Obeticholic acid for non-alcoholic steatohepatitis and fibrosis
Obeticholic acid is a modified form of a bile acid. It works by attaching to receptors in the liver and gut called farnesoid X receptors (FXRs) which control the production of bile. By attaching to these receptors, obeticholic acid reduces the production of bile in the liver, preventing it from building up and damaging the liver tissue. FXRs are also involved in the control of inflammation and fibrosis as well as the handling of fats and glucose in the liver which are important processes in the development of NASH. If licensed, obeticholic acid may offer the first pharmacological treatment option for patients with NASH who currently have few effective therapies available.
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. …
Seladelpar for primary biliary cirrhosis – second or subsequent line
Primary biliary cirrhosis (PBC), is a long-term liver disease in which the pipes (ducts) that transports bile in the liver become damaged. PBC causes the build-up of bile in the liver and eventually may lead to scarring. Nine out of ten people with PBC are women. The exact cause of PBC is not known, although …
Cenicriviroc for non-alcoholic steatohepatitis (NASH)
Cenicriviroc is a new experimental once-daily oral tablet that may improve liver fibrosis in patients with NASH by targeting and blocking the immune and inflammatory pathways responsible for fibrosis. If licensed, cenicriviroc has the potential to establish itself as both a single-agent and as a cornerstone treatment for NASH.
Liprotamase (Sollpura) for exocrine pancreatic insufficiency due to cystic fibrosis
Liprotamase is a new drug for the treatment of exocrine pancreatic insufficiency (EPI), a condition where the pancreas does not produce enough enzymes for digesting fat, carbohydrates and proteins. EPI is common in patients who have cystic fibrosis (CF), which is the most common inherited disease in the UK.Liprotamase works by replacing the lacking pancreatic …