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Innovation Observatory > Reports > Haematology and Blood Products

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Drugs

September 2018

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.

Drugs

August 2018

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.

Drugs

August 2018

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.

Drugs

June 2018

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.

Drugs

June 2018

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.

Drugs

February 2018

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.

Drugs

Drugs

November 2017

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.

Drugs

November 2017

Crizanlizumab (formerly SelG1) for Sickle Cell Disease

Crizanlizumab is a medicine under development for the preventative treatment of SCD painful crisis.

Drugs

August 2017

Darbepoetin alfa (Aranesp) for treatment of anaemia in adults with low or intermediate-1-risk myelodysplastic syndromes

Myelodysplastic syndromes (MDS) are a group of blood disorders, in which the bone marrow does not produce enough healthy red blood cells, white blood cells and/or platelets. Faulty cells often die earlier than normal cells and the body destroys some abnormal cells, leaving the patient with low blood counts. This can result in anaemia (not …

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