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This search function provides links to outputs produced by NIHR Innovation Observatory. These are briefing notes or reports on new or repurposed technologies. This search will not return all technologies currently in development as these outputs are produced as required for our stakeholders.

Innovation Observatory > Drugs

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Drugs

January 2021

Belzutifan for von Hippel-Lindau disease-associated clear cell renal cell carcinoma – first line

Belzutifan is in clinical development for the treatment of von Hippel-Lindau (VHL) disease-associated clear cell renal cell carcinoma (ccRCC). VHL is a hereditary disease that results in the body producing an increased amount of the protein hypoxia inducible factor (HIF) – 2α. This leads to an increased risk of tumours developing in various parts of the body including the kidney. Renal cell carcinoma (RCC) is the most common type of kidney cancer. ccRCC is named as such because when this type of tumour is viewed under a microscope the cells appear clear. Currently patients with VHL disease have limited treatment options which focus on surgically removing tumours once they have developed.

Drugs

January 2021

Pembrolizumab for recurrent or metastatic nasopharyngeal cancer

Pembrolizumab is an immunotherapy drug administered by intravenous (IV) infusion. It works by improving the activity of white blood cells (T-cells) by blocking a protein, PD-L1, thereby increasing the ability of the immune system to kill cancer cells. If licenced, pembrolizumab could provide an additional treatment option for patients with platinum pre-treated recurrent or metastatic nasopharyngeal cancer.

Drugs

January 2021

Phelinun for reduced intensity conditioning treatment prior to allogeneic haematopoietic stem cell transplantation

Phelinun is in development as a conditioning treatment prior to allogenic haematopoietic stem cell transplantation (HSCT) for various haematological diseases in children and adults. HSCT involves infusing stem cells from a donor into a patient whose bone marrow is damaged or defective. Conditioning treatment prior to HSCT is required in order to kill cancer cells, remove existing bone marrow from the patient and suppress the immune system, to reduce the risk of rejection from the transplant. Conditioning treatment is essential for a successful transplant but there are major associated side effects, including increased mortality. Therefore, there is a need for less toxic conditioning treatments.

Drugs

January 2021

Mosunetuzumab for relapsed or refractory B-cell follicular lymphoma – third-line and greater

Mosunetuzumab is in clinical development for the treatment of patients with relapsed or refractory B-cell follicular lymphoma (FL). FL is a type of slow growing blood cancer that affects white blood cells. In FL, the affected white blood cells start to multiply in an abnormal way and begin to aggregate in certain parts of the body such as the lymph nodes. A recurrence of lymphoma after going into complete remission is known as a relapse. Lymphoma that is unresponsive to therapy it is called refractory lymphoma. The condition can become more difficult to treat if it is relapsed or refractory, as treatment options are limited.

Drugs

January 2021

Nivolumab in combination with ipilimumab for cisplatin-ineligible patients with untreated, unresectable or metastatic urothelial cancer– first line

Nivolumab in combination with ipilimumab is in development for cisplatin-ineligible patients with previously untreated, unresectable or metastatic urothelial cancer (UC). UC occurs on the lining of the renal pelvis, ureter, bladder and urethra, and other parts of the urinary system. Metastatic UC occurs when the cancer has spread to other parts of the body. Durable responses are rare with current standard of care treatments. Therefore, treatment approaches with longer-term disease control and extending to broader metastatic UC patient populations are needed.

Drugs

January 2021

Ranibizumab Biosimilar for Age-related Macular Degeneration

Neovascular age-related macular degeneration (nAMD), also known as wet age‐related macular degeneration (wet AMD), is a chronic eye disease characterised by the formation and proliferation of blood vessels underneath the retina (a layer of tissue in the back of the eye that senses light and sends images to the brain). nAMD is a leading cause of central sight loss and blindness.

Drugs

January 2021

Inolimomab for acute Graft versus Host Disease

Inolimomab is being developed for the treatment of grade II to IV steroid-refractory acute Graft versus Host Disease (aGvHD) in adult patients after allogeneic haematopoietic stem cell transplantation (aHSCT). GvHD is characterised as a frequent complication of bone marrow transplantation and involves a reaction between the donor cells and the recipient’s native tissues, leading to injury of the recipient’s tissues. GvHD occurs in acute and chronic form. The organs most commonly affected in aGvHD are the stomach and the intestines, the skin, and the liver. Up to 50% of aGvHD patients do not respond to initial steroid treatment and are left with few therapeutic options.

Drugs

January 2021

Trastuzumab deruxtecan for unresectable or metastatic HER2-low (HR+/-) breast cancer – after chemotherapy

Trastuzumab deruxtecan is in clinical development for the treatment of adults with HER2-low, hormone receptor (HR) positive or HR negative (HR+/-), unresectable and/or metastatic breast cancer (BC) who have previously been treated with chemotherapy. Metastatic BC (stage IV) is when the cancer has spread beyond the breast and nearby lymph nodes, whilst unresectable refers to cancer that cannot be treated by surgery. HER2 receptors help control how a healthy breast cell grows, divides, and repairs itself. In the case of IHC 0 and 1+ results or IHC+ with a negative ISH assay, BC is considered HER2-low (or HER2-negative). Breast tumours are tested for both oestrogen receptors (ER) and progesterone receptors (PR). About 74 % of all BCs are both HR-positive (HR+) and HER2-negative. In England, adults with stage 4 breast cancer, that were diagnosed between 2012-2016 and followed up to 2017, had a 66% survival rate one year after diagnosis.

Drugs

January 2021

Ravulizumab for paroxysmal nocturnal haemoglobinuria in paediatrics

Ravulizumab is in clinical development for the treatment of paediatric patients with paroxysmal nocturnal hemoglobinuria (PNH). PNH is a rare blood condition in which red blood cells are attacked by the body’s immune system. PNH is a chronic condition that is associated with complications that can be severely debilitating and life-threatening including abdominal pain, kidney problems, fatigue, shortness of breath, bleeding and blood clots, dysphagia, organ damage and premature mortality.

Drugs

January 2021

Durvalumab and bevacizumab in addition to transarterial chemoembolisation for locoregional hepatocellular carcinoma

Durvalumab and bevacizumab in addition to transarterial chemoembolisation (TACE) is in clinical development for treating patients with locoregional hepatocellular carcinoma (HCC). HCC is the most common type of primary liver cancer in adults and the most common cause of death in people with cirrhosis (scarring of the liver). It usually presents at an advanced stage and has a poor prognosis. The current standard of care can only slow the progression of the cancer and extend survival.

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