Belimumab is in clinical development for the treatment of adults with active lupus nephritis (LN) who are uncontrolled on current standard of care. LN is a complication affecting kidney function brought on by systemic lupus erythematosus (SLE), a chronic autoimmune, inflammatory disease that affects different organs. Around a third of people suffering with SLE will develop LN. The kidney damage seen in LN occurs due to a person’s immune cells (B Cells) attacking their own kidneys which causes inflammation and affects overall kidney function. Current treatment involves suppression of the immune system and management of symptoms, but it is not always effective. If left untreated, LN can lead to kidney failure which requires dialysis or a kidney transplant.
Belimumab is a monoclonal antibody which binds to a soluble B cell survival factor known as BLyS, thereby stopping the growth and activity of B cells. It is given by intravenous infusion and is intended to be added on to current standard of care therapies. Belimumab is currently approved as an add-on therapy for adult patients with SLE in the EU/UK and if licensed for LN, will offer a new targeted therapy that is able to treat patients who cannot tolerate the side effects related to the current standard of care.
Avapritinib is in clinical development for the treatment of advanced systemic mastocytosis (SM) in adults. SM is a condition where mast cells grow uncontrollably and accumulate in body organs/tissues such as the skin, internal organs, lymph nodes and bones. Mast cells are immune cells that release inflammatory mediators that are important in the body’s allergic responses. When mast cells are present in large numbers there is a high release of these mediators leading to symptoms such as itching, fever, abdominal pain, nausea and vomiting. In advanced SM, mast cells collect in such high quantities that they lead to organ damage and dysfunction, bone fractures and anaemia.