Sutimlimab for primary cold agglutinin disease
Sutimlimab is a first-in-class monoclonal antibody in development for the treatment of cold agglutinin disease, a rare form of autoimmune haemolytic anaemia, caused by cold-reacting autoantibodies. These antibodies bind to red blood cell membranes and destroy them, leading to anaemia. Symptoms include chronic debilitating fatigue, acrocyanosis, shortness of breath and other anaemia-related symptoms, leading to a poor quality of life and increased health resource utilization. In chronic cold agglutinin disease, the patient is more symptomatic during winter months. Therapeutic options may range from using warm clothing and avoiding exposure to cold weather to blood transfusions and chemotherapy.
Atezolizumab in combination with nab-paclitaxel as neoadjuvant treatment for early stage triple-negative breast cancer
Atezolizumab is a cancer medicine that enhances T-cell (part of the immune system) activity against tumours. Nab-paclitaxel is a chemotherapy that combines the chemotherapy drug paclitaxel with a protein called albumin. It inhibits cell growth by preventing cell division. The combination may offer an additional neoadjuvant treatment option to improve clinical efficacy in the treatment of people with early stage TNBC, an aggressive disease with no approved targeted therapy.
Edaravone for amyotrophic lateral sclerosis
Edaravone as an intravenous injection is in clinical development for people with amyotrophic lateral sclerosis (ALS). ALS is a neurological condition that affects nerve cells in the brain and
spinal cord. It results in gradual weakness and wasting of muscles of the body. Respiratory muscles are involved as the disease progresses, leading to shortness of breath and ultimately
death. Little is known about the cause of the disease, and there is currently no cure.
Human plasminogen for hypoplasminogenemia
Plasminogen (human) as intravenous infusion is in clinical development for people with hypoplasminogenemia. Hypoplasminogenemia is an ultra-rare, chronic, genetic condition associated with inflamed growths on the mucous membranes, the moist tissues that line body openings such as the eye, mouth, nasopharynx, trachea, and female genital tract. The growths are caused by the deposition of fibrin (a protein involved in blood clotting) and by inflammation. Growths can lead to severe medical problems including vision loss, ulcers of the gastrointestinal tract, and breathing difficulties caused by obstruction of the airway.
Polatuzumab vedotin in combination with rituximab and bendamustine for the treatment of diffuse large B-cell lymphoma
Polatuzumab vedotin is a first-in-class antibody drug specifically developed for the treatment of cancers that affect the blood and lymph system. It is a monoclonal antibody that acts by selectively binding to CD79b, a protein which is abundantly expressed on the surface of B-cells. It is administered as an intravenous infusion, absorbed by the cancer cells and the chemotherapy agent linked to the antibody releases inside the cancer cells and destroys them. Adding polatuzumab vedotin to bendamustine with rituximab may improve effectiveness in patients whose disease had come back or in whom other treatment had not worked well enough.
Angiotensin II acetate (Giapreza) for vasopressor resistant hypotension due to distributive shock
Angiotensin II acetate is in clinical development for the treatment of catecholamine and/or vasopressin resistant hypotension due to distributive shock. Shock is a critical condition in which blood pressure drops so low that the brain, kidneys and other vital organs cannot receive enough blood flow to function properly. Distributive shock is the most common type of shock that is most commonly caused by bacterial or fungal infection (septic shock) or allergic reaction (anaphylactic shock). Severe shock is usually treated with drugs called catecholamines or vasopressin. Shock that does not respond to treatment with these drugs is resistant shock.
Ranibizumab for proliferative and non-proliferative diabetic retinopathy
Ranibizumab as intravitreal injections is in clinical development for the treatment of diabetic retinopathy in both its proliferative and non-proliferative forms. Diabetic retinopathy, is a disease that affect the retinas and other parts of the inner eye. It is a chronic progressive condition of the retinal blood vessels due to prolonged raised blood glucose. When new blood vessels and scar tissue form on the retina causing bleeding within the eye and loss of vision, it is called proliferative diabetic retinopathy.
Pegargiminase in addition to pemetrexed and cisplatin for advanced malignant pleural mesothelioma – first line
Pegargiminase is a medicinal product currently being developed to be given in addition to pemetrexed and cisplatin for the treatment of advanced malignant pleural mesothelioma. Malignant pleural mesothelioma is rare a type of cancer that affects the outer linings of the lungs and the internal chest wall. Mesothelioma is often diagnosed at an advanced stage. Malignant pleural mesotheliomas are most often surgically unresectable, and they respond poorly to current chemotherapy and radiation therapy.
Pembrolizumab (Keytruda) for relapsed/refractory classical Hodgkin lymphoma
Pembrolizumab, given by intravenous infusion, acts by binding to a protein called antiprogrammed death‐ligand 1 (PD‐L1) that is found on the cancer cells or immune cells trying
to attack cancer cells. Binding to this protein can lead to the activation of the body’s immune system to fight tumour cells. In cHL, pembrolizumab has promising results. If licensed it will
provide a treatment option for cHL patients who have failed ASCT or are not eligible for it.
Leuprorelin acetate for advanced breast cancer in pre or perimenopausal women – adjuvant
Leuprorelin is a gonadotropin-releasing hormone (GnRH) analogue, when given chronically will block the production of oestrogen and progesterone hormones from the ovaries. A similar drug currently commonly used is goserelin, which is given as an injection every 4 weeks. If licensed, leuprorelin acetate will offer an additional adjuvant treatment option for pre and perimenopausal women with advanced breast cancer that is suitable for hormonal manipulation.