Takeda’s EXKIVITY▼(mobocertinib) granted Conditional Marketing Authorisation in Great Britain

• Takeda UK Ltd. is pleased to announce that the Medicines and Healthcare products Regulatory Agency (MHRA) has granted a conditional marketing authorisation to EXKIVITY (mobocertinib) as a monotherapy treatment for adult patients with epidermal growth factor receptor (EGFR) Exon 20 insertion mutation-positive (Exon20ins+) locally advanced or metastatic non-small cell lung cancer (NSCLC), who have received prior platinum-based chemotherapy.^1,2
• EGFR Exon20ins NSCLC is an extremely rare type of lung cancer that mainly affects younger people and non-smokers; historically, it has had a very poor prognosis due the aggressive nature of the disease and there being no targeted treatments routinely available for this specific type of lung cancer.³
• The considerable unmet need in this patient population means oral mobocertinib has been granted an Innovation Passport by the MHRA, enabling participation and approval via Project Orbis, an international programme that reviews and approves promising cancer drugs to help patients access treatments faster.¹
• Takeda will now ensure that eligible patients across the UK have access to mobocertinib, whilst broad NHS reimbursement is sought.

LONDON, UK, 18^th March 2022 – Takeda UK Ltd. is pleased to announce that the Medicines and Healthcare products Regulatory Agency (MHRA) has granted a conditional marketing authorisation to EXKIVITY (mobocertinib) in Great Britain as a monotherapy treatment for adult patients with epidermal growth factor receptor (EGFR) Exon 20 insertion mutation-positive (Exon20ins+) locally advanced or metastatic non-small cell lung cancer (NSCLC), who have received prior platinum-based chemotherapy.^1,2 This approval marks a significant milestone for patients with this rare and aggressive disease, as there have previously been no targeted treatments NHS reimbursed for this specific type of lung cancer.³ This also marks the first Takeda medicine to be approved via Project Orbis.

EGFR Exon20ins+ NSCLC mainly affects younger people and non-smokers and carries a worse prognosis than other EGFR mutations. This is because of the aggressive nature of the disease and there being no targeted treatments NHS reimbursed that specifically target the EGFR Exon20ins mutation. Current treatment options for other EGFR mutations (tyrosine kinase inhibitors (TKIs) and chemotherapy) only provide limited benefit for Exon20ins+ NSCLC patients.^3-7 EGFR Exon20ins mutations account for five to 10 percent of all EGFR NSCLC cases and approximately two percent of all NSCLC cases.^7-11 Due to the rarity of the disease and the typical patient profile, it can often go undiagnosed for some time meaning most patients are diagnosed at Stage IV.

Angela Terry, Chair of EGFR Positive UK commented on the MHRA decision: “The impact of a cancer diagnosis is devastating enough, but to then understand that there are no treatments routinely available for your specific type of disease can often make patients feel frustrated and alone. So, 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.”

Mobocertinib is a first-in-class, oral TKI that was granted an Innovation Passport by the MHRA, satisfying the entry point for Project Orbis.^1,12 This is due to the significant unmet need in this rare and aggressive cancer and the ability of mobocertinib to provide patients with an oral targeted treatment that has demonstrated clinically meaningful outcomes, alongside a manageable safety profile.¹² The Project Orbis programme provides a framework for concurrent submission and review of oncology products among international partners. It aims to deliver faster patient access to innovative cancer treatments with potential benefits over existing therapies. Project Orbis is coordinated by the US Food and Drug Administration (FDA).

Professor Sanjay Popat, Consultant Medical Oncologist, The Royal Marsden NHS Foundation Trust commented: “The accelerated approval of mobocertinib is testament to the difference this treatment could make to patients that are in critical need of a targeted treatment option. The clinically meaningful benefits and generally manageable side effect profile that mobocertinib can offer to patients marks a step change in the treatment of this disease and provides hope to patients and their families. The oral administration also adds further value to this treatment option, not only from a patient experience perspective, but also in reducing the number of hospital visits for patients whilst we still navigate the Global pandemic.”

The conditional marketing authorisation of mobocertinib for EGFR Exon20ins+ NSCLC in the platinum pre-treated setting is based on a phase I/II multicentre, single-arm, open-label study. Eligible patients (N=86) with EGFR Exon20ins+ NSCLC who had previously been treated with platinum-based chemotherapy received mobocertinib at a dose of 160 mg once daily until disease progression or intolerable toxicity.^12,2

Mobocertinib met the primary endpoint by demonstrating a confirmed objective response rate (cORR) of 26 percent, as assessed by an independent review committee (IRC) and, in addition, achieved a cORR of 34 percent as per investigator-assessment.² Additional data in 28 platinum pre-treated patients receiving mobocertinib at 160 mg once daily from a phase I/II dose-finding and expansion study demonstrated a 36 percent cORR, as assessed by an IRC, and 39 percent as per investigator.²

A pooled analysis of platinum pre-treated patients receiving mobocertinib 160 mg (n=114) demonstrated a cORR of 28 percent (35 percent per investigator assessment), a median duration of response of 17.5 months, a median progression free survival of 7.3 months and a median overall survival of 24 months, all assessed by an IRC. This pooled analysis was presented at ASCO 2021.^12,13

The safety profile observed with mobocertinib was generally manageable and was considered consistent with that of other TKIs. The most common adverse reactions (>20%) were diarrhoea, rash, nausea, stomatitis, vomiting, decreased appetite, paronychia, fatigue, dry skin, and musculoskeletal pain. Nineteen patients (17%) discontinued due to AEs, most commonly diarrhoea (4%) and nausea (4%).^12,13

Takeda will now ensure that eligible patients across the UK have access to mobocertinib, whilst broad NHS reimbursement is sought. For example, Takeda is working with NHS England to make mobocertinib available to patients in England via a National Orbis Drug Access Arrangement.

Mobocertinib will also be assessed by UK health technology assessment bodies, starting with the National Institute for Health and Care Excellence (NICE) which aims to publish its recommendation towards the end of 2022.

Emma Roffe, Oncology Country Head – UK & Ireland, Takeda UK Ltd. said: “Takeda is extremely pleased that the promise of mobocertinib has been recognised by the MHRA via Project Orbis, and that approval has been expedited for patients in dire need of targeted treatment options. We have worked in close partnership with the clinical community, the regulatory authority and NHS England to demonstrate the value of this innovative treatment and continue to have patients front of mind in our decision making by ensuring that mobocertinib is made available to patients prior to potential NHS reimbursement later in the year.”

Contact

Media in UK

Danielle Smith

T: +44 7796 714 210

danielle.smith@takeda.com