FDA Grants Special Status for Kyverna's KYV-101 to Help Treat Severe Stiff-Person Syndrome
The FDA has fast-tracked a new treatment called KYV-101 for people with severe stiff-person syndrome, which has shown promising improvements in walking, pain, and stiffness with manageable side effects.
By
Isabella Ciccone, MPH
| Published on July 27, 2024
5 min read
Amanda Piquet, MD
The FDA has granted special regenerative medicine advanced therapy designation to Kyverna Therapeutics’ KYV-101, a type of treatment called CAR T-cell therapy, to help people with severe stiff-person syndrome (SPS) who haven’t responded to other treatments.1 Amanda Piquet, MD, director of the Autoimmune Neurology Program at University of Colorado Anschutz Medical Campus, expressed her gratitude for the progress being made in SPS treatment through collaborative research and FDA support.
"As a physician dedicated to optimizing diagnosis and treatments for patients with autoimmune neurological disorders, I am grateful to be able to witness and contribute to the advancement of treatments and patient outcomes in stiff person syndrome via collaborative research efforts with leading players in the field and the support of the FDA oversight," she told The Educated Patient's sister site NeurologyLive®.
In June 2024, Kyverna shared results from a case study involving a 59-year-old woman with SPS who was treated with KYV-101.2 The woman’s symptoms began in 2014, and after receiving the treatment in Germany, her condition improved significantly.3 Her walking speed more than doubled within 20 days, and she was able to walk up to 6 kilometers with the help of a walker by day 90, a huge improvement from only walking a few meters at the start. She also experienced less stiffness and pain, with stiffness in her knee almost disappearing, and her pain dropping to zero after day 50. Her fatigue levels also improved, and she needed less medication as time went on.
The patient did have some mild side effects from the treatment, including a fever and low blood pressure caused by cytokine release syndrome (CRS), but these were successfully managed with medication. There were also temporary increases in her liver enzymes, which improved on their own by day 45.
Researchers believe KYV-101 shows promise as a new long-lasting treatment for SPS and other similar autoimmune diseases. The study authors plan to explore further how the treatment affects the brain, B-cells (a type of immune cell), and long-term outcomes. Kyverna’s CEO, Peter Maag, emphasized the company’s commitment to creating patient-focused clinical trials to provide new and effective treatments for autoimmune diseases without the need for long-term immunosuppressive therapy.
"Kyverna is focused on supporting patients and their clinical unmet needs, building patient centric clinical trials to allow access to the latest medical advances in the CAR-T space for autoimmune disease. We are proud to be able to investigate the potential of safe and effective long lasting treatment that can lead to meaningful, durable remission for patients suffering from autoimmune diseases like SPS," Maag told NeurologyLive. "The data generated from all our ongoing trials will improve our understanding how KYV-101 can represent a paradigm shift in patients' treatment without relying on long-term immunosuppressive therapies."
An original version of this article was published on sister site Neurology Live.
REFERENCES
1. Kyverna's KYV-101 Receives U.S. FDA RMAT Designation for KYV-101 in the Treatment of Patients With Refractory Stiff-Person Syndrome. News Release. Kyverna Therapeutics. Published July 15, 2024. Accessed July 24, 2024. https://www.prnewswire.com/news-releases/kyvernas-kyv-101-receives-us-fda-rmat-designation-for-kyv-101-in-the-treatment-of-patients-with-refractory-stiff-person-syndrome
2. Faissner S, Motte J, Sgodzai M, et al. Successful use of anti-CD19 CAR T cells in severe treatment-refractory stiff-person syndrome. Proc Natl Acad Sci USA. 2024;121(26):e2403227121. doi: doi.org/10.1073/pnas.2403227121
3. First-in-disease use of Kyverna Therapeutics' KYV-101 in patient with severe stiff-person syndrome published in proceedings of the national academy of sciences (PNAS). News release. Kyverna Therapeutics, Inc. June 17, 2024. Accessed June 18, 2024. https://ir.kyvernatx.com/news-releases/news-release-details/first-disease-use-kyverna-therapeutics-kyv-101-patient-severe
