rAAVrh74.MHCK7.micro-dystrophin for Duchenne muscular dystrophy
rAAVrh74.MHCK7.micro-dystrophin is a medicinal product in clinical development for the treatment of children aged 3 months to 7 years with Duchenne muscular dystrophy (DMD). DMD is a rare progressive neuromuscular disorder caused by a gene mutation (change). DMD is caused by an absence of dystrophin, a protein that helps keep muscle cells intact. It affects …
Tideglusib for congenital myotonic dystrophy
Tideglusib is being development for the treatment of congenital myotonic dystrophy type 1 (CMD1). CMD1 is a form of myotonic dystrophy type 1 (DM1), a rare, genetically determined neuromuscular disorder. CMD1 begins at or around the time of birth and is characterised by severe muscle weakness, cognitive impairment and other developmental abnormalities. The condition usually occurs when the mother already has DM1 and then it is passed on to her child in a more severe form. CMD1 is typically associated with significant medical morbidity and early death. No specific treatment is currently on offer, although supportive care to manage symptoms is available.
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.
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.
Xeomin for sialorrhea in children and adolescents aged 2 to 17 years
Xeomin is one formulation of botulinum neurotoxin type A. It is injected into salivary glands and reduces saliva production. Currently, it is recommended as a specialist option for treating sialorrhea in children and adolescents, but is not yet licensed for this indication. It is an alternative to anticholinergic drugs which are also used to reduce saliva flow, but which can have side effects in a number of patients. If licensed, Xeomin will offer an additional treatment option for chronic sialorrhea in children and adolescents aged 2 to 17 years.
Levosimendan for respiratory function in amyotrophic lateral sclerosis
Levosimendan works through binding to a protein called troponin C, which sensitises cardiac and skeletal muscles to calcium and increases their force of contraction. This increased force of contraction is thought to increase diaphragm function and support respiratory dysfunction. Levosimendan is given as an oral capsule and if licensed, it will offer a treatment option for patients with ALS, potentially delaying the need for mechanical ventilation support.
Abilify MyCite (aripiprazole with sensor) for bipolar disorder and schizophrenia
Abilify MyCite is a drug-device combination comprising aripiprazole embedded with an Ingestible Event Marker (IEM) sensor. The aripiprazole attaches in the brain to receptors which help normalise the activity of the brain. The drug is packaged within an innovative system which helps patients (and healthcare workers) monitor when the medication is taken through integration with a sensor patch and app. Abilify MyCite is different to other ways of monitoring whether medication has been taken because it gives results of the actual medication taken, without need for blood or urine samples, as opposed to an estimate and if licensed, will offer an additional treatment option for patients with BP1 and SCH.
Eptinezumab for prevention of Migraine
Eptinezumab is a drug, which potentially reduces the occurrence of migraine by blocking the CGRP ligand (a protein) from attaching to, and activating its receptor; a process thought to be involved in migraine. In clinical trials, eptinezumab appears effective and well tolerated in the prevention of migraine. If licenced, eptinezumab will offer an additional option for the preventative treatment of migraine with or without aura in adults.
Aducanumab for mild cognitive impairment due to Alzheimer’s disease
Aducanumab is a highly selective human antibody that specifically targets the β-amyloid (Aβ) protein which builds up in abnormal levels in the brain of people with Alzheimer’s disease. High levels of Aβ result in the protein clumping together to form plaques which disrupt neurone function. Aducanumab is given through intravenous infusion and if licensed, would offer an additional treatment option for Alzheimer’s disease and the first to treat the underlying disease rather than the symptoms.
Tofersen for the treatment of amyotrophic lateral sclerosis caused by mutations in the SOD1 gene
Tofersen (BIIB067) is in clinical development for the treatment of amyotrophic lateral sclerosis (ALS – also known as motor neurone disease) caused by mutations in the SOD1 gene (SOD1-ALS). ALS is a progressive disease of the nervous system, where nerve cells in the brain and spinal cord that control voluntary movement gradually deteriorate, causing loss of muscle function and paralysis. ALS is a debilitating and life-threatening disease. The gradual loss of neurons leads to a paralysing effect on muscles used for breathing, which usually leads to death from respiratory failure.