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.
Daridorexant for Insomnia
Daridorexant is a medicinal product in clinical development for the treatment of adults with insomnia. Insomnia means regularly having problems sleeping, which results in impaired daytime functioning. Insomnia can be categorised according to its duration, into short-term insomnia (lasting less than 4 weeks) and long-term (or persistent) insomnia (lasting 4 weeks or longer).
Lasmiditan for acute treatment of migraine
Lasmiditan is a medicinal product currently in clinical development for the acute treatment of migraine. A migraine is usually a moderate or severe headache felt as a throbbing pain on one side of the head. Many people also have symptoms such as nausea, vomiting and increased sensitivity to light or sound. Migraines may be with aura (specific warning signs just before the migraine begins, such as seeing flashing lights), although the most common type is without aura (no specific warning signs).
Remimazolam for procedural sedation
Remimazolam has completed its main clinical development studies for procedural sedation in adults. Procedural sedation is a technique of administering sedatives or dissociative agents, with or without analgesics, to induce a mental state that allows the patient to tolerate unpleasant diagnostic and therapeutic procedures such as colonoscopy while maintaining cardiorespiratory function. This enables medical procedures to be carried out with the patient benefit of markedly reduced discomfort and no major added risk.
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.