Odevixibat is currently in clinical development for the treatment of cholestatic liver disease in Alagille syndrome (ALGS) patients. ALGS is an inherited developmental disorder, caused by a mutation in the JAGGED1 gene or in the NOTCH2 gene. The mutation causes problems with early embryonic development leading to abnormalities in various parts of the body. ALGS […]
ASTX727 is in clinical development for patients with de novo or secondary acute myeloid leukaemia (AML), who are not candidates for standard induction chemotherapy. De novo AML is where there has been no past medical history of the condition or exposure to the drugs which commonly treat it, secondary AML is where patients have had […]
Natalizumab biosimilar is being developed as a biosimilar medicine to the approved monoclonal antibody natalizumab and is proposed to be used to treat adult patients relapsing remitting multiple sclerosis (RRMS). Multiple sclerosis is an autoimmune condition where the body’s immune system attacks the brain and nerves. RRMS is characterised by attacks of new or increasing […]
REGN5458 is in clinical development for the treatment of adults with multiple myeloma (MM) whose disease has returned following treatment. MM is a form of cancer that occurs in immune cells found in bone marrow. The disease occurs due to uncontrolled duplication of these immune cells, known as plasma cells. Symptoms of MM can include […]
Empagliflozin is in clinical development for the treatment of adult patients with chronic kidney disease (CKD). In CKD, the kidneys do not function as well as they should. This leads to the leakage of blood and protein into the urine. CKD can be developed due to age, but the main risk factors are diabetes, hypertension, […]
Fordadistrogene movaparvovec is a medicinal product in clinical development for the treatment of ambulatory boys greater than or equal to 4 years of age to 7 years with a confirmed genetic diagnosis of Duchenne muscular dystrophy (DMD). DMD is a severe and rare progressive neuromuscular disorder caused by a gene mutation (change). DMD is caused […]
Bimekizumab is in clinical development for the treatment of Hidradenitis Suppurativa (HS) in adults. HS is a painful, long-term skin condition that causes abscesses and scarring on the skin. The exact cause of hidradenitis suppurativa is unknown, but it occurs near hair follicles where there are sweat glands, usually around the groin, bottom, breasts and […]
Tislelizumab is being developed for the first-line treatment of unresectable hepatocellular carcinoma (HCC). HCC is the most common type of liver cancer and the leading cause of cancer related death worldwide. This type of cancer develops from the main liver cells, called hepatocytes. Liver cancer is more common in people who have long‐term damage to […]
Leniolisib is in clinical development for patients with Activated Phosphoinositide 3-kinase Delta Syndrome (APDS). APDS is an inherited disorder where the patient is unable to fight infections because the immune system (the body's natural defences) does not work properly. APDS is caused by defects in the genes that control the production of a protein called […]
Glucarpidase (Voraxaze), previously known as carboxypeptidase G2, is a carboxypeptidase G2 enzyme that hydrolyses the terminal glutamate residue from methotrexate.2 Glucarpidase hydrolyses methotrexate rapidly to the inactive metabolites DAMPA (4-[[2,4-diamino-6-(pteridinyl)methyl]-methylamino]-benzoic acid) and glutamate in patients with methotrexate-induced nephrotoxicity and delayed methotrexate excretion.3 It has been shown that the conversion of methotrexate to DAMPA relieves stress […]
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