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.
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).
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.
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.
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.
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.
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.
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.