Olaparib in addition to abiraterone for metastatic castration-resistant prostate cancer – First line
Olaparib is administered orally in tablet form and can lead to cancer cell death by blocking DNA repair by an enzyme (protein) called PARP. By blocking PARP enzymes, the damaged DNA in cancer cells cannot be repaired, and the cells die. Abiraterone works by stopping the body making testosterone which subsequently stops the cancer growing. If licensed, this combination would provide a first-line treatment for men with mCRPC.
Ranibizumab port delivery system for the treatment of age-related macular degeneration
Ranibizumab is a type of antibody that is targeted against a particular protein. Ranibizumab has been designed to attach to and block a substance called vascular endothelial growth factor A (VEGF-A). VEGF-A is a protein that makes blood vessels grow and leak fluid and blood, damaging the macula (the central part of the retina). By blocking VEGF-A, ranibizumab reduces the growth of the blood vessels and controls the leakage and swelling. The port delivery system (PDS) will include a device which is permanently surgically implanted in the eye and filled with a special formulation of ranibizumab; this will reduce the amount of hospital visits required and reduce the burden of repeat intravitreal injections. If licensed, this technology will provide an additional treatment option for patients with nAMD.
Faricimab for neovascular age-related macular degeneration
Faricimab is an antibody given by intravitreal injection that binds to both VEGF-A and angiopoietin-2 which results in blood vessels becoming more stable, leaking less blood and fluid and reduced inflammation. Faricimab has been shown in clinical trials to have an extended durability compared to other anti-VEGF agents so fewer injections will be required. If licensed, faricimab will offer an additional treatment option for patients with neovascular AMD.
Brolucizumab for visual impairment due to diabetic macular oedema
Brolucizumab is in clinical development for the treatment of visual impairment due to diabetic macular oedema (DMO). DMO is a condition affecting the retina, the nerve layer at the back of the eye. The central part of the retina known as the macula, is responsible for fine detail vision, both for near (reading) and for distance. In patients with DMO, the fine meshwork of blood vessels supplying nutrients and oxygen to the macula become damaged and leaky due to the high levels of glucose in the bloodstream in some patients with diabetes. If left untreated, the leakage will potentially permanently damage the retinal nerve cells and eventually produce scarring, which can be irreversible.
rAAVrh74.MHCK7.micro-dystrophin for Duchenne muscular dystrophy
rAAVrh74.MHCK7.micro-dystrophin is a medicinal product in clinical development for the treatment of children aged 3 months to 7 years with Duchenne muscular dystrophy (DMD). DMD is a rare progressive neuromuscular disorder caused by a gene mutation (change). DMD is caused by an absence of dystrophin, a protein that helps keep muscle cells intact. It affects …
Tideglusib for congenital myotonic dystrophy
Tideglusib is being development for the treatment of congenital myotonic dystrophy type 1 (CMD1). CMD1 is a form of myotonic dystrophy type 1 (DM1), a rare, genetically determined neuromuscular disorder. CMD1 begins at or around the time of birth and is characterised by severe muscle weakness, cognitive impairment and other developmental abnormalities. The condition usually occurs when the mother already has DM1 and then it is passed on to her child in a more severe form. CMD1 is typically associated with significant medical morbidity and early death. No specific treatment is currently on offer, although supportive care to manage symptoms is available.
Polatuzumab vedotin in addition to R-CHP for diffuse large B-cell lymphoma – first line
Polatuzumab vedotin is a first-in-class drug specifically developed for the treatment of cancers that affect the blood and lymph system. It is an antibody that binds to CD79b, which is a protein on the surface of cancerous B-cells. It is administered as an intravenous infusion, absorbed by the cancer cells and the chemotherapy agent linked to the antibody releases inside the cancer cells, stops them from dividing and kills them. If licenced polatuzumab vedotin in addition to R-CHP would offer an additional treatment option for patients with untreated DLBCL.
Leriglitazone for X-linked andrenoleukodystrophy
Leriglitazone is expected to work in patients with ALD by activating receptors called ‘PPAR gamma’, found in the mitochondria, which are components within cells that generate energy. Leriglitazone will improve the ALD pathogenic cascade by improving mitochondrial dysfunction, reducing oxidative stress and neuroinflammation, promoting demyelination and halting axonal degeneration. Hence, leriglitazone is expected to protect cells from damage and slow the progression of the disease. If licensed, leriglitazone would offer the first drug treatment option for adult ALD patients with AMN who currently have no effective therapies available.
Pegunigalsidase alfa for Fabry disease – first-line
Pegunigalsidase alfa is produced by a method known as ‘recombinant DNA technology’: it is made by cells into which a gene (DNA) has been introduced, which makes them able to produce the enzyme. The replacement enzyme has also been modified to reduce the rate at which it is removed from the body, allowing it to act for longer. Pegunigalsidase alfa was designed to increase amount of medicine in the blood and reduce the ability of a substance to provoke an immune response, thereby enhancing efficacy compared with available products. If licensed, pegunigalsidase alfa would offer an additional first-line, long -term ERT options for adult and paediatric patients (>3 yr) diagnosed with FD.
Faricimab for diabetic macular oedema
Faricimab is in clinical development for adults with diabetic macular oedema (DMO). DMO is a type of eye disease where blood vessels leak fluid into the retina. Vision loss occurs when the fluid reaches the macula (the centre of the retina that provides sharp vision) and builds up, causing swelling. Over time, DMO can cause central vision to become blurred, eventually these changes become permanent. Current treatments use steroid implants, or an injection of anti-vascular endothelial growth factor-A (VEGF-A), in the eye to stop the condition from worsening.