Ibrutinib for chronic Graft versus Host Disease
Ibrutinib belongs to class of drugs called Bruton’s Tyrosine Kinase (BTK) inhibitors that work against defective B lymphocytes, which are a type of white blood cells affected by these diseases. Earlier studies have demonstrated that ibrutinib works by blocking the BTK signalling pathway, resulting in reduced production of the defective blood cells in GvHD and some other types of blood cancers. Ibrutinib is available as tablets and taken orally. If licensed, ibrutinib will offer an additional first line treatment option for cGvHD which may improve patients’ quality of life by helping reduce the dose of steroids used and reduce the severity of GvHD symptoms.
Avapritinib for advanced systemic mastocytosis
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.
Mepolizumab for hypereosinophilic syndrome – add-on therapy
Mepolizumab is a medicinal product currently in development as an add-on for the treatment of hypereosinophilic syndrome (HES). HES is a rare group of inflammatory disorders characterised by an overproduction of eosinophils (a type of disease-fighting white blood cell). When eosinophils infiltrate certain tissues, they can cause inflammation and organ damage which, over time, can impact patients’ day-to-day ability to function. Although any organ system can be involved in HES, the heart, central nervous system, skin, and respiratory tract are the most commonly affected.
Ruxolitinib for chronic graft versus host disease (cGvHD)
Ruxolitinib is in clinical development for chronic graft versus host disease (cGvHD). After a donor stem cell transplant, the donor’s stem cells (the graft) may sometimes react against the host’s own cells. This is called GVHD. cGvHD may happen more than three months after transplant. It can develop from acute GVHD or happen on its …
Ruxolitinib for acute graft versus host disease (aGvHD)
Ruxolitinib is in clinical development for acute graft versus host disease (aGvHD). After a donor stem cell transplant, the donor’s stem cells (the graft) may sometimes react against the host’s own cells. This is called GVHD. aGVHD is most likely to happen in the first three months after transplant. The symptoms depend on which parts of the body are affected. It often causes an itchy skin rash. If the bowel, the stomach or the liver are affected, the patient may have sickness and diarrhoea. aGVHD is graded by how severe it is. It goes from grade 1, which is mild, to grade 4 which is very severe. Current standard treatment includes the use of steroids but this is often associated with significant side effects. Steroid resistance in GvHD may also develop which is difficult to treat and associated with a high mortality.
Belimumab for lupus nephritis
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.
BCX7353 for prevention of acute attacks of angioedema in hereditary angioedema
BCX7353 is an oral treatment that works by blocking the activity the specific pathway that becomes overactive in patients with angioedema. By blocking this pathway, BCX7353 is expected to reduce the number of angioedema attacks. Early studies of BCX7353 has shown that it helped to prevent swelling and inflammation in HAE. In addition, BCX7353 has the distinct advantage over current treatment of being administered orally, making it easier to use for routine prevention of HAE attacks.
Apremilast for oral ulcers in active Behçet’s disease
Apremilast works by suppressing the activity of inflammatory pathway caused by molecules such as interleukins and tumour necrosis factors. In turn this reduces inflammation. Currently, specific curative treatment options for Behçet’s disease are very limited and treatment relies on controlling symptoms and relieving pain. Apremilast has shown promise in targeting oral ulcers in patients with Behçet’s disease, and if licensed it could be an effective treatment option for this patient group.
Lenabasum for Diffuse Cutaneous Systemic Sclerosis
Lenabasum is being developed for diffuse cutaneous SS as a tablet to be taken twice per day. It works by binding to immune cells and triggers a process which reduces inflammation and the scaring and thickening of tissues which usually happens in SS. If licensed, lenabasum has the potential to reduce the scaring which happens in diffuse cutaneous SS and improve symptoms.