Crizotinib for paediatric ALK-positive anaplastic large cell lymphoma or inflammatory myofibroblastic tumour
Anaplastic large cell lymphoma (ALCL) and inflammatory myofibroblastic tumour (IMT) are both rare conditions that usually affect children and young adults. ALCL is a type of cancer that occurs when T-cells, which contribute to the body’s immune system, become abnormal. IMTs are most commonly non-cancerous (benign), however in some cases the tumour can become cancerous (malignant). Both conditions can be ALK-positive (ALK+), this means that the tumour cells have a mutation in anaplastic lymphoma kinase (ALK) resulting in uncontrolled cell replication. These conditions may not be able to be treated through surgical removal of the tumour (unresectable); they may also come back after treatment (relapse) or be resistant to current treatment options (refractory).
Pembrolizumab in combination with lenvatinib for advanced melanoma – first line
Pembrolizumab is an immunotherapy, meaning it stimulates the body’s immune system by triggering T-cells (a type of white blood cells) to find and kill cancer cells. Lenvatinib is a targeted therapy drug that inhibits cancer growth by preventing the formation of new blood vessels. These drugs modulate different aspects of tumour biology, combining them, may result in improved efficacy and help overcome resistance to immunotherapy. Pembrolizumab in combination with lenvatinib (pembrolizumab administered intravenously and lenvatinib administered orally) would offer an additional first-line treatment option for previously untreated and unresectable advanced melanoma.
Liposomal bupivacaine for post-operative pain in children
Liposomal bupivacaine is in clinical development for the treatment of post-operative pain in paediatric patients. Post-operative pain is a common occurrence in patients who have undergone surgery. Managing post-operative pain is important in order for patients to have a positive post-operative experience and to enhance their recovery, and for the prevention of chronic post-surgical pain in the long-term.
Cemiplimab in combination with chemotherapy for advanced or metastatic non-small cell lung cancer – first-line
Cemiplimab in combination with chemotherapy is in clinical development for the treatment of advanced or metastatic non-small cell lung cancer (NSCLC). NSCLC makes up the majority of lung cancers in the UK and at the metastatic stage (stage IV), the disease has already spread from the lungs to other sites. Symptoms of lung cancer include a persistent cough, shortness of breath, coughing up blood, aches and pains in the chest or shoulder, loss of appetite, weight loss and fatigue. Most patients with NSCLC are diagnosed at the advanced/metastatic stage where curative treatment with surgery is unsuitable. Advanced NSCLC is not usually curable; there is therefore the need for additional treatment options.
Vadadustat for treating anaemia in chronic kidney disease
Vadadustat is in development for the treatment of anaemia in patients with chronic kidney disease (CKD). Anaemia is a condition where the body has fewer red blood cells (RBC) to carry oxygen throughout the body resulting in a decline in function of the body’s organs and tissues. Symptoms of anaemia in CKD patients include weakness, fatigue, headaches, dizziness and difficulty breathing. Anaemia is common in people with CKD because diseased kidneys produce less erythropoietin (EPO) which is needed to make RBCs. Current treatment for anaemia in CKD involves erythropoietin stimulating agents (ESAs) however, they may result in adverse side-effects so there is a need for safer treatment options.
Tirzepatide for treating type 2 diabetes mellitus
Tirzepatide is a new type of drug that is administered by subcutaneous injection and acts on two proteins known as the gastric inhibitory peptide (GIP) and glucagon-like peptide-1 (GLP-1) and results in more insulin being released from the pancreas. Tirzepatide has the potential to improve blood sugar levels and increase weight loss compared to current treatment options. If licenced, tirzepatide will offer an additional treatment option for patients with T2DM.
Setmelanotide for obesity in Bardet-Biedl syndrome and Alström syndrome
Setmelanotide is a protein that binds to a specific receptor to activate areas in the brain that control appetite. This reduces hunger sensations and therefore obesity. This is important in BBS and AS as obesity and hyperphagia are symptoms in both syndromes and these conditions can have damaging effects on the health of patients. If licensed, setmelanotide, given as an injection under the skin, will offer a first pharmacological treatment for BBS and AS.
Cabozantinib in combination with atezolizumab for advanced hepatocellular carcinoma –first line
Oral cabozantinib is a tyrosine kinase inhibitor (TKI) that works by blocking the activity of enzymes known as tyrosine kinases which can be found in certain receptors in cancer cells. Intravenous atezolizumab is a monoclonal antibody, a type of protein designed to recognise and attach to a protein called PD-L1, which is present on many cancer cells. Their combination has shown promising antitumour activity and tolerability in patients with solid tumours including HCC. If licensed, cabozantinib in combination with atezolizumab will provide an additional first-line treatment option for patients with advanced HCC.
Loncastuximab Tesirine for relapsed or refractory diffuse large B-cell lymphoma – third line
Loncastuximab tesirine is administered by intravenous infusion. It is an antibody-drug conjugate (ADC) where an antibody is joined to a drug that is designed to kill lymphoma cells. The antibody targets a protein called CD19, which is found on the surface of lymphoma cells. The drug is then released into the lymphoma cells resulting in the death of these cells. If licenced, loncastuximab tesirine will offer an additional treatment option for patients with relapsed or refractory DLBCL.