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.
Ofatumumab, taken as an injection under the skin (subcutaneous), reduces the number of B cell lymphocytes, a type of white blood cell which is thought to influence the abnormal immune response that causes the attack on the myelin coating of nerves in MS patients. This is thought to lead to a reduction in the number of these immune cells attacking the myelin sheath that surround and protect the nerves. If licensed, ofatumumab may offer an additional treatment option for patients with relapsing MS. Besides offering the possibility of self-administration by the patient, subcutaneous treatment of ofatumumab can effectively reduce the number of brain lesions without leading to severe depletion of immune B-cells, which is one of the consequences of treatment with intravenous ofatumumab.
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.