Budesonide granules for induction of remission in lymphocytic colitis – first line

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.

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 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 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.