Xeomin for sialorrhea in children and adolescents aged 2 to 17 years
Xeomin is one formulation of botulinum neurotoxin type A. It is injected into salivary glands and reduces saliva production. Currently, it is recommended as a specialist option for treating sialorrhea in children and adolescents, but is not yet licensed for this indication. It is an alternative to anticholinergic drugs which are also used to reduce saliva flow, but which can have side effects in a number of patients. If licensed, Xeomin will offer an additional treatment option for chronic sialorrhea in children and adolescents aged 2 to 17 years.
Icosapent ethyl for reducing the risk of cardiovascular events
Icosapent ethyl is in clinical development as a treatment to reduce the risk of cardiovascular events in high-risk patients who have their cholesterol levels controlled with statin treatment, but have elevated triglycerides and other cardiovascular risk factors. Cardiovascular events include heart attack, angina and stroke. These diseases are the main cause of death in the UK, accounting for over a quarter of deaths each year. Patients receiving statin treatment are still at a high risk and would benefit from treatment to reduce cardiovascular events.
Upadacitinib for Ankylosing Spondylitis
Upadacitinib acts by selectively blocking a protein called Janus-Associated Kinase 1 (JAK1 and JAK1/3). JAKs contribute to the processes within the cell to produce an immune or inflammatory response. There is an emerging body of evidence establishing that JAK dependent enzymes are major contributors to the progression of immunemediated diseases such as AS and that blocking such enzymes can be beneficial. Upadacitinib is taken orally and if licensed, it will offer an additional treatment option for patients with active AS.
Avatrombopag in addition to standard of care for chronic immune thrombocytopenia
Avatrombopag is given as an oral tablet and works by mimicking the action of a hormone called thrombopoietin (TPO) which is responsible for causing pre-curser cells to mature into platelets. Avatrombopag binds to TPO receptors resulting in increased platelet production and increased platelet count. Results from clinical studies have demonstrated that avatrombopag is safe and efficacious. If licensed, avatrombopag could offer an additional treatment option for patients with chronic ITP who have had limited response to previous therapy.
Avatrombopag for chemotherapy induced thrombocytopenia in patients with nonhaematological cancers
Avatrombopag is administered orally. It works by attaching to a hormone called thrombopoietin, which stimulates the production of platelets by attaching to receptors (targets) in the bone marrow and helping in increasing the platelet count. Avatrombopag does not interact with polyvalent cations (calcium, magnesium, iron, selenium, zinc, etc.) in foods, mineral supplements, or antacids that could reduce systemic exposure and efficacy. If licensed, avatrombopag will provide a treatment option for CIT in patients with active non-haematological cancers.
LN-145 for recurrent, metastatic or persistent cervical carcinoma
LN-145 is composed of T-cells (a type of immune cell) that are collected from the patient and grown to large numbers in the laboratory. They are then administered intravenously into the patient alongside IL-2 (a protein that activates T-cells), in order to encourage them to recognise and destroy the tumour. Therefore, if licensed, LN-145 would offer an additional treatment option to patients with recurrent, metastatic or persistent cervical cancer, who currently have limited treatment options.
Daratumumab in addition to bortezomib, lenalidomide and dexamethasone for multiple myeloma – first line
Daratumumab is a type of immune therapy that is administered subcutaneously and acts by inhibiting the growth of cancer cells in MM via a surface protein called CD38. If licenced it is hoped daratumumab in addition to bortezomib, lenalidomide and dexamethasone could provide better long term outcomes for patients with newly diagnosed multiple myeloma who are ineligible for high-dose chemotherapy with autologous stem cell transplant.
Relugolix in combination with estradiol/norethindrone acetate for moderate to severe symtpoms of uterine fibroids
Relugolix is a small molecule that binds to the gonadotropin-releasing hormone receptor in the pituitary gland, decreasing the release of hormones which control oestrogen and progesterone production by the ovaries. Results from clinical trials demonstrated that relugolix in combination with estradiol and norethisterone acetate reduced menstrual bleeding in women with uterine fibroids. Relugolix is administered orally, and if licensed would offer an additional treatment option for women moderate to severe symptoms associated with uterine fibroids.
Cemiplimab as a monotherapy for advanced or metastatic non-small-cell lung cancer whose tumours express PD-L1 – first line
Cemiplimab is a type of protein called an antibody, which can bind to PD-1 and prevent it interacting with PD-L1. Therefore, it allows the T-cells to attack the cancer cells. Cemiplimab is administered by intravenous infusion. The results from clinical trials have suggested that intravenous administration of cemiplimab increases overall survival in individuals with advanced/metastatic NSCLC who are positive for PD-L1. Therefore, if licensed, cemiplimab would offer an additional treatment option for patients with advanced/metastatic PD-L1 positive NSCLC.
177Lu-PSMA-617 for metastatic castrationresistant prostate cancer – third line
177Lu-PSMA-617 delivered via intravenous infusion. It works by releasing an energetic beta particle to precisely deliver cell-killing radiation to the site of disease to kill cancer cells. Earlier studies indicate that 177Lu-PSMA-617 may demonstrate clinical benefit in patients with mCRPC in the third-line setting, where patients have no clear standard of care, having exhausted all key treatment options. If licensed, 177LuPSMA-617 will provide a treatment option for patients in this setting.