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.
Remimazolam is an ultra-short-acting intravenous benzodiazepine sedative/anaesthetic that in the human body is rapidly transformed (metabolized) to an inactive metabolite. Remimazolam is structurally similar to the commonly used sleep-inducing drug midazolam, but it incorporates the pharmacokinetic properties of remifentanil to make the offset of sedation faster and more predictable. If licensed, remimazolam will offer an additional therapy option for procedural sedation in adults during a diagnostic and/or therapeutic procedures.
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.