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.
Obeticholic acid for non-alcoholic steatohepatitis and fibrosis
Obeticholic acid as a tablet is in clinical development for the treatment of advanced liver fibrosis due to non-alcoholic steatohepatitis (NASH). Non-alcoholic fatty liver disease (NAFLD) is the general term for conditions in which an excessive amount of fat is stored in the liver, but where this excess is not caused by heavy alcohol use. A build-up of fat alone is called simple fatty liver (steatosis). NASH occurs when the presence of fat leads to liver cell damage and inflammation (hepatitis). Over time, liver cell damage and inflammation due to NASH can cause scarring (fibrosis) of the liver which may increase until advanced. Once there is advanced fibrosis due to NASH, there is a higher risk of progression to cirrhosis and its complications such as liver failure, liver cancer, and death. Although simple fatty liver, NASH, and advanced fibrosis due to NASH are distinct types of NAFLD in terms of the risk to health they pose, they are considered stages of the same disease through which people will progress if lifestyle interventions are not effective.
Ustekinumab for Moderately to Severely Active Ulcerative Colitis
Ustekinumab is a drug which is given as an injection and works by blocking the molecules that are involved in the inflammation which occurs in UC. Ustekinumab is already licenced for use in people with Crohn’s disease, which is another type of IBD. If ustekinumab is licenced this would provide another treatment option for people with moderate to severe active UC.
Selonsertib for Non-alcoholic Steatohepatitis (NASH)
Selonsertib is an investigational oral small molecule inhibitor of ASK1, a protein that mediates inflammation, apoptosis (cell death) and fibrosis in settings of oxidative stress. Oxidative stress can be increased in many pathological conditions including liver diseases such as NASH. NASH currently has no effective treatment apart from lifestyle interventions. If licensed, selonsertib will offer a new treatment option for NASH as no effective pharmacological therapies currently exist.
Timolumab for Primary Sclerosing Cholangitis
Timolumab is currently being developed to treat PSC. Timolumab is given by injection into the vein which blocks a molecule called VAP-1 from working. VAP-1 helps immune cells enter areas of inflammation (such as the bile ducts in PSC) where they can further contribute to inflammation. By blocking this process, timolumab has the potential to prevent some of the inflammation in PSC and slow the progress of the disease.
Tenapanor for Irritable Bowel Syndrome with Constipation
Irritable bowel syndrome with constipation (IBS-C) is a common condition that affects the digestive system (the gut). Symptoms may include stomach cramps, bloating and constipation. The exact cause is unknown, and IBS-C is often a lifelong condition. While there is no cure, dietary changes and the use of medication can often help control symptoms. IBS-C …
Volixibat for non-alcoholic steatohepatitis (NASH)
Currently, there are no approved therapies for the treatment of NASH, but doctors recommend dietary changes and exercise to prevent or slow disease progression. Volixibat is a new experimental once-daily oral tablet that may improve NASH by targeting and blocking a protein (apical sodium-dependent bile acid transporter) found in the liver. This is thought to …
A4250 for progressive familial intrahepatic cholestasis
Progressive familial intrahepatic cholestasis (PFIC) is a rare, inherited condition that usually begins in infancy. The condition affects the liver, hindering or stopping the flow of bile from the liver. Bile flow is needed for fats, nutrients and vitamins to be absorbed into the body, and also to help the body get rid of toxins. …