MHRA) as part of Project Orbis in March 2022.
In March 2022, Great Britain was the first country, outside of the USA, to grant a conditional marketing authorisation to mobocertinib, a first-in-class, oral tyrosine kinase inhibitor (TKI) specifically designed to target EGFR Exon 20 insertion mutations. A significant milestone hailed as ‘game-changing’ by NHS England at the time. This final positive NICE recommendation for mobocertinib reflects the close collaboration between Takeda, NICE, NHS England and the clinical and patient communities to ensure the value of mobocertinib was realised for patients in significant need of a targeted therapy for this extremely rare and aggressive cancer. It also overturns the draft negative decision published within the Appraisal Consultation Document (ACD) in August 2022.
Angela Terry, chair of EGFR Positive UK, commented: “This positive recommendation is fantastic news for patients. Particularly as they have previously faced frustration and isolation due to the limited treatment options available for this aggressive disease. Having a medicine specifically designed to tackle their type of cancer will give patients the much-needed hope of improved outcomes and increased confidence that the management of their disease is catching up with other types of lung cancer. We are pleased that Takeda was able to work closely with NICE and NHS England to demonstrate the value of mobocertinib and enable much needed access for patients.”
The expedited regulatory approval of mobocertinib in Great Britain was made possible via Project Orbis, an international regulatory framework to deliver faster access to innovative cancer medicines. Mobocertinib satisfied the entry point for Project Orbis when the MHRA granted the treatment an Innovation Passport via the Innovative Licensing and Access Pathway (ILAP); this was due to its ability to provide patients who have a high unmet need with an oral targeted treatment that has demonstrated clinically meaningful outcomes, alongside a manageable safety profile.
Professor Sanjay Popat, consultant medical oncologist, The Royal Marsden NHS Foundation Trust, commented: “Mobocertinib offers the patient and clinical community a significant step-change in the battle against this rare disease; not only as a proven effective targeted treatment but one that can be taken orally at home – providing enhanced patient experience and reducing the number of required hospital visits. This positive decision from NICE means that clinical management of this devastating type of lung cancer is brought more in line with other forms of the disease where targeted treatments are already routinely available. Now is the time to ensure patients are effectively diagnosed, through optimising biomarker testing for this specific mutation.”
Takeda worked diligently to ensure that eligible patients across Great Britain had access to mobocertinib as quickly as possible following its conditional marketing authorisation. We worked with NHS England to make it available to patients in England via a National Orbis Drug Access Arrangement (List D) on a budget-neutral basis and have also maintained compassionate use across the devolved nations to ensure equity of access whilst HTAs are ongoing. Reimbursement in Scotland is pending and depends on the ongoing evaluation by the Scottish Medicines Consortium (SMC).
Emma Roffe, Oncology Country head – UK & Ireland, Takeda UK, said: “We are extremely pleased with this decision and that patients in England and Wales will now have routine access to mobocertinib. The significant unmet need in this rare cancer has been the true inspiration for Takeda; to ensure access to this innovative medicine as soon as possible and in our collaboration with NICE and NHSE to overcome the initial draft negative decision. We acknowledge that the inherent uncertainties that come with such rare disease can make access decisions challenging, but we did everything we could to address those uncertainties and ensure the value of mobocertinib was realised for the benefit of patients.”