Luspatercept for the treatment of adults with non-transfusion dependent beta (β)-thalassaemia
Luspatercept is under clinical development for the treatment of adult patients with beta-thalassaemia who don’t regularly require blood transfusion. Thalassaemia is a commonly inherited blood disorder resulting from an abnormality in one of the genes that affects the production of haemoglobin, a protein in red blood cells that carries oxygen throughout the body . Beta-thalassaemia is a subtype caused by a specific gene mutation. People with thalassaemia produce either little or no normal haemoglobin. Current treatment options for beta-thalassaemia are limited to blood transfusions with its associated risks and complications.
OTL-103 for Wiskott-Aldrich syndrome
OTL-103 is administered intravenously. It is made up of immature bone marrow cells (called CD34+ cells) taken from the patient. It works by correcting cells using a modified virus that contains the correct gene for the WAS protein. When these corrected cells are transplanted back into the patient, they populate the bone marrow and produce healthy platelets and immune cells that produce the WAS protein, thereby relieving the symptoms of the disease. If licensed, OTL-103 will provide a treatment option for patients with WAS.
Tecarfarin for preventing venous thromboembolism
Tecarfarin, a vitamin K reductase, is given by oral administration and works by blocking the liver from using vitamin K to make clotting factors. Clotting factors work with blood cells called platelets that trigger the clotting process to form a blood clot. Stopping the activation of these vitamin K dependent clotting factors means the blood takes much longer to clot so there is a decreased risk of VTE. Tecarfarin works in the same way as warfarin, which is the current best treatment option for preventing VTE, but is expected to be safer and deliver more predictable clotting. If licensed, tecarfarin would offer an additional treatment option for preventing VTE in patients who require long-term anticoagulation therapy.
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.
Burosumab for x-linked hypophosphataemia in adults
Burosumab, administered by subcutaneous injection, is a monoclonal antibody (a type of protein) designed to recognise and attach to the FGF23 protein. By attaching to the FGF23 protein, the medicine blocks its activity, allowing the kidneys to reabsorb phosphate into the bloodstream and restore normal levels of phosphate in the blood. In studies of adults with XLH, burosumab is associated with normalisation of serum phosphorus, restoration of bone quality and improvements in pain, stiffness, fatigue, as well as healing of fractures compared to placebo.
Potassium citrate and potassium bicarbonate for distal renal tubular acidosis – first line
ADV7103 is an innovative product with a prolonged-release formulation, designed to maintain sustained release over a twelve-hour period for dRTA treatment. As the combination is alkaline (pH greater than 7) and contains potassium, it is expected to neutralise excess acid in the blood and restore levels of potassium. The product was developed as a multi particulate formulation in 2mm granules that contains two active pharmaceutical ingredients. Bicarbonate + potassium citrate is tasteless and easy to administer, in small-size format that offer flexible, personalized dosing which makes it easier to take for patients of all ages. The ability of ADV7103 to normalize biological disorders caused by dRTA throughout the course of treatment has been shown in a Phase III extension study. If licensed, ADV7103 will offer a potentially curative treatment option for patients with dRTA, who currently have few approved therapies available.
Etranacogene dezaparvovec for treating moderately severe or severe haemophilia B
Etranacogene dezaparvovec is given to patients by intravenous injection. It works by using a harmless viral vector to insert a highly functional copy of the F9 gene. This means that the body can make more functional factor IX clotting protein so the blood does not take as long to clot following an injury and there is no spontaneous bleeding into the joints, muscles or brain. If licensed, etranacogene dezaparvovec will offer an additional treatment option for adult males with moderately severe or severe haemophilia B.
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.