FDA Grants Kyverna Fast Track Designation for Treatment of Refractory Myasthenia Gravis

Rare Daily Staff

The U.S. Food and Drug Administration granted Fast Track designation Kyverna Therapeutics for its experimental therapy KYV-101, a CAR T-cell product for the treatment of the rare, autoimmune condition refractory myasthenia gravis.

Myasthenia gravis (MG) is associated with muscle weakness in tissues throughout the body, potentially manifesting in partial paralysis of eye movements, problems in chewing and swallowing, respiratory problems, speech difficulties, and weakness in skeletal muscles. MG patients develop antibodies that lead to an immunological attack on critical signaling proteins at the junction between nerve and muscle cells, thereby inhibiting the ability of nerves to communicate properly with muscles. The symptoms of the disease can be transient and in the early stages of the disease can remit spontaneously. However, as the disease progresses, symptom-free periods become less frequent and disease exacerbations can last for months. Disease symptoms reach their maximum levels within two to three years in approximately 80 percent of patients. Up to 20 percent of MG patients experience respiratory crisis at least once in their lives.

KYV-101 is an autologous, fully human CD19 CAR T-cell product in development for use in B cell-driven autoimmune diseases. The CAR in KYV-101 was designed by the National Institutes of Health to improve tolerability and tested in a 20-patient phase 1 trial in oncology. Results were published by the NIH in Nature Medicine.

Fast track designation is a program intended to facilitate and expedite development and review of new drugs to address unmet medical need in the treatment of a serious or life-threatening condition.

Kyverna is currently conducting two trials of KYV-101 in patients with lupus nephritis, an autoimmune disease in which more than half of patients do not achieve a complete response to current therapies and are at risk of developing kidney failure. Additional clinical trials of KYV-101 in systemic sclerosis, myasthenia gravis, and multiple sclerosis are in preparation. The company believes that the differentiated properties of KYV-101 are critical for the potential success of CAR T cells as autoimmune disease therapies.

This is the second time KYV-101 has received a Fast Track designation, after obtaining the first one for lupus nephritis earlier this year.