Liraglutide for weight management in obese adolescents aged 12-17 years old
Liraglutide acts like a hormone the body produces naturally that regulates appetite, known as glucagon-like-peptide (GLP-1). By activating areas of the brain that regulate appetite, liraglutide may decrease feelings of hunger which can lead to lower calorie intake and weight loss. If licensed, liraglutide may improve long-term outcomes for weight management in obese adolescents 12-17 years old who currently have limited treatment options.
Nivolumab in combination with cabozantinib for metastatic renal cell carcinoma – first-line
Nivolumab works by improving the activity of white blood cells thereby increasing the ability of the immune system to kill cancer cells. Cabozantinib works to stop signals that cancer cells use to divide and grow. It is thought that when used in combination, both drugs may be more effective than each drug on its own. If licenced, nivolumab in combination with cabozantinib may improve long-term outcomes in mRCC patients who currently have limited treatment options.
Lumasiran for primary hyperoxaluria type I
Lumasiran, which is administered as a subcutaneous injection, is designed to reduce the levels of an enzyme called glycolate oxidase produced by the liver. Oxalate production is therefore inhibited. By reducing oxalate production, lumasiran has the potential to prevent the actual disease process that develops in PH1. If licensed, lumasiran may provide the first pharmacological treatment option for patients with PH1 who do not have any approved treatment.
Nivolumab in combination with ipilimumab for oesophageal squamous cell carcinoma – first line
Nivolumab and ipilimumab are immune therapy medicinal products that are currently licensed as a combination treatment of advanced cancers such as melanoma and kidney cancer. Nivolumab works by improving the activity of white blood cells (T-cells) thereby increasing the ability of the immune system to kill cancer cells. Ipilimumab works in a different way but also to increase the activity of T-cells. It is thought that when used together, both drugs may be more effective than each on its own. Both drugs given by injection and the combination may offer a treatment option for patients with advanced, recurrent or metastatic oesophageal squamous cell carcinoma who have not been treated previously.
BCX7353 for prevention of acute attacks of angioedema in hereditary angioedema
BCX7353 is an oral treatment that works by blocking the activity the specific pathway that becomes overactive in patients with angioedema. By blocking this pathway, BCX7353 is expected to reduce the number of angioedema attacks. Early studies of BCX7353 has shown that it helped to prevent swelling and inflammation in HAE. In addition, BCX7353 has the distinct advantage over current treatment of being administered orally, making it easier to use for routine prevention of HAE attacks.
Olaparib for BRCAm or ATM mutated metastatic castration-resistant prostate cancer
Olaparib is taken orally and works by blocking a protein called poly [adenosine diphosphate-ribose] polymerase (PARP). PARP is an important protein which tries to fix damaged deoxyribonucleic acid (DNA). By blocking PARP from fixing damaged DNA, the tumour cells may die. If licensed, olaparib will offer an additional treatment option for men with metastatic castrate-resistant prostate cancer with BRCAm or ATM mutations who have progressed following a prior new hormonal agent.
Sotagliflozin for type 2 diabetes mellitus
Sotagliflozin is one of a new class of drugs that increase elimination of glucose in the urine. They offer the advantages of a reduced risk of low blood sugar. Sotagliflozin reduces the reabsorption of glucose from the intestines and kidneys, thus improving blood glucose control in diabetes mellitus. If licensed, sotagliflozin will offer an additional treatment option for patients with T2DM.
Tanezumab for moderate to severe chronic pain associated with osteoarthritis and chronic low back pain
Tanezumab is in clinical development for the treatment of moderate to severe chronic pain associated with osteoarthritis and chronic low back pain. Pain is an unpleasant sensory experience associated with damage to body tissues due to an injury, physical pressure, or inflammation of some part of the body. Chronic pain is persistent or recurrent and lasts for longer than 12 weeks. Moderate pain interferes significantly with daily living activities, and severe pain is disabling and causes an inability to perform daily living activities. Effective long-term treatment options for managing moderate to severe chronic pain are limited. Currently available pain medicines like opioids and analgesics may increase the risks of addiction, gastrointestinal, cardiovascular and renal problems.