Lumacaftor and Ivacaftor (Orkambi) for Cystic Fibrosis Homozygous for the F508del Mutation in Patients Aged 6‐11 Years
Lumacaftor and ivacaftor (Orkambi) is a medicine used to treat cystic fibrosis in patients aged 6 years and above who have a genetic mutation called the F508del mutation. This mutation affects the gene for a protein called cystic fibrosis transmembrane conductance regulator (CFTR) which is involved in regulating the production of mucus and digestive juices. Orkambi is used in patients who have inherited the mutation from both parents and therefore have the mutation in both copies of the CFTR gene. Orkambi is taken orally as tablets twice a day. It offers a new treatment option for patients with cystic fibrosis with the advantage of targeting the actual disease process rather than just the symptoms.
Mexiletine for the Symptomatic Treatment of Myotonic Disorders – First Line
Mexiletine is under registration in Europe for the symptomatic treatment of myotonic disorders. It is administered as an oral capsule and it exerts its action by reducing the rate of contraction in the heart and other muscles. Currently it is used unlicensed in the UK, meaning that it is not currently approved but some healthcare providers consider it to be potentially beneficial based on research or professional experience. There are currently no licensed treatment options available to treat symptoms of myotonia, therefore, if licensed, mexiletine will offer access to an approved treatment option.
Dupilumab for Children Aged 12 Years to 17 Years with Moderate to Severe Atopic Dermatitis
Dupilumab is a monoclonal antibody medicine that is currently in development as an injection under the skin (subcutaneous) for the treatment of moderate to severe AD in children. Dupilumab is already licensed in the UK for the treatment of moderate-to-severe atopic dermatitis in adults. If licensed, dupilumab will offer an additional treatment option for children aged 12 years to 17 years with moderate to severe atopic dermatitis uncontrolled on current therapies.
Triptorelin Pamoate (subcutaneous injection) for Prostate Cancer
Triptorelin is being developed as an injection under the skin (subcutaneous) for the treatment of locally advanced or metastatic prostate cancer. It is already marketed for this condition but is given by injection deep into the muscles (intramuscular). Triptorelin is an artificial analogue of natural gonadotropin‐releasing hormone that acts to slowly reduce the level of testosterone in the body. The first administration of triptorelin stimulates an increase in testosterone levels but prolonged administration leads to a fall in plasma testosterone or oestradiol to castrate levels which is maintained for as long as the product is administered. Triptorelin as a subcutaneous injection formulation has the potential advantage of improved safety and local tolerability when compared to intramuscular injection formulation.
CER-001 for Familial Primary Hypoalphalipoproteinemia
CER-001 is an engineered complex of the major structural protein of HDL, in combination with key phospholipids. Administered through intravenous (IV) infusion, CER-001 mimics the structure and function of natural HDL to stimulate the removal of excess cholesterol and other lipids. There is currently limited treatment options available specifically for FPHA. If licensed, CER-001 may offer a new treatment option for FPHA by aiding in the transport and elimination of excess cholesterol and other lipids in the liver.
KTE-C19 for Relapsed/Refractory B-precursor Acute Lymphocytic Leukaemia in Adults
KTE-C19 is new type of therapy where T-cells (a type of immune white blood cell) are collected from a patient and engineered to be able to recognise molecules on the surface of cancer cells, which triggers the T-cells to attack and kill the cancer cells. KTE-C19 is given to patients by a single infusion. If KTE-C19 is licenced for use in adults with refractory/relapsed acute lymphoblastic leukaemia, it will provide an additional, cancer-specific treatment option for this population who currently have limited treatment options.
Isatuximab in Addition to Pomalidomide and Dexamethasone for Relapsed and/or Refractory Multiple Myeloma
Isatuximab is in development as a treatment option for relapsed and refractory MM. It is intended to be added to pomalidomide and dexamethasone which are drugs already available to treat the condition. Isatuximab is administered intravenously as a solution concentrate and the unique way it acts may offer an additional treatment option for relapsed and refractory MM patients who have tried and failed to respond on current therapies.
Atezolizumab in Addition to Chemotherapy for Stage IV Squamous Non-Small Cell Lung Cancer – First Line
Atezolizumab is a monoclonal antibody designed to recognise and attach to a protein called ‘programmed death-ligand 1’ (PD-L1), which is present on the surface of many cancer cells. PD-L1 switches off immune cells that would otherwise attack cancer cells. By attaching to PD-L1 and reducing its effect, atezolizumab increases the ability of the immune system to attack the cancer cells and thereby slow down the progression of the disease. Atezolizumab is administered by intravenous infusion. If licensed, atezolizumab in addition to chemotherapy will offer an additional first line treatment option for patients with untreated, advanced, squamous non-small cell lung cancer.
Apalutamide in Addition to Abiraterone and Prednisone/Prednisolone for Metastatic Castration-resistant Prostate Cancer – First Line
Apalutamide is an oral tablet in development for the treatment of metastatic castration-resistant prostate cancer, when used in addition to abiraterone acetate and prednisone/prednisolone.
Apalutamide acts by blocking the androgen receptor (target binding site of various steroid hormones including testosterone) to prevent the effects of testosterone in the prostate and the body. If licensed, apalutamide in addition to abiraterone and prednisone/prednisolone will offer an additional treatment for metastatic castration-resistant prostate cancer in those who are not yet recommended to receive chemotherapy.
Selonsertib for Non-alcoholic Steatohepatitis (NASH)
Selonsertib is an investigational oral small molecule inhibitor of ASK1, a protein that mediates inflammation, apoptosis (cell death) and fibrosis in settings of oxidative stress. Oxidative stress can be increased in many pathological conditions including liver diseases such as NASH. NASH currently has no effective treatment apart from lifestyle interventions. If licensed, selonsertib will offer a new treatment option for NASH as no effective pharmacological therapies currently exist.