Liposomal cytarabine-daunorubicin for treating relapsed or refractory acute myeloid leukaemia in paediatric patients.
Liposomal cytarabine-daunorubicin is made up of the chemotherapy drugs daunorubicin and cytarabine contained within fat-based particles called liposomes. Liposomal cytarabinedaunorubicin is delivered by intravenous infusion and provides controlled release of daunorubicin and cytarabine. Daunorubicin works by interrupting the copying of DNA which is necessary for cancer cell growth and cytarabine works by inhibiting DNA production to kill cancerous cells. If licenced, liposomal cytarabine-daunorubicin may offer an additional treatment option for paediatric patients with relapsed or refractory AML.
Istradefylline for patients with Parkinson’s disease experiencing end of dose fluctuations
Istradefylline is a selective adenosine A2A receptor inhibitor; these receptors are found in the region of the brain that suffers degeneration in PD. The treatment works by increasing the levels of dopamine in the substantia nigra. Current treatment for PD is associated with adverse events. Several phase III clinical trials have shown istradefylline, when taken orally, to be safe and effective in the treatment of PD during OFF periods. If licensed, istradefylline will provide a new therapeutic option to managing people with Parkinson’s as the first non-dopaminergic add-on treatment.
Bintrafusp alfa for locally advanced or metastatic biliary tract cancer – second-line
Bintrafusp alfa, delivered via intravenous injection, is a novel bi-functional fusion protein that is composed of an antibody fused with a receptor. Chemotherapy aims to kill cancer cells by directly attacking them, whereas bintrafusp alfa works twofold by preventing cancer from being resistant to a patient’s immune system and stopping cancer cells from growing.
ABBV-951 for motor fluctuations in Parkinson’s disease
ABBV-951 is administered under the skin (subcutaneous infusion) to deliver therapeutic quantities of the drugs levodopa and carbidopa. Levodopa can be converted by the body into dopamine in order to supplement the low levels of dopamine in PD patients and improve motor symptoms. Carbidopa makes more levodopa available for transport into the brain. ABBV-951 eliminates the ‘wearing off’ effect by providing a continuous infusion of levodopa into the bloodstream so there is less fluctuation in dopamine levels and improved motor control. If licenced, ABBV-951 would provide an additional treatment option for PD patients who are levodopa responsive and who have inadequately controlled motor fluctuations.
Pralsetinib for RET-fusion positive metastatic non-small-cell lung cancer
Pralsetinib is an investigational, once-daily oral precision therapy designed to selectively target, and potentially inhibit, RET mutations, regardless of the tissue of origin. If licensed, pralsetinib will offer a treatment option for adult patients with RET-fusion positive advanced NSCLC, who currently have no highly selective therapies available.
Tofacitinib for Ankylosing Spondylitis
The active substance in tofacitinib works by blocking the action of enzymes (proteins) known as Janus kinases. These enzymes play an important role in the process of inflammation that occurs in rheumatoid, psoriatic arthritis and ulcerative colitis. By blocking the enzymes’ action, tofacitinib helps reduce the inflammation and other symptoms of these diseases. Tofacitinib is administered orally. If licensed, tofacitinib would offer an additional treatment option for patients with active AS, who have responded inadequately to conventional therapy.
Pevonedistat in addition to azacitidine for Higher-Risk Myelodysplastic Syndromes, Chronic Myelomonocytic Leukemia and Low Blast Acute Myeloid Leukemia – first-line
Pevonedistat, administered by IV infusion, blocks the activity of an enzyme in the body called NEDD8-activating enzyme (NAE). NAE is involved in the growth and spread of cancer cells. By blocking NAE in patients with AML, pevonedistat is expected to prevent the degradation of certain proteins affecting key pathways including DNA repair, the cell cycle, and cell survival, thereby preventing the development and worsening of cancer. Chemotherapy drugs, such as azacitidine, work in different ways to stop the growth of cancer cells. If licensed, pevonedistat in addition to azacitidine would offer an additional first-line treatment option for adults with HR-MDS, CMML, or LB-AML.
Plinabulin in addition to docetaxel for advanced, metastatic non-small-cell lung cancer – second line or greater
Plinabulin is an intravenously administered drug that binds to and affects the function of the protein tubulin within cells of the body. In certain cells of the immune system called Dendritic Cells (DCs) tubulin targeting with plinabulin activates a protein named GEF-H1 which boosts the ability of DCs to activate T-cells, which go on to kill cancer cells. These effects are thought to contribute towards the anti-cancer benefits of plinabulin. If licensed, plinabulin in addition to docetaxel will provide a new regimen for advanced NSCLC.
Budesonide granules for induction of remission in lymphocytic colitis – first line
Budesonide is a corticosteroid that exerts anti-inflammatory effects in the gastrointestinal tract. The treatment works by binding with high affinity to intracellular glucocorticoid receptors. In one phase III clinical trial, budesonide was shown to be significantly effective in inducing clinical remission in lymphocytic colitis patients in comparison to a placebo. If licensed, budesonide will offer a treatment option for patients with lymphocytic colitis.