Business
Kyverna's KYV-101 Receives U.S. FDA RMAT Designation for KYV-101 in the Treatment of Patients With Refractory Stiff-Person Syndrome
The application was evaluated based on the positive clinical outcomes of KYV-101 in patients treated in Germany under a named-patient treatment option The
About this update from Kyverna Therapeutics, Inc.
[{"type":"text","content":"The application was evaluated based on the positive clinical outcomes of KYV-101 in patients treated in Germany under a named-patient treatment option\nThe Regenerative Medicine Advanced Therapies designation will allow Kyverna to receive expert guidance on efficient drug development and use of surrogate endpoints from senior FDA officials\nEMERYVILLE, Calif., July 15, 2024 /PRNewswire/ -- Kyverna Therapeutics, Inc. (Kyverna), a patient-centered, clinical-stage biopharmaceutical company focused on developing cell therapies for patients suffering from autoimmune diseases, announced today the designation as Regenerative Medicine Advanced Therapy (RMAT) by the U.S. Food and Drug Administration (FDA) for its autologous, fully human CD19 chimeric antigen receptor (CAR) T-cell product candidate, KYV-101, to be used for the treatment of patients suffering from refractory stiff-person syndrome. \n\n \n \n \n \n \n \n\n \n\"Stiff-person syndrome has devastating and life-altering effects on patients suffering from this rare autoimmune disease,\" said Amanda Piquet, M.D., director of the Autoimmune Neurology Program at CU Anschutz Medical Campus, Aurora, CO. \"I look forward to the data that will emerge from the KYSA-8 trial as this trial could drastically change the treatment landscape for SPS.\"\n\"We are eager to begin generating data from our sponsored trial to advance the knowledge on a potential immunological reset of the patient's immune system,\" said Peter Maag, Ph.D., chief executive officer at Kyverna. \"We are humbled by the resilience of the SPS patients and their hope for a potential paradigm-shifting treatment option that could provide durable, immunosuppressant-free remission.\"\nAbout Stiff Person Syndrome (SPS) SPS is a rare, progressive neurological autoimmune disorder causing debilitating muscle stiffness in the torso, arms, and legs, impacting the ability to walk or move. Patients typically present with muscle spasms and stiffness, resulting in difficulty turning and bending. When stiffness is severe, the patients' posture resembles a statue. Muscle spasms and stiffness can be precipitated by unexpected stimuli, including sounds, like a phone ring or a siren, sudden touches or conditions triggering anxiety and emotional upset which, when severe, are misdiagnosed as a primary anxiety disorder1. \nAbout KYV-101KYV-101 is...