According to a story from BioSpace, the biotechnology company Viela Bio recently announced that the US Food and Drug Administration (FDA) has awarded an experimental therapy in development by the company Breakthrough Therapy designation. The therapy that received the designation is inebilizumab, which is being developed for neuromyelitis optica spectrum disorder (NMOSD).
About Neuromyelitis Optica Spectrum Disorders
Neuromyelitis optica spectrum disorders (NMOSD) is a term meant to include both neuromyelitis optica patients and those that lack the APQ4 auto antibody but still present similarly otherwise. This disorder is also known as Devic’s disease. It is characterized by inflammation of the optic nerve and spinal cord along with destruction of the myelin sheath, an insulating, protective layer surrounding nerve cells. It is considered an autoimmune disease in which the immune system mistakenly begins attacking parts of the body. It is frequently associated with other diseases, such as viral infection and antiMOG associated encephalomyelitis, that latter of which can be a direct cause in some cases. Symptoms include blindness, urinary incontinence, spastic paralysis of the legs and arms, reduced sensation, and overall muscle weakness. Symptoms can be treated, but many patients are left with a degree of impairment. To learn more about neuromyelitis optica, click here.
About Breakthrough Therapy Designation
There is a clear and urgent need for improved therapeutic options for neuromyelitis optica spectrum disorder. Breakthrough Therapy designation is intended to streamline the development process for therapies that are designed to treat severe, life-threatening diseases. To earn this designation, a therapy must display clinically relevant advantages compared to currently available treatments on one or more critical endpoints. There are a number of benefits for the recipient company if they receive Breakthrough Therapy designation, including greater organizational commitment from the FDA, more guidance on drug testing and development, and access to a scientific liaison that can shorten the review period.
The designation is based on encouraging results from a study of 231 patients with neuromyelitis optica spectrum disorder. After a 6.5 month initial treatment period, in which the effect of inebilizumab was compared to placebo, the study entered an open label extension in which all patients were treated every six months.