Lumacaftor and Ivacaftor (Orkambi) for Cystic Fibrosis Homozygous for the F508del Mutation in Patients Aged 6‐11 Years
Lumacaftor and ivacaftor (Orkambi) is a medicine used to treat cystic fibrosis in patients aged 6 years and above who have a genetic mutation called the F508del mutation. This mutation affects the gene for a protein called cystic fibrosis transmembrane conductance regulator (CFTR) which is involved in regulating the production of mucus and digestive juices. Orkambi is used in patients who have inherited the mutation from both parents and therefore have the mutation in both copies of the CFTR gene. Orkambi is taken orally as tablets twice a day. It offers a new treatment option for patients with cystic fibrosis with the advantage of targeting the actual disease process rather than just the symptoms.
Nintedanib for Systemic Sclerosis Associated Interstitial Lung Disease
Nintedanib (OFEV®) is a medicine that is being developed for the treatment of Systemic Sclerosis associated interstitial lung disease (SSc-ILD). It acts by targeting the specific mechanisms by which scarring of the lungs occur, reducing progression of the disease. It is given by mouth as capsules. Nintedanib (as OFEV) is already available in the EU for the treatment of idiopathic pulmonary fibrosis (IPF). Because SSc-ILD and IPF share similarities in how the underlying lung scarring, or fibrosis, forms in people with the disease, the development of nintedanib for the treatment of SSc-ILD may address unmet needs. If licensed, nintedanib may offer an additional treatment option (the first licensed) for patients with SSc-ILD.
Benralizumab for Chronic Obstructive Pulmonary Disease (COPD)
Benralizumab is a new drug being developed for the treatment of chronic obstructive pulmonary
disease. It is administered by injection under the skin and acts by targeting specific proteins that
causes the airway to narrow. If benralizumab is licensed for use in the UK, it could be a new treatment
option for patients with chronic obstructive pulmonary disease that may improve quality of life and
reduce the number of exacerbations.
Tralokinumab for severe, uncontrolled asthma
Asthma is a long-term condition that affects the lungs and breathing. People with asthma have symptoms such as coughing, wheezing, chest tightness and breathlessness. Some people have symptoms despite trying various treatments or may suffer from more sudden ‘asthma attacks’. Severe asthma attacks can lead to hospitalisation, and on rare occasions can be life threatening. …
Nyxoah Genio™ System for obstructive sleep apnoea
The Nyxoah Genio™ system is a device to treat people with obstructive sleep apnoea (OSA) who are not suitable for continuous positive airway pressure (CPAP) therapy. OSA is a common sleep disorder where the walls of the throat relax and narrow during sleep, interrupting normal breathing. The Nyxoah Genio™ system consists of a device which …
Molgramostim inhalation for autoimmune pulmonary alveolar proteinosis
Molgramostim inhalation is a new drug to treat autoimmune pulmonary alveolar proteinosis (PAP). Autoimmune PAP is a rare lung disease that causes a build-up of protein material in the air sacs of the lungs, making it difficult to breath. Molgramostim inhalation is breathed in and works by altering the body’s immune response, helping to prevent …
Anti-Pseudomonas aeruginosa IgY for pseudomonal infection in cystic fibrosis
Pseudomonas aeruginosa is a bacterium that can damage the lungs of patients with cystic fibrosis even more, it can be difficult to treat, and once it becomes well-established, it may never be properly cleared from the lung.Anti-Pseudomonas aeruginosa IgY is a new drug for the treatment of infection with Pseudomonas aeruginosa in patients with cystic …
Neurally Adjusted Ventilatory Assist (NAVA)
Neurally Adjusted Ventilatory Assist (NAVA) is a respiratory monitoring and support device for neonatal and paediatric patients, developed by Maquet Getinge Group. NAVA is a mechanical ventilation method that is characterised by a proportional response to diaphragm electromyography (EMG), originating from centrally controlled phrenic nerve stimulation. The device consists of a single-patient use nasogastric feeding …