Deferiprone for transfusional iron overload in sickle cell disease and other anaemias – First Line
Deferiprone is in clinical development for patients with sickle-cell disorder (SCD) and other anaemias that are suffering from iron overload due to frequent transfusions to increase their red blood cell count. SCD is a group of inherited disorders where the red blood cells become hard and sticky and look like a C-shaped farm tool called a “sickle”. The sickle red blood cells die early, and patients often require blood transfusions. Iron overload is an effect of frequent transfusions in SCD. Excess iron in the body can be toxic to major organs like the heart and liver.
Treprostinil for chronic thromboembolic pulmonary hypertension
Treprostinil is in clinical development for patients with chronic thromboembolic pulmonary hypertension (CTEPH). CTEPH is a progressive disease caused by blood clots forming in the lungs that do not dissolve. These clots restrict blood flow through the lungs and cause scar tissue to form on the inside of the pulmonary arteries (arteries that supply the lungs) resulting in pulmonary hypertension. This means the heart has to work harder to pump the blood and can weaken the heart muscles. Some patients may be offered surgery to remove the scar tissue but in patients who are inoperable or who have persistent/recurrent CTEPH after surgery there is a need for additional treatment options.
Clobetasol propionate for postoperative pain and inflammation associated with cataract surgery
Clobetasol propionate ophthalmic nanoemulsion is in development for the treatment of post-operative pain or inflammation following cataract surgery. A cataract is a cloudy area in the lens, which can develop slowly and progress to general loss of transparency, which may lead to functional impairment and visual deterioration. Cataract surgery is the only effective treatment to improve or maintain vision, however, inflammation and pain are common difficulties following this treatment.
JZP-458 for acute lymphoblastic leukaemia and lymphoblastic lymphoma
Acute lymphoblastic leukaemia (ALL) is a type of cancer affecting white blood cells, which results in overproduction of faulty cells. These cells take over the bone marrow leading to anaemia, infection, bruising and bleeding. ALL is a rare condition, usually affecting more children than adults. Lymphoblastic lymphoma (LBL) is similar to ALL, they differ in where they often occur in the body (lymph nodes and thymus gland in LBL, and blood and bone marrow in ALL), and they are both treated in the same way.
Pembrolizumab as adjuvant therapy for resected high-risk stage II melanoma
Melanoma is the most common skin cancer in the UK and is characterised by changes to mole size or shape. It is when melanocytes (cells that give skin its tan or brown colour) grow uncontrollably. Stage II melanoma is where the cancer has spread locally, as opposed to other areas of the body, and can result in a larger tumour sometimes with broken skin. Patients with high-risk stage 2 melanoma cancer may have an increased chance of the cancer returning after surgical resection (removal of the tumour) because of the tumour being thicker and deeper in the skin, which makes it difficult to remove all cancerous cells. There is currently no recommended systemic treatment after surgical resection if the cancer comes back in patients with stage II melanoma.
Trastuzumab deruxtecan for metastatic HER2 – positive breast cancer – second line
Trastuzumab deruxtecan is in clinical development for the treatment of adults with HER2- positive, unresectable and/or metastatic breast cancer who have previously been treated with trastuzumab and taxane. HER2-positive breast cancer is when the cancer tests positive for HER2 protein, which promotes the growth of cancer cells and tend to be more aggressive than other types of breast cancer. Metastatic breast cancer (stage IV) is when the cancer has spread beyond the breast and nearby lymph nodes to other organs in the body, while unresectable means that the cancer cannot be treated by surgery. Treatment of the disease often involves the use of anti-HER2 therapies, chemotherapy, or a combination of both.
Plinabulin in combination with G-CSF for chemotherapy-induced neutropenia
Plinabulin in combination with G-CSF is being developed for the prevention of chemotherapy-induced neutropenia (CIN) in patients with solid tumours receiving myelosuppressive chemotherapy with docetaxel, doxorubicin, and cyclophosphamide (TAC). Neutropenia is a condition associated with low levels of neutrophils, a type of white blood cell, which is a common side effect of cancer treatment. Severe neutropenia can lead to life-threatening complications due to resulting infections, and cause reduction or delay of chemotherapy treatment, potentially compromising the benefit to cancer patients.
Cemiplimab for recurrent, persistent or metastatic cervical cancer – second line
Cemiplimab is in clinical development for the treatment of recurrent, persistent, or metastatic cervical cancer. Recurrent cancer is when the cancer returns months or years after the original treatment; persistent cancer is when the tumour does not respond to treatment or a second tumour develops despite the completion of treatment. Metastatic cancer is when the …
Lorlatinib for advanced ALK-positive non-small cell lung cancer – first line
Lorlatinib is being investigated for the treatment of advanced anaplastic lymphoma kinase (ALK) positive non-small cell lung cancer (NSCLC). NSCLC comprises the majority of lung cancers in the UK. Symptoms of lung cancer include a persistent cough, shortness of breath, coughing up blood, aches and pains in the chest or shoulder, loss of appetite, weight loss and fatigue. While current treatments exist for ALK-postive NSCLC, significant unmet medical need remains for more effective treatment options as treatment with current options inevitably leads to further progression of the disease over time.