Lumasiran for primary hyperoxaluria type 1 in paediatric patients aged up to 5 years
Lumasiran is in clinical development for the treatment of primary hyperoxaluria type I (PH1) in paediatric patients up to 5 years old. PH1 is a very rare disease caused by certain genetic mutations, in which excess production of a substance called oxalate results in the deposition of oxalate crystals in the kidneys and urinary tract. This leads to stone formation and kidney failure with significant morbidity and mortality. Treatment options for PH1 include vitamin B6 which is known to reduce the body’s production of oxalate, dietary recommendations to prevent kidney stones and combined liver-kidney transplantation before or after development of end-stage kidney failure.
Nivolumab in combination with cisplatin and fluorouracil for oesophageal cancer – first-line
Nivolumab in combination with cisplatin and fluorouracil is in clinical development for patients with unresectable, advanced, recurrent or metastatic oesophageal squamous cell cancer cell carcinoma. Advanced oesophageal cancer begins in the food pipe and spreads to other parts of the body. Squamous cell cancers develop from the cells that make up the inner lining of the oesophagus. Symptoms include difficulty swallowing, persistent acid indigestion or heartburn, weight loss, pain in the throat, and chronic cough. Lifestyle factors are attributed to most oesophageal cancers, including smoking and being overweight.
Belimumab for lupus nephritis
Belimumab is in clinical development for the treatment of adults with active lupus nephritis (LN) who are uncontrolled on current standard of care. LN is a complication affecting kidney function brought on by systemic lupus erythematosus (SLE), a chronic autoimmune, inflammatory disease that affects different organs. Around a third of people suffering with SLE will develop LN. The kidney damage seen in LN occurs due to a person’s immune cells (B Cells) attacking their own kidneys which causes inflammation and affects overall kidney function. Current treatment involves suppression of the immune system and management of symptoms, but it is not always effective. If left untreated, LN can lead to kidney failure which requires dialysis or a kidney transplant.
Olaparib monotherapy for BRCA-mutated platinum-sensitive relapsed ovarian, fallopian tube or primary peritoneal cancer
Olaparib is a medicinal product currently in development for the treatment of Breast Cancer Gene (BRCA)-mutated relapsed ovarian, fallopian tube or primary peritoneal cancer in patients who have received at least 2 prior platinum treatments and have progressed at least 6 months after their last platinum treatment. Ovarian cancer includes a group of tumours that arise from diverse types of tissue contained in the ovary and can often spread from the ovary to any surface within the abdominal cavity including the fallopian tubes and peritoneal cavity.
Pertuzumab and trastuzumab (fixed-dose combination) in addition to chemotherapy for breast cancer
Pertuzumab/trastuzumab (fixed-dose combination (FDC) in addition to chemotherapy is in clinical development as a subcutaneous formulation (SC) for the adjuvant treatment of adults with the human epidermal growth factor receptor 2 (HER2) positive early and metastatic breast cancer. HER2 positive breast cancer is a subtype in which the HER2 receptor is over expressed on the cell surface. HER2 promotes the growth of cancer cells and this breast cancer subtype tends to be more aggressive than other types. Metastatic breast cancer is when cancer has spread beyond the breast and nearby lymph nodes to other organs in the body. Treatment of the disease often involves the use of anti-HER2 therapies, chemotherapy or a combination of both.
Migalastat for Fabry disease in children aged 12 to 15 years
Migalastat works by stabilizing the body’s own dysfunctional enzyme, so it can clear the accumulated disease substrate in patients who have amenable mutations. Migalastat is currently licenced for patients aged 16 or over with Fabry disease and an amenable mutation. If the license is extended, migalastat may offer an additional treatment option for paediatric subjects 12 to 15 years old with Fabry disease and an amenable mutation, who weight over 45Kgs.
Durvalumab in combination with tremelimumab for unresectable hepatocellular carcinoma – first line
Durvalumab in combination with tremelimumab is in clinical development for patients with unresectable hepatocellular carcinoma (HCC), the most common type of liver cancer that occurs mainly in patients with underlying chronic liver disease and cirrhosis. Unresectable HCC occurs when the cancer has spread to lymph nodes or to other organs and cannot be treated by surgery. Unresectable HCC is often diagnosed late in life and has a poor prognosis. It is a debilitating condition with many distressing symptoms, including pain, digestive problems and weight loss. The current standard of care can only slow the progression of the cancer and extend survival.
Ibalizumab in combination with other antiretroviral(s) for multidrug-resistant HIV-1 infection
Ibalizumab in combination with other antiretroviral(s) has been developed for the treatment of adults infected with multidrug-resistant HIV-1 (MDR HIV-1). HIV is a virus that damages the cells in the immune system and weakens the ability to fight everyday infections and diseases. HIV-1 is the most common type of HIV infection, which is incurable but there are effective treatments that enable most infected people to live a healthy life. Antiretroviral medicines are used to treat HIV. There are however limited effective treatments for some treatment-experienced patients with extensive MDR HIV-1.
Cabotegravir in combination with rilpivirine long acting injection for the treatment of HIV-1 infection
Cabotegravir and rilpivirine long acting injections are in development for the treatment of HIV-1 patients that are considered clinically suitable for injectable therapy. HIV (Human Immunodeficiency Virus) is a type of viral infection caused by a virus referred as 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. Usually patients take between one and 4 or 6 tablets a day. Failing to do so will result in a weaken immune system and increased vulnerability to infections.
Trastuzumab emtansine in combination with pertuzumab for HER2-positive early breast cancer – adjuvant therapy
Trastuzumab emtansine consists of an anti-HER2 therapy (trastuzumab) and a chemotherapy agent (emtansine or DM1) combined together as an antibody-drug conjugate. Trastuzumab specifically binds to cancer cells that are HER2-positive which provides a targeted delivery of the cytotoxic DM1 inside cancer cells, potentially limiting damage to healthy tissue. Pertuzumab, is designed to attach to HER2, and stop HER2 producing signals that cause the cancer cells to grow. The combination is thought to provide a more comprehensive, dual blockade of HER pathways and prevent tumour cell growth and survival, and if licenced, will offer an additional adjuvant treatment option for patients with HER2- positive early breast cancer.