Upadacitinib for non-radiographic axial spondyloarthritis
Upadacitinib is in clinical development for the treatment of adults with non-radiographic axial spondyloarthritis (nr-axSpA). This condition affects patients predominantly in the spine and other areas of the body and is caused by inflammation. The inflammation can lead to back pain, fatigue and in serious cases to severe disability as the bones of the spine fuse into a …
Budesonide for Ulcerative Proctitis
Budesonide is in clinical development for ulcerative proctitis. Ulcerative proctitis is a mild form of ulcerative colitis, a chronic inflammatory bowel disease (IBD) consisting of small ulcers that develop on the colon’s lining, which can cause rectal bleeding and recurring diarrhoea. About a quarter of the patients that are diagnosed with ulcerative colitis may also …
Tisagenlecleucel for relapsed or refractory follicular lymphoma
Tisagenlecleucel is in clinical development for the treatment of relapsed or refractory follicular lymphoma. Follicular lymphoma is the most common type of slow-growing lymphoma that develops in B-cells (a type of immune cell). Relapsed refers to a disease that grows again after a period of remission while refractory refers to a disease that does not …
Beremagene geperpavec for dystrophic epidermolysis bullosa
Dystrophic epidermolysis bullosa (DEB) is caused by a mutation in a collagen gene that controls skin strength and stability. This mutation can be inherited from one or both parents, which determines the severity of the condition (those who inherit the mutation from both parents have more severe symptoms). DEB symptoms develop from birth or early …
Sparsentan for treating Focal Segmental Glomerulosclerosis
Sparsentan is in clinical development for the treatment of patients with primary and genetic focal segmental glomerulosclerosis (FSGS). FSGS is a rare kidney disease characterised by dysfunction in the part of the kidney that filters blood (the glomeruli) and causes serious scarring that leads to permanent kidney damage and even kidney failure. When glomeruli become …
Capmatinib for advanced non-small cell lung cancer
Capmatinib is in clinical development for the treatment of advanced non-small cell lung cancer (NSCLC). NSCLC makes up the majority of lung cancers in the UK and at the metastatic stage (stage IV), the disease has already spread from the lungs to other sites. Symptoms of lung cancer include a persistent cough, shortness of breath, …
Crizotinib for paediatric ALK-positive anaplastic large cell lymphoma or inflammatory myofibroblastic tumour
Anaplastic large cell lymphoma (ALCL) and inflammatory myofibroblastic tumour (IMT) are both rare conditions that usually affect children and young adults. ALCL is a type of cancer that occurs when T-cells, which contribute to the body’s immune system, become abnormal. IMTs are most commonly non-cancerous (benign), however in some cases the tumour can become cancerous (malignant). Both conditions can be ALK-positive (ALK+), this means that the tumour cells have a mutation in anaplastic lymphoma kinase (ALK) resulting in uncontrolled cell replication. These conditions may not be able to be treated through surgical removal of the tumour (unresectable); they may also come back after treatment (relapse) or be resistant to current treatment options (refractory).
Pembrolizumab in combination with lenvatinib for advanced melanoma – first line
Pembrolizumab is an immunotherapy, meaning it stimulates the body’s immune system by triggering T-cells (a type of white blood cells) to find and kill cancer cells. Lenvatinib is a targeted therapy drug that inhibits cancer growth by preventing the formation of new blood vessels. These drugs modulate different aspects of tumour biology, combining them, may result in improved efficacy and help overcome resistance to immunotherapy. Pembrolizumab in combination with lenvatinib (pembrolizumab administered intravenously and lenvatinib administered orally) would offer an additional first-line treatment option for previously untreated and unresectable advanced melanoma.