Risankiziumab for moderate to severe crohn’s disease
Risankizumab is in development as a treatment option for moderate to severe Crohn’s disease (CD). CD is a type of inflammatory bowel disease which can affect any part of the digestive system. CD causes inflammation and ulceration, which affects food digestion, nutrient absorption, and waste elimination. Symptoms include abdominal pain, diarrhoea, weight loss and fatigue. …
Mirikizumab for the treatment of moderately to severely active ulcerative colitis
Mirikizumab is in clinical development for the treatment of adult patients with moderately to severely active ulcerative colitis (UC). UC is a long-term condition where the colon and rectum (parts of the bowel), become inflamed. Small ulcers can develop on the colon’s lining, which can cause rectal bleeding and recurring diarrhoea. The symptoms of ulcerative …
Maralixibat for cholestatic liver disease in patients with Alagille syndrome
Maralixibat is an orally administered drug that blocks certain channels called ileal bile acid transporters through which the bile acids leave the intestine to reach the blood vessels that carry them back to the liver. By blocking these channels, maralixibat is expected to help reduce the amount of toxic bile acids in the liver, thereby reducing the itching and other quality of live measures in patients with Alagille syndrome. If licensed, maralixibat will provide a treatment option for patients with Alagille syndrome, a disease of unmet clinical need.
Budesonide granules for induction of remission in lymphocytic colitis – first line
Budesonide is a corticosteroid that exerts anti-inflammatory effects in the gastrointestinal tract. The treatment works by binding with high affinity to intracellular glucocorticoid receptors. In one phase III clinical trial, budesonide was shown to be significantly effective in inducing clinical remission in lymphocytic colitis patients in comparison to a placebo. If licensed, budesonide will offer a treatment option for patients with lymphocytic colitis.
Dapagliflozin for chronic kidney disease
Dapagliflozin blocks the action of a protein in the kidneys called sodium-glucose cotransporter 2 (SGLT2). As blood is filtered by the kidneys, SGLT2 stops glucose in the bloodstream from being passed out into the urine. By blocking the action of SGLT2, dapagliflozin causes the kidney to pass out more glucose in the urine, thereby reducing the levels of glucose in the blood. SGLT2 inhibitors have exhibited beneficial effects on kidney function in those with diabetes and more recently has shown improvements in kidney function and all-cause mortality in those with CKD without a diabetes. If licensed, dapagliflozin may provide a treatment option specifically for people with CKD.
Etrolizumab for treating moderately to severely active ulcerative colitis in adults
Etrolizumab is a new monoclonal antibody (an immune protein) delivered by subcutaneous injection. The treatment works by targeting molecules called integrins to control the immune response and prevent the accumulation of immune molecules, which cause inflammation in individuals with a form of ulcerative colitis where inflammation is not mediated by a signalling protein called tumour necrosis factors (TNF) alpha (‘non-TNF-α’) and who are therefore intolerant to TNF blockers. This represents a new target group as current therapies focus mainly on anti-TNF inflammation. In one study, etrolizumab showed a greater reduction of intestinal lymphocyte infiltration in comparison to standard treatment.
Maralixibat for Progressive Familial Intrahepatic Cholestasis Type 2
Maralixibat is an orally administered drug that is expected to reduce the level of bile acids. It is expected to interfere with the process by which most bile acids in the intestines are recovered and delivered back to the liver through the blood, thereby reducing the liver damage and itching seen in patients with PFIC2. If licensed, maralixibat will provide the first disease-modifying treatment option for patients with PFIC2, a disease of unmet clinical need.
Filgotinib for moderately to severely active ulcerative colitis
Filgotinib is in clinical development for the treatment of patients with moderately to severely active ulcerative colitis. Ulcerative colitis is a type of inflammatory bowel disease that causes inflammation and ulcers in the bowel and rectum which can cause diarrhoea, abdominal pain and faecal urgency or incontinence. The symptoms of ulcerative colitis often follow a pattern of relapse and remission where they have periods of none or mild symptoms followed by periods of increased symptoms flare-up (‘active’). There are no curative therapies for ulcerative colitis and current treatment aims to relieve symptoms during a flare-up and maintain remission by preventing symptoms from returning.
Imlifidase for kidney transplantation in highly sensitised patients with chronic kidney disease
Imlifidase is in clinical development for enabling kidney transplantation in highly sensitised patients with chronic kidney disease (CKD). CKD is a long-term irreversible condition where the kidneys do not work as well as they should. Kidney transplantation is considered to be treatment of choice for patients with end stage kidney disease. Many patients on the waiting list for organ transplantation carry antibodies to human leukocyte antigen (HLA), which is known as being ‘sensitised.’ Patients who are highly sensitised may find it difficult getting a donor and may not be able to receive a transplant due to increased risk of kidney rejection.
OTL-200 for Metachromatic Leukodystrophy
OTL-200 is a gene therapy that involves extraction of certain stem cells from a patient’s bone marrow or blood. These stem cells are genetically modified and then returned to the patient by intravenous infusion to deliver the corrected version of the gene to the cells in charge of creating key proteins. The corrected cells then produce the protein that was missing or defective prior to treatment, aiming to halt disease progression or modify its natural course. If licensed, OTL-200 will offer a potentially curative treatment option for patients with MLD, who currently have no effective therapies available.