Valoctocogene Roxaparvovec for Severe Haemophilia A
Valoctocogene roxaparvovec is a gene therapy; it can modify the genes (functional units of heredity) of individuals with haemophilia A so that they can produce the clotting protein needed to allow the blood to clot. If licensed, valoctocogene roxaparvovec would be the first gene therapy for severe haemophilia A. Valoctocogene roxaparvovec administered as a single treatment would be sufficient to maintain normal levels of factor VIII in adult males with severe haemophilia A, and might reduce the need for regular factor VIII prophylaxis (preventative treatment).
Romiplostim for idiopathic thrombocytopenic purpura in adult patients who are refractory to other treatments
Romiplostim is a medicinal product that is being developed for the treatment of adult patients with idiopathic thrombocytopenic purpura (ITP) who are refractory to other treatments. ITP is the condition of having a low platelet count due to unknown cause. It is also known as immune thrombocytopenic purpura. Many people with ITP do not have symptoms, however people with very low platelet count can have symptoms such as pin prick rash, easy bruising, nosebleeds, gum bleeds, black mouth blisters, fatigue, and heavy periods. Most of the currently available treatments have significant side effects with some treatments leaving patients are at increased risk of infections.
Ropeginterferon alfa-2b for polycythaemia vera without symptomatic splenomegaly
Ropeginterferon alfa-2b for injection is under development for the treatment of polycythaemia vera (PV), a rare blood disease in which the body makes too many red blood cells. The extra red blood cells make the blood thicker than normal and as a result, blood clots can form more easily. These clots may block blood flow through arteries and veins, which can cause a heart attack or stroke. Thicker blood also does not flow as quickly and may prevent organs from getting enough oxygen. A mutation, or change, in a gene called JAK2 is the major cause of PV. This gene makes a protein that helps the body produce blood cells. PV develops slowly and may not cause symptoms for years. PV has no cure, but treatments can help control the disease and its complications.
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.
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.
Givosiran for acute hepatic porphyria (AHP)
Givosiran is made of a short, synthetic strand of genetic material called ‘small interfering RNA’ (siRNA) that has been designed to interfere with the production of an enzyme involved in an early step in making haem. By blocking this early step of haem production in patients with AHP, givosiran is expected to prevent the next steps which produce substances that accumulate in the body and cause the symptoms of the disease.
Ravulizumab for atypical haemolytic uraemic syndrome in adults and children – first line
Ravulizumab works by inhibiting a component in the complement system called C5. It is given intravenously and has the potential to increase patient’s quality of life and to decrease treatment burden due to its extended effect that enables every 8-week dosing. If licensed, ravulizumab will offer an additional first-line treatment option for adults and children with aHUS.
Luspatercept for adult patients with beta-thalassemia who require red blood cell transfusions
Luspatercept is a recombinant engineered protein designed to attach to certain proteins that slow down the maturation of red blood cells. This leads to the production of healthy red blood cells and increased haemoglobin levels, leading to improved symptoms in patients with beta-thalassemia intermedia and major. Luspatercept is a novel approach for treating anaemia, with potential to improve many patients’ lives by reducing or eliminating the need for frequent and lifelong blood transfusions.
L-glutamine for Sickle Cell Disease
The amino acid L-glutamine has been developed as an oral powder formulation to reduce the acute complication of sickle cell disease. It is thought L-glutamine works by reducing cell inflammation and promote the cellular uptake of oxygen. If licensed, L-glutamine oral powder may offer an additional therapy option for those with sickle cell disease who currently have few effective therapies available.
BPX‐501 to improve Immune Recovery and Graft versus Host Disease (GvHD) after Haploidentical Stem Cell Transplant
BPX‐501 is a medicinal product made of T‐cells, a type of white blood cell, extracted from a donor who is partly matched to a patient undergoing HSCT. These T‐cells are expected to help the patient to fight off viral infections while their immune system is being restored with the transplanted stem cells. However, the transplanted T‐cells can cause GvHD. BPX‐501 incorporates a safety mechanism where the T‐cells are genetically modified to include a ‘suicide gene’. If the patient develops GvHD, a medicine called rimiducid is given to switch on the suicide gene in the T‐cells. This causes the T cells to die, thus preventing worsening of the GvHD. BPX‐501 has the potential to reduce hospital admissions, infections and improve survival when used in combination with standard care.