Emapalumab for Primary Haemophagocytic Lymphohistiocytosis
Emapalumab is a type of drug called a monoclonal antibody, under development for the treatment of primary HLH in children. It acts by neutralising IFNγ activity produced by over‐stimulated cells of the immune system. It is administered as an intravenous infusion and may be used in addition to current treatment.
Voxelotor for Sickle Cell Disease
Voxelotor is as an oral, once-daily therapy that is being developed for treatment of SCD. It is designed to work by helping haemoglobin hold onto more oxygen as the red blood cells travel through the body.
Momelotinib for myelofibrosis
Myelofibrosis is a rare condition affecting the bone marrow and most people with this condition do not have any symptoms. Bone marrow is where blood cells are made. In myelofibrosis, scar tissue builds up inside the bone marrow and blood cells are not made properly. Occasionally the spleen (which functions as a blood filter) may …
Caplacizumab for thrombotic thrombocytopenic purpura
Caplacizumab is a drug that is being given for the treatment of thrombotic thrombocytopenic purpura. The drug is injected under the skin and given alongside the current treatment of plasma exchange.
Begelomab (Begesand) for steroid resistant acute graft versus host disease
Begelomab is a new drug for people whose first treatment for GvHD has failed. Begelomab is given in a drip (directly into a vein). Begelomab is currently being studied to see how well it works and whether they it is safe to use in people with GvHD. If begelomab is licensed for use in the …