The European Commission (EC) has granted approval for the label extension of
Kaftrio (ivacaftor/tezacaftor/elexacaftor), in a combination regimen with ivacaftor.

This has been recommended for the treatment of CF in patients aged six- to 11-years-old who have at least one F508del mutation in the CF transmembrane conductance regulator (CFTR) gene.

The approval means that more than 1,500 children are now eligible to receive a
treatment for the underlying cause of CF for the first time.

Kaftrio, in combination with ivacaftor, is an oral medicine designed to increase the quality and function of the CFTR protein at the cell surface. This increases the amount of mature protein at the cell surface by binding different sites on the CFTR protein.

Ivacaftor is designed to facilitate the ability of CFTR proteins to transport salt and water across the cell membrane. In combination, the medicines work together to help clear mucus from the airways.

The National Institute for Health and Care Excellence (NICE) has issued a final appraisal determination (FAD) recommending MSD’s (known as Merck in the US and Canada) Keytruda (pembrolizumab) when used in combination with carboplatin and paclitaxel as an option for untreated metastatic squamous non-small-cell lung cancer (NSCLC) in adults.

The approval follows a successful monitoring and patient access period through the Cancer Drugs Fund. The decision from NICE marks pembrolizumab as the first immunotherapy to be routinely used in first line settings for metastatic squamous NSCLC, regardless of PD-L1 expression, when combined with chemotherapy.

In the last 40 years, the survival rate of lung cancer has changed very little, from 3% to 5%. There are usually no symptoms of lung cancer in the early stages and between 72-76% of people are diagnosed at a late stage, when the cancer has spread outside the lungs.

The National Institute for Care Excellence (NICE) has published a positive final appraisal document recommending routine NHS use of venclyxto (venetoclax) with azacitidine for the treatment of aggressive blood cancer acute myeloid leukaemia (AML). Approximately 3,200 people are diagnosed with AML in the UK every year.

The treatment – developed by AbbVie and Roche – is recommended as an option for untreated AML in adults when intensive chemotherapy is unsuitable.

The combination offers patients a treatment option with significantly improved overall survival, increased rates of rapid and durable remission and blood transfusion
independence, compared to treatment using azacitidine alone.

Median survival for AML is just six to ten months. During this period patients often require hospitalisation and frequent blood transfusions, resulting in a significant impact on quality of life.

Venetoclax treatment can be taken at home, reducing patient dependency on blood transfusions and potentially meaning fewer hospital visits for the clinically vulnerable patients who are more susceptible to coronavirus infections.

A new blood test, developed by cancer researchers at the University of Oxford, can detect whether cancers have metastasised in the body and is the first of its kind, using nuclear magnetic resonance (NMR) metabolomics. These identify the presence of biomarkers in the blood called metabolites which are produced by cancer cells.

The study, published in Clinical Cancer Research, a journal of the American Association of Cancer Research, analysed samples from 300 patients with non-specific but concerning symptoms of cancer.

Cancer was correctly detected in 19 out of every 20 patients with cancer and among those with cancer, metastatic disease was identified with an overall accuracy of 94%.

It is the first technology able to determine the metastatic status of a cancer from a blood test, without prior knowledge of the primary cancer type. It is hoped the test will help clinicians detect cancer and assess cancer stage.