Ravulizumab for treating generalised myasthenia gravis
Ravulizumab is currently in clinical development for the treatment of adults with generalised myasthenia gravis (gMG). gMG is a long-term autoimmune disorder where the body’s own immune system mistakenly attacks healthy cells in the neuromuscular junction (NMJ). The NMJ is a region where nerve cells transmit signals to muscle cells to result in muscle contraction. …
Betula verrucosa for tree pollen allergy-induced moderate to severe rhinoconjunctivitis
Betula verrucosa is in clinical development for the treatment of adults with confirmed tree pollen allergy-induced moderate to severe rhinoconjunctivitis. Allergic rhinoconjunctivitis is an allergic disorder of the nose and resulting in a chronic, mostly eosinophilic, inflammation of the nasal mucosa and conjunctiva. When the conjunctiva is also involved, the term rhinoconjunctivitis is more accurate. …
Belimumab in combination with rituximab for systemic lupus erythematosus (SLE)
Belimumab in combination with a cycle of rituximab is in clinical development for adults with Systemic Lupus Erythematosus (SLE). SLE is an autoimmune disease where the body’s immune system attacks its own tissues and organs. Patients will often have periods where their symptoms flare-up and periods where their symptoms settle down. Mild SLE can cause …
Mavorixafor for WHIM syndrome
Mavorixafor is an oral drug expected to reduce the activity of the CXCR4 receptor and allow neutrophils to be released from the bone marrow into the blood stream, thereby helping the body to fight infections. If licenced, mavorixafor would provide the first targeted treatment option for WHIM syndrome in patients aged 12 years and above, whose care is currently limited to the treatment of the different symptoms of WHIM syndrome.
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.