Oral targeted treatment for non-small cell lung cancer granted conditional marketing authorisation

The Medicines and Healthcare products regulatory agency (MHRA) has granted conditional marketing authorisation to mobocertinib (Exkivity) in Great Britain.

The treatment will be available to 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.

The biopharmaceutical company Takeda say the 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.

No routinely available treatments until now

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 said: “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.”

She said it is therefore “fantastic news” that a targeted treatment is now available, and hopes that it will mean that “more patients are tested more effectively at diagnosis for this specific mutation “something that has historically been quite variable across the country.”

Clinically meaningful outcomes and a manageable safety profile

Mobocertinib was granted an Innovation Passport by the MHRA after it demonstrated clinically meaningful outcomes, alongside a manageable safety profile.

In a study of 86 patients with the disease, mobocertinib demonstrated a confirmed objective response rate (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 independent review committee.

The safety profile observed with mobocertinib was generally manageable and was considered consistent with that of other (tyrosine kinase inhibitors (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%).

Professor Sanjay Popat, Consultant Medical Oncologist, The Royal Marsden NHS Foundation Trust said the drug “marks a step change” in the treatment of this disease, with further value added as it is orally administered, reducing the number of hospital visits required by patients.

NICE aim to publish its recommendation by the end of the year

Takeda will now ensure that eligible patients across the UK have access to mobocertinib, whilst broad NHS reimbursement is sought.

The drug will also be assessed by National Institute for Health and Care Excellence (NICE) and other UK health technology assessment bodies, with the aim of publishing its recommendation by the end of 2022.