Omar Sinno, MD, Medical Lead of Rare Disease at UCB, describes the company’s dedication to developing treatment for rare diseases like myasthenia gravis.
Myasthenia gravis is a chronic autoimmune neuromuscular disease characterized by weakness of the skeletal muscles. Common symptoms include weakness of the muscles, especially in the face that make it difficult to control eye movement, facial expressions, chewing, talking, and swallowing. The condition results from a defect in the transmission of nerve impulses to muscles, which is due to the presence of antibodies against acetylcholine receptors or other neuromuscular endplate receptors. Treatment is limited to managing symptoms and there are no targeted therapies or cures currently available for this rare condition.
As Dr. Sinno explains, UCB’s interest in developing a targeting treatment for myasthenia gravis was sparked by an employee’s frustration with the treatment landscape for rare neuromuscular disorders. This employee had a relative with one such disorder who was being treated with intravenous immunoglobulin (IVIg). The employee saw that the treatment options available for these disorders only manage the symptoms of the disease rather than target the disease itself. Through his work, the FcRn inhibitor came about which, according to Dr. Sinno, was the impetus for UCB to move into the rare neuromuscular disorder space.
Currently, two molecules are being investigated by UCB to treat myasthenia gravis (rozanolixizumab; zilucoplan) and they are in phase 3 clinical trials (NCT03971422; NCT04115293).
To learn more about myasthenia gravis, visit our Myasthenia Gravis Learning Center here.
