Lacosamide for primary generalised tonic-clonic seizures – adjunctive therapy
Lacosamide is a medicinal product that is being developed for the treatment of Primary Generalised Tonic-Clonic Seizures (PGTCS). Epilepsy is a neurological disorder that is characterised by an imbalance in the excitation and inhibition of the brain and this imbalance causes a phenomenon known as a seizure. Seizures are brief increases in electrical activity within …
Ofatumumab for relapsing multiple sclerosis
Ofatumumab is in clinical development for the treatment of relapsing multiple sclerosis (MS). Multiple sclerosis is an autoimmune disease, meaning the body’s own immune cells (which usually fight infection) attack and damage the nerves and brain. This causes a range of issues including problems with walking, balance, memory and thinking as well as pain, tiredness and many other symptoms.
Fenfluramine hydrochloride for treatment of seizures associated with Lennox-Gastaut syndrome
Fenfluramine belongs to a class of drugs called the selective serotonin releasing agonists which stimulates multiple 5-HT receptor sub-types through the release of serotonin. Fenfluramine may also act on other receptors and these actions may help to reduce the frequency of seizures. When added to other standard anti-epileptic treatments, fenfluramine hydrochloride has shown preliminary evidence of reducing seizure frequency. If licensed, fenfluramine hydrochloride may offer an additional treatment option for patients with Lennox-Gastaut syndrome.
Nerinetide for acute ischaemic stroke
Nerinetide is an innovative new drug that protects the brain during an acute ischaemic stroke. As a neuroprotectant, nerinetide does not dissolve blood clots, but effectively pauses the toxic chemical reactions triggered by stroke. Treatment with nerinetide is being positioned to be initiated as soon as possible after the onset of symptoms. If licensed, nerinetide would provide critical time to patients with a stroke by stopping the loss of brain cells until further treatment can be administered.
BIIB092 for progressive supranuclear palsy
BIIB092 is a product that is being investigated for the treatment of progressive supranuclear palsy (PSP). PSP is a rare condition that is a result of destruction of nerve cells in certain parts of the brain causing problems with balance, movement, vision, speech and swallowing. In patients with PSP, an abnormal form of a protein called tau accumulates in specific areas of the brain by spreading from brain cell to brain cell leading to their damage. Over time, PSP gets progressively worse, with people becoming severely disabled within three to five years of onset. Currently, there is no cure for PSP and no treatment to slow down the disease.
Selumetinib for neurofibromatosis type 1
Selumetinib is in clinical development for children with neurofibromatosis type 1 (NF1), also called von Recklinghausen’s disease. NF1 is a rare genetic disorder characterized by the development of multiple benign tumours of nerves and skin and areas of abnormal skin colour. NF1 is caused by mutation in a gene that regulates the production of a …
Edaravone for amyotrophic lateral sclerosis
Edaravone as an intravenous injection is in clinical development for people with amyotrophic lateral sclerosis (ALS). ALS is a neurological condition that affects nerve cells in the brain and
spinal cord. It results in gradual weakness and wasting of muscles of the body. Respiratory muscles are involved as the disease progresses, leading to shortness of breath and ultimately
death. Little is known about the cause of the disease, and there is currently no cure.
Inebilizumab for Neuromyelitis optica
Inebilizumab is a humanised monoclonal antibody that is in clinical development for reducing the risk of an attack in patients with Neuromyelitis Optica Spectrum Disorders (NMOSD). NMOSD, previously known as NMO and as Devic’s disease, is a rare, disabling autoimmune disease of the central nervous system. It predominantly affects the optic nerve and spinal cord, …
Sarizotan for Respiratory Symptoms Associated Rett Syndrome
Sarizotan is an oral medicinal product that is being developed for the treatment of respiratory symptoms associated with Rett syndrome. Sarizotan works by binding to serotonin and dopamine receptors. By stimulating serotonin and dopamine receptors, sarizotan replaces the effect of some of the missing serotonin in the brain and spinal cord. This is expected to help restore normal breathing rhythm in patients with Rett syndrome. Currently, there is no cure for Rett syndrome and treatment focuses on managing the symptoms. If licensed, sarizotan could become the first therapy approved for treatment of Rett Syndrome patients.