November 2019
Cabotegravir (oral tablet) in combination with rilpivirine (oral tablet) for the short-term treatment of HIV-1 infection
Cabotegravir and rilpivirine tablets are in development as an oral lead-in therapy for a period of approximately one month as a short-term oral bridging treatment for Human Immunodeficiency Virus 1 (HIV-1) patients that are considered eligible for the long-acting injectable cabotegravir and rilpivirine therapy. HIV is a type of viral infection caused by a type of virus referred to as a retrovirus. HIV-1 is the most common and highly communicable type of HIV. HIV is a lifelong, chronic disease that nowadays can be managed with antiretroviral therapies (ARTs). Since HIV virus can quickly adapt and become resistant, a combination of ART drugs is normally used. Usually patients take between one and 4 or 6 tablets a day. Failing to do so will result in a weakened immune system and increased vulnerability to infections.
November 2019
Fostemsavir for HIV-1 infection for heavily treatment-experienced adults
Fostemsavir is in clinical development for human immunodeficiency virus-1 (HIV-1) infection in heavily treatment-experienced adults. HIV is a virus that damages the cells in the immune system and weakens the body’s ability to fight everyday infections and diseases. HIV infection is incurable but there are effective treatments that enable most infected people to live a healthy life. Antiretroviral (ARV) medicines are used to treat HIV. However, poor adherence and the subsequent development of drug resistance is one reason why HIV treatment can fail. A drug-resistant HIV strain is one which is less susceptible to the effects of one or more anti-HIV drugs. For highly treatment-experienced patients who have failed on a number of treatment regimens and have limited treatment options, complex regimens including drugs to which the virus is partially resistant may be required.
November 2019
Palovarotene for flare-ups in fibrodysplasia ossificans progressiva – first line
Palovarotene is in clinical development for episodic use in the prevention of heterotopic ossification (HO) in patients with fibrodysplasia ossificans progressiva (FOP). FOP is a disabling condition, caused by the formation of bony bars within the muscles of the body (HO). This bone formation is usually first noticed in early childhood as a series of hard lumps in the neck or along the spine. These lumps, which may be tender, gradually shrink in size as the affected muscles are replaced by bone. The appearance of bony lumps in muscles is usually spontaneous but can also be provoked by any injury to the muscles.
November 2019
Treosulfan in combination with fludarabine for paediatric non-malignant disease before allogeneic stem cell transplant
Treosulfan in addition to fludarabine is in clinical development for paediatric non-malignant disease prior to allogeneic stem cell transplant. Treosulfan is a medicine given to patients before they have a bone marrow transplant from a donor known as ‘allogeneic haematopoietic stem cell transplantation’. It is used as a ‘conditioning’ treatment to clear the patient’s bone marrow and make room for the transplanted bone marrow cells, which can then produce healthy blood cells. Treosulfan is used together with another medicine called fludarabine for the treatment of a variety of disorders that require a bone marrow transplant.
November 2019
Ruxolitinib for chronic graft versus host disease (cGvHD)
Ruxolitinib is in clinical development for chronic graft versus host disease (cGvHD). After a donor stem cell transplant, the donor’s stem cells (the graft) may sometimes react against the host’s own cells. This is called GVHD. cGvHD may happen more than three months after transplant. It can develop from acute GVHD or happen on its …
November 2019
Secukinumab for enthesitis-related arthritis and juvenile psoriatic arthritis in children and adolescents from the age of 2 years
Secukinumab as a subcutaneous injection is in clinical development for the treatment of enthesitis related arthritis (ERA) and juvenile psoriatic arthritis (JPsA). These conditions belong to a group of arthritis conditions of unknown cause known as juvenile idiopathic arthritis which affect children. JPsA patients have arthritis and psoriasis, an inflammatory skin disease and ERA patients have arthritis and enthesitis, inflammation of the ligaments and tendons. These conditions are the result of the immune system mistakenly attacking the body’s own cells at the joints and the skin or tendons, causing swelling, pain and reduced mobility.
November 2019
Upadacitinib for atopic dermatitis
Upadacitinib is in development for the treatment of moderate to severe atopic dermatitis (AD). AD is a chronic inflammatory skin disease that affects both children and adults and is characterised by redness, itchiness, and scaling of the skin. Some people only have small patches of dry skin, but others may experience widespread red, inflamed skin …
November 2019
Filgotinib for moderately to severely active ulcerative colitis
Filgotinib is in clinical development for the treatment of patients with moderately to severely active ulcerative colitis. Ulcerative colitis is a type of inflammatory bowel disease that causes inflammation and ulcers in the bowel and rectum which can cause diarrhoea, abdominal pain and faecal urgency or incontinence. The symptoms of ulcerative colitis often follow a pattern of relapse and remission where they have periods of none or mild symptoms followed by periods of increased symptoms flare-up (‘active’). There are no curative therapies for ulcerative colitis and current treatment aims to relieve symptoms during a flare-up and maintain remission by preventing symptoms from returning.
November 2019
Ruxolitinib for acute graft versus host disease (aGvHD)
Ruxolitinib is in clinical development for acute graft versus host disease (aGvHD). After a donor stem cell transplant, the donor’s stem cells (the graft) may sometimes react against the host’s own cells. This is called GVHD. aGVHD is most likely to happen in the first three months after transplant. The symptoms depend on which parts of the body are affected. It often causes an itchy skin rash. If the bowel, the stomach or the liver are affected, the patient may have sickness and diarrhoea. aGVHD is graded by how severe it is. It goes from grade 1, which is mild, to grade 4 which is very severe. Current standard treatment includes the use of steroids but this is often associated with significant side effects. Steroid resistance in GvHD may also develop which is difficult to treat and associated with a high mortality.
November 2019
Atezolizumab for muscle-invasive urothelial carcinoma –adjuvant
Atezolizumab is currently in clinical development for the treatment of patients muscle-invasive urothelial cancer (MIUC) including muscle-invasive bladder cancer (MIBC) and upper tract urothelial cancer (UTUC) patients who are at high risk following resection. MIBC is a cancer that spreads into the thick muscle deep in the bladder wall. MIBC starts in the inner bladder layer and then grows in the deep muscle. While UTUC can arise along any part of the urinary tract lined with urothelium with the majority of cases in the lower tract and rest in the upper tract. Over time the tumour may grow outside the bladder into tissues close by and then may spread to lymph nodes, the lungs, the liver and other parts of the body. The current standard care of treatment includes a surgery which might not be adequate and some patients might be at high risk for recurrence.
