Medicine Industry's Gateway to NICE

Iptacopan is in development for the treatment of patients with complement 3 glomerulopathy (C3G) which is a rare disease. C3 is a blood protein that plays a key role in the normal immunity. The damaging build-up of the C3 protein in kidneys of patients with C3 glomerulopathy mostly results from dysregulation of the alternative complement […]

Upadacitinib is currently in clinical development for the treatment of giant cell arteritis (GCA) in adults aged 50 years and older. GCA is a disease of blood vessels where the arteries, particularly those in the neck and at the side of the head (the temples), become inflamed. Common symptoms include headaches with severe pain and […]

Neoadjuvant (before surgery) nivolumab in addition to gemcitabine and cisplatin followed by adjuvant (additional) nivolumab is in clinical development for muscle-invasive bladder cancer (MIBC). MIBC is when the cancer has spread beyond the lining of the bladder and into the muscle layer. The most well-known risk factor for MIBC is smoking, and the most common […]

Giroctocogene fitelparvovec is in development for the treatment of moderately severe to severehaemophilia A in adult males. Haemophilia A is a rare bleeding disorder, caused by lack of bloodclotting factor VIII which facilitates blood clotting and reduces bleeding events. The absence offactor VIII makes patients prone to excessive and uncontrolled bleeding. In severe cases, this […]

Sarilumab is currently in clinical development for the treatment of children and adolescents aged 2-17 with polyarticular Juvenile Idiopathic Arthritis (JIA) or oligoarticular extended JIA subtype. Arthritis is inflammation of the joints, and juvenile idiopathic arthritis means the arthritis of unknown origin present in children and adolescents. Symptoms of polyarticular JIA include joint pain, swelling […]

Sirolimus-eluting collagen implant is in clinical development for prevention of arteriovenous (AV) vascular access dysfunction in patients with end stage renal disease (ESRD) who are undergoing haemodialysis. ESRD, also known as stage 5 chronic kidney disease (CKD) require either a kidney transplant or dialysis to remove excess fluid and waste products from the blood when […]

An anaplastic astrocytoma is a rare cancerous brain tumour that grows from astrocyte brain cells. Anaplastic means the cells divide rapidly and don’t look like normal cells in structure or function. They affect adults and children and are often treated by neurosurgery followed by radiotherapy and sometimes chemotherapy. They’re more common in adults between the […]

Venglustat is in clinical development for the treatment of patients with Gaucher disease type 3 (GD3) who have been effectively treated with Enzyme Replacement Therapy (ERT) for at least 3 years. Gaucher Disease (GD) is a genetic disorder caused by an enzyme deficiency where fatty substances build up in areas such as the spleen, liver, […]

PXT3003 is currently in clinical development for treatment of patients aged 16-65 years with Charcot-Marie-Tooth disease type 1A (CMT1A). CMT is a group of inherited conditions that damage the peripheral nerves (reside outside the brain and spinal cord). CMT1 is characterised by muscle weakness and atrophy, which can lead to repeated ankle sprains, and changes […]

Pirtobrutinib is currently in clinical development for the treatment of patients with chronic lymphocytic leukaemia (CLL) or small lymphocytic lymphoma (SLL) who have previously received treatment with the current standard of care. CLL and SLL are types of cancer in which too many white blood cells (B-cells) are produced, these cells build up in the […]

Galcanezumab is in clinical development for the preventative treatment of paediatric patients with episodic or chronic migraine. Migraine is one of the most common neurological conditions in the paediatric population with unique clinical characteristics that can evolve with age. These characteristics can include shorter attacks, different location of pain (bilateral in younger children), and light […]

Pegcetacoplan is in clinical development for the treatment of patients with amyotrophic lateral sclerosis (ALS), a form of motor neurone disease. ALS is a progressive disease where the nerve cells responsible for sending instructions to the muscles gradually deteriorate, leading to weakness, muscle wasting and paralysis. ALS is a debilitating and life-threatening disease. Currently, there […]

Ravulizumab is in development for the treatment of thrombotic microangiopathies following haematopoietic stem cell transplantation (HSCT-TMA) in children, adolescents, and adults. Thrombotic microangiopathies (TMA) are clinical syndromes defined by the destruction of red blood cells, low platelets, and organ damage due to the formation of microscopic blood clots in capillaries and small arteries. Haematopoietic stem […]

Tiragolumab in combination with atezolizumab is in clinical development to treat patients withunresectable oesophageal squamous cell carcinoma (ESCC) following chemoradiotherapy. ESCCis a type of cancer that begins in the thin, flat cells lining the food pipe (oesophagus) and mayspread to other parts of the body. Unresectable refers to cancer which cannot be removedcompletely by surgery. […]

Fosdenopterin is currently in clinical development for the treatment of paediatric patients aged 1 day to 5 years with molybdenum cofactor deficiency (MoCD) type A. Patients with MoCD type A cannot produce a substance known as cyclic pyranopterin monophosphate (cPMP). MoCD type A is a rare genetic disease that can appear shortly after birth and […]

Pembrolizumab in combination with Bacillus Calmette-Guerin (BCG) is in development for the treatment of high-risk non-muscle invasive bladder cancer (NMIBC) that is recurrent or persistent following BCG induction or naïve to BCG treatment. Bladder cancer is when cancerous cells develop in the lining of the bladder. NMIBC is the most common type of bladder cancer […]

Cabotegravir is in clinical development as an alternative method of protecting an individual from contracting the human immunodeficiency virus type 1 (HIV-1). HIV is a chronic life altering condition that requires, daily antiretroviral therapy (ART) as a regular treatment routine. If left unmanaged it can lead to serious complications, such as acquired immunodeficiency syndrome (AIDS). […]

Linerixibat is in clinical development for the treatment of primary biliary cholangitis (PBC). PBC is a chronic autoimmune disease where bile (digestive fluid) flow from the liver is disrupted (i.e., cholestasis), resulting in excess bile acids in circulation. Cholestatic pruritus is an itch which is internal and cannot be relieved by scratching. The cause of […]

WP1048 is in clinical development for the treatment of adults with grass pollen allergy. Grass pollen allergy, also known as hay fever or allergic rhinitis, is caused by an immune reaction to proteins found in pollen from various types of grass. The allergy presents symptoms such as itchy eyes, blocked and inflamed nose and persistent […]

Alpelisib is in development for the treatment of the rare disease PIK3CA-related overgrowth spectrum (PROS). PROS is a group of genetic conditions where parts of the body become abnormally overgrown and can affect anywhere from the limbs to the brain, resulting in a wide range of symptoms which can include: pain, fatigue, or seizures depending […]

Palbociclib in combination with trastuzumab with or without pertuzumab and endocrine therapy is currently in clinical development for use as a maintenance therapy for patients with hormone receptor positive (HR+) and human epidermal growth factor receptor 2 positive (HER2+) metastatic breast cancer who have had prior treatment. Breast cancer occurs when abnormal cells in the […]

Telisotuzumab vedotin is in development for the treatment of c-Met+ non-small cell lung cancer. Non-small cell lung cancer is the most common form of lung cancer. A c-Met is a protein in humans that is encoded by the MET gene that leads to growth in cells. Smoking is the cause of most lung cancers and […]

Invimestrocel is in clinical development for the treatment of acute ischaemic stroke within 18-36hours of onset. Ischaemic stroke is the most common type of stroke. It happens when a bloodvessel is blocked by a blood clot, cutting off blood flow to part of the brain (ischaemia). Withoutblood supply, brain cells can be damaged or destroyed […]

Alpelisib in combination with olaparib is in clinical development for the treatment of adults with platinum-resistant or refractory, high-grade serous/endometrioid ovarian cancer, including fallopian tube cancer or primary peritoneal cancer. These cancers occur in the female reproductive system and are often grouped together as they are treated in the same way. Tumours can occur in […]

Benralizumab is currently in clinical development for relapsing or refractory eosinophilic granulomatosis with polyangiitis (EGPA). EGPA, formally known as Churg-Strauss Syndrome, is a rare disease characterised by blood vessel inflammation. This inflammation can restrict blood flow to organs and tissues, sometimes permanently damaging them. Relapsed refers to a disease that has come back, and refractory […]

Nivolumab in combination with ipilimumab is in clinical development for the treatment of previously untreated locally advanced non-small-cell lung cancer (NSCLC). NSCLC is the most common form of lung cancer. Locally advanced NSCLC means the cancer is in more than one lobe of the lung, or it has spread to lymph nodes or nearby structures […]

Guselkumab is in clinical development for the treatment of moderately to severely active ulcerative colitis (UC). UC is a long-term condition where parts of the large bowel become inflamed, causing urgent bloody diarrhoea and abdominal pain. The symptoms of UC often follow a pattern where individuals with the condition have periods of no symptoms or […]