Medicine Industry's Gateway to NICE

Dersimelagon is being developed for the treatment of erythropoietic protoporphyria (EPP) or X-linked protoporphyria (XLP). EPP and XLP are rare diseases that cause intolerance to light. Inpatients with EPP or XLP, exposure to light can lead to symptoms such as pain and swelling of theskin, which prevent patients from being able to spend time outdoors […]

Acalabrutinib in combination with bendamustine and rituximab is in clinical development for thefirst line treatment of mantle cell lymphoma (MCL). MCL is a rare and aggressive form of nonHodgkin lymphoma. It affects the B-cells (a type of white blood cells) and develops in the lymphnodes. The abnormal B-cells start to collect in the lymph nodes […]

Ribociclib with endocrine therapy (ET) is currently in clinical development for patients with earlybreast cancer with hormone receptor positive (HR+)/human epidermal receptor 2 negative(HER2-) subtype. Early breast cancer is where the disease is limited to the breast region and hasnot spread to other parts of the body. Symptoms include swelling of breast regions, breast ornipple […]

Bardoxolone methyl is in clinical development for the treatment of patients aged 12 years andolder with Alport syndrome. Alport syndrome is a genetic condition characterized by kidneydisease, hearing loss, and eye abnormalities. It is caused by a defect (mutation) in a gene for aprotein in the connective tissue, called collagen. There are currently no licensed […]

Atezolizumab with chemotherapy is currently in clinical development for the treatment of earlyrelapsing, recurrent triple-negative breast cancer (TNBC). TNBC is an uncommon form kind ofbreast cancer where the tumour cells do not have any of the receptors that are commonly foundin breast cancer. TNBC is often more aggressive and harder to treat compared to other […]

Sacubitril-valsartan is currently in clinical development for the treatment of paediatric chronicheart failure (HF) with reduced ejection fraction. HF is a clinical syndrome of symptoms and signsthat suggest the efficiency of the heart to pump blood around the body is impaired. The classicalpresenting symptoms of HF in young children are feeding difficulties, growth failure, irritability,and […]

Masitinib in combination with riluzole is in clinical development for the treatment of patients withthe amyotrophic lateral sclerosis (ALS), form of motor neurone disease (MND). ALS is aprogressive disease of the nervous system, where nerve cells in the brain and spinal cord thatcontrol voluntary movement gradually deteriorate, causing loss of muscle function and paralysis.ALS is […]

Crovalimab is currently in clinical development for patients with paroxysmal nocturnalhaemoglobinuria (PNH). PNH is a rare, acquired, life-threatening disease of the blood. The diseaseis characterised by destruction of red blood cells, blood clots, and impaired bone marrow function.It is a blood condition where blood cells are prone to be attacked by part of the body’s […]

Macitentan and tadalafil as a fixed-dose combination (FDC) is in clinical development for thetreatment of pulmonary arterial hypertension (PAH). PAH is a rare disorder in which there issevere narrowing of the arteries of the lungs. More pressure is needed to force blood through thenarrowed artery which this leads to high blood pressure in the lungs

Pembrolizumab in combination with chemoradiotherapy is in clinical development for thetreatment of patients with high-risk, locally advanced cervical cancer. Cervical cancer ariseswhen abnormal cells lining the cervix begin to grow uncontrollably and create tumours. Cervicalcancer may not present symptoms, however the most common symptoms seen are unusualvaginal bleeding, pain during sex, vaginal discharge and pain […]

Momelotinib is currently in clinical development for the treatment of symptomatic myelofibrosiswith anaemia. Myelofibrosis is a rare blood cancer, where the bone marrow is too active so scartissue builds up inside the bone marrow and blood cells cannot develop properly. The reductionin blood cells can result in anaemia, amongst other symptoms such as tiredness, shortness […]

The port delivery system with ranibizumab (PDS) is in clinical development for the treatment ofdiabetic macular oedema (DMO). DMO is swelling of the macula caused by diabetes or byblockage of the veins behind the retina and it is the most common cause of sight loss in peoplewith diabetes. DMO occurs when blood vessels damaged by […]

Atezolizumab and bevacizumab in combination is in clinical development for treating adults with high riskof hepatocellular carcinoma (HCC) coming back (recurring), after complete surgical removal(resection/ablation) of the affected tissue. HCC is the most common type of primary liver cancer, whichaffects men more than women, and is more likely to develop the older a person gets.

Idecabtagene vicleucel is in clinical development for the treatment of adult patients with relapsedor refractory multiple myeloma (MM) who have received at least 2 but no greater than 4 priorMM regimens. MM is a rare, incurable blood cancer that forms in the plasma calls in the bonemarrow, inside some of the large bones of the […]

Baricitinib is in clinical development for children aged 1 to 17 years with juvenile idiopathicarthritis (JIA). JIA is defined as arthritis of unknown causes that manifests itself before the age of16 years and persists for at least 6 weeks, while excluding other known conditions. JIA occurswhen the body's immune system attacks its own cells and […]

Semaglutide is in clinical development for the treatment of overweight and obese adolescentsbetween the ages of 12 and 17. Obesity is a chronic disease and global public health challengeand the prevalence of childhood obesity rising in the UK. Obesity increases the risk of developinga range of health conditions in childhood and later life, including stroke, […]

Durvalumab with bevacizumab is intended to treat hepatocellular carcinoma (HCC), the mostcommon type of liver cancer, where there is a high risk of the cancer returning after curativetreatment. It is common that people with HCC are asymptomatic (do not show symptoms relatedto the cancer). There is increased risk of developing HCC if you have underlying […]

ABP 959 is in development for the treatment of paroxysmal nocturnal haemoglobinuria (PNH).PNH is a rare condition, which occurs due to a genetic mutation within stem cells in the bonemarrow. In PNH, red blood cells lack specific proteins on their surface which normally protectthem from being destroyed (a process called haemolysis) by the body's normal […]

Cenobamate is in clinical development as an adjunctive therapy for adults with primarygeneralized tonic-clonic (PGTC) seizures. A generalised tonic-clonic seizure is the type of epilepticseizure which is defined as a seizure that has a tonic phase (stiffening of the muscles) followed byclonic muscle contractions (rhythmical jerking).

A food allergy is when the body’s immune system reacts to proteins from specific foods, as if itwere a threat. Common foods that cause an allergic reaction are milk, eggs, peanuts, tree nuts,fish, and shellfish. Most food allergies affect younger children under the age of 3 years old,however peanut and tree nut allergies are longer-lasting […]

Gantenerumab is currently in clinical development for the treatment of early Alzheimer’s disease(AD). AD is a progressive neurological disease which is caused by loss of function and death ofneurones, the cells that carry signals or information messages to and from the brain and the restof the body. It is the most common type of dementia.

Odevixibat is currently in clinical development for the treatment of cholestatic liver disease in Alagille syndrome (ALGS) patients. ALGS is an inherited developmental disorder, caused by a mutation in the JAGGED1 gene or in the NOTCH2 gene. The mutation causes problems with early embryonic development leading to abnormalities in various parts of the body. ALGS […]

ASTX727 is in clinical development for patients with de novo or secondary acute myeloid leukaemia (AML), who are not candidates for standard induction chemotherapy. De novo AML is where there has been no past medical history of the condition or exposure to the drugs which commonly treat it, secondary AML is where patients have had […]

Natalizumab biosimilar is being developed as a biosimilar medicine to the approved monoclonal antibody natalizumab and is proposed to be used to treat adult patients relapsing remitting multiple sclerosis (RRMS). Multiple sclerosis is an autoimmune condition where the body’s immune system attacks the brain and nerves. RRMS is characterised by attacks of new or increasing […]

REGN5458 is in clinical development for the treatment of adults with multiple myeloma (MM) whose disease has returned following treatment. MM is a form of cancer that occurs in immune cells found in bone marrow. The disease occurs due to uncontrolled duplication of these immune cells, known as plasma cells. Symptoms of MM can include […]

Empagliflozin is in clinical development for the treatment of adult patients with chronic kidney disease (CKD). In CKD, the kidneys do not function as well as they should. This leads to the leakage of blood and protein into the urine. CKD can be developed due to age, but the main risk factors are diabetes, hypertension, […]

Fordadistrogene movaparvovec is a medicinal product in clinical development for the treatment of ambulatory boys with a confirmed genetic diagnosis of Duchenne muscular dystrophy (DMD). DMD is a severe and rare progressive neuromuscular disorder caused by a gene mutation (change). DMD is caused by an absence of functional dystrophin, a protein that helps keep muscle […]