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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 > Reports > Drugs

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Drugs

April 2020

Pembrolizumab inaddition to chemotherapy for persistent, recurrent or metastatic cervical cancer – first line

Pembrolizumab is an immunomodulatory medicinal product, meaning that it helps the immune system to recognise and attack cancer cells. It is administered by intravenous infusion and is currently licensed in the UK for melanoma, non-small cell lung cancer and urothelial cancer – amongst others. If licensed, pembrolizumab, in addition to chemotherapy, would offer an alternative treatment for those with advanced, recurrent or metastatic cervical cancer.

Drugs

April 2020

Valoctocogene Roxaparvovec for Severe Haemophilia A

Valoctocogene roxaparvovec is a gene therapy; it can modify the genes (functional units of heredity) of individuals with haemophilia A so that they can produce the clotting protein needed to allow the blood to clot. If licensed, valoctocogene roxaparvovec would be the first gene therapy for severe haemophilia A. Valoctocogene roxaparvovec administered as a single treatment would be sufficient to maintain normal levels of factor VIII in adult males with severe haemophilia A, and might reduce the need for regular factor VIII prophylaxis (preventative treatment).

Drugs

April 2020

Liposomal bupivacaine for post-operative pain

Liposomal bupivacaine is in late-stage clinical development for the treatment of postoperative pain in adults. Post-operative pain is a common occurrence for those patients who have surgery. Controlling post-operative pain is important for ensuring a good patient experience, optimising post-operative outcomes and enhancing recovering, and the prevention of chronic postsurgical pain in the longer term.

Drugs

April 2020

Eptinezumab for prevention of Migraine

Eptinezumab is a drug, which potentially reduces the occurrence of migraine by blocking the CGRP ligand (a protein) from attaching to, and activating its receptor; a process thought to be involved in migraine. In clinical trials, eptinezumab appears effective and well tolerated in the prevention of migraine. If licenced, eptinezumab will offer an additional option for the preventative treatment of migraine with or without aura in adults.

Drugs

April 2020

Anifrolumab for moderately to severly active, autoantibody-positive systemic lupus erythematosus

Anifrolumab is a drug designed to specifically block type I IFN signalling by binding to part of the type I IFN receptor and therefore preventing activity of all IFNs that are involved in the inflammatory pathway. If licensed, anifrolumab will offer an add-on treatment option for patients with moderately to severely active SLE.

Drugs

April 2020

Rivoceranib for locally advanced or metastatic gastric or gastro-oesophageal junction cancer – after two or more therapies

Rivoceranib is an oral medicine with potential anti-tumour activities. It works by binding to a receptor responsible for preventing cell movement and multiplication and decrease the tumour mass. If licensed, rivoceranib will provide an additional treatment option for patients with locally advanced, unresectable or metastatic gastric or gastrooesophageal junction cancer that has progressed since last treatment with at least two prior lines of therapies.

Drugs

March 2020

Baloxavir marboxil for the post-exposure prevention of influenza in people aged 12 years and older

Baloxavir marboxil is given by mouth as tablets and works in a different way to existing flu medications by blocking a specific process which influenza viruses use to multiply within the body. There is evidence that this medication may be effective in people for whom existing flu medicines do not work. Additionally, only one dose of baloxavir marboxil is needed whereas existing flu prevention medications need to be taken over several days. If licenced, baloxavir marboxil may offer an additional prevention option for individuals who have been in contact with someone who has influenza.

Drugs

March 2020

Baloxavir marboxil for the treatment of influenza in patients aged 12 years and older and are at high risk of developing influenza complications

Baloxavir marboxil is given by mouth as tablets within 48 hours of symptom onset and works in a different way to existing flu medications by blocking a specific process which influenza viruses use to multiply within the body. There is evidence that this medication may be effective in people for whom existing flu medicines do not work. Additionally, only one dose of baloxavir marboxil is needed whereas existing flu medications need to be taken over several days. If licenced, baloxavir marboxil may offer an additional treatment option for patients with influenza.

Drugs

March 2020

Eladocagene exuparvovec for aromatic l-amino acid decarboxylase deficiency

Eladocagene exuparvovec is a type of gene replacement therapy which involves the transfer of the gene encoding the production of the enzyme needed by the brain for the formation of dopamine and serotonin. The gene therapy is injected via a surgical procedure into an area of the brain called the putamen. By increasing production of the AADC-enzyme, this therapy increases dopamine production in the target area of the brain and improves motor and cognitive symptoms in patients. If licensed, eladocagene exuparvovec will provide the first medicinal treatment option for adult and child patients with AADC-deficiency, a disease of very high unmet clinical need.

Drugs

March 2020

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.

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