Upadacitinib for Ankylosing Spondylitis
Upadacitinib acts by selectively blocking a protein called Janus-Associated Kinase 1 (JAK1 and JAK1/3). JAKs contribute to the processes within the cell to produce an immune or inflammatory response. There is an emerging body of evidence establishing that JAK dependent enzymes are major contributors to the progression of immunemediated diseases such as AS and that blocking such enzymes can be beneficial. Upadacitinib is taken orally and if licensed, it will offer an additional treatment option for patients with active AS.
Tofacitinib for polyarticular juvenile idiopathic arthritis
Tofacitinib works by blocking the action of enzymes known as Janus kinases. These enzymes play an important role in the inflammatory process. By blocking the enzymes’ action, tofacitinib helps reduce the inflammation and other symptoms of these diseases. Tofacitinib is an oral alternative to the available biological medicines for children with JIA, most of which are administered by injection. If licensed, tofacitinib may offer an additional treatment option for polyarticular JIA with the potential advantage of improving the quality of life of children due to its oral formulation.
NOVOCART 3D for articular cartilage defects of the knee
NOVOCART 3D Autologous Chondrocyte Transplantation System is a biologic-device combination product composed of autologous (the patient’s own) chondrocytes (cartilage producing and maintaining cells) seeded on a bioresorbable three-dimensional collagen scaffold, which is secured directly into the defect. The use of NOVOCART 3D involves two separate surgeries. In the first surgery, a small amount of knee cartilage is removed from the patient’s affected knee and sent to the company to be processed for re-implantation. In a second surgery, the damaged area of the knee cartilage is cleaned and the sponge-like scaffold containing the autologous chondrocytes is implanted. If licensed, NOVOCART 3D will offer an additional treatment option for repair of articular cartilage defects of the knee.
Ixekizumab for axial spondyloarthritis
Ixekizumab is an engineered antibody designed to bind and obstruct the pro-inflammatory interleukin-17A (IL-17A) signalling molecule. It has been suggested that IL-17 may be a crucial mediator of inflammation in the pathway that leads to the development and progression of axial spondyloarthritis. By blocking this pathway, ixekizumab may help prevent joint inflammation, bone erosion, and bone fusion in a patient population where few alternative therapies exist in instances of previous treatment failure.
Tizanidine (Intranasal spray) for Acute Low Back Pain
Tizanidine as an intranasal spray is being developed as a new treatment option for patients with acute low back pain. It works by blocking pain sensations that are sent to the brain, temporarily relaxing muscle tone. Tizanidine is already available in the tablet form as a short-acting muscle relaxer used to relieve the stiffness and restriction of muscles due to injuries or diseases of the spinal cord. If licensed, tizanidine prepared for intranasal administration will offer an alternative to the oral administration for patients with acute low back pain when short term dosing and a fast effect is desired.
Mexiletine for the Symptomatic Treatment of Myotonic Disorders – First Line
Mexiletine is under registration in Europe for the symptomatic treatment of myotonic disorders. It is administered as an oral capsule and it exerts its action by reducing the rate of contraction in the heart and other muscles. Currently it is used unlicensed in the UK, meaning that it is not currently approved but some healthcare providers consider it to be potentially beneficial based on research or professional experience. There are currently no licensed treatment options available to treat symptoms of myotonia, therefore, if licensed, mexiletine will offer access to an approved treatment option.
Anakinra for Still’s disease
Anakinra is a drug which blocks a particular inflammatory protein which causes inflammation and is thought to play a role in the activity of Still’s disease. Anakinra is administered by injection under the skin (subcutaneously) and can be used in new onset patients or those with continued disease activity. As of February 2018, anakinra has been licenced for the treatment of adults, adolescents, children and infants aged 8 months and older patients with Still’s disease who have not responded to treatment with NSAIDs or steroids, and therefore require further treatment to control their disease.
Forigerimod plus Standard of Care for Systemic Lupus Erythematosus
Systemic lupus erythematosus (SLE) is a long-term condition causing inflammation to the joints, skin and other organs. Symptoms presented are usually very general, including fever, joint pain and skin rash but can progress to the most severe, e.g. kidney failure. SLE typically has patterns of flare-ups where the condition gets worse for a period of time. The disease is likely to be caused by a combination of genetic and lifestyle factors and most commonly affects middle-aged women and those of African-Caribbean ethnicity.
Tocilizumab (RoActemra) for Adults with Systemic Sclerosis – Second Line
There is currently no cure for systemic sclerosis and treatment options focus on the management of symptoms. Tocilizumab is intended for the treatment of systemic sclerosis in adults. It is currently in development and early research indicates that it is able to target the key mechanisms thought to be responsible for the disease. If licensed, …