Patients affected stiff person syndrome are one step closer to having access to a new treatment.
Kyverna Therapeutics’ new drug, KYV-101, has been designated by the U.S. Food and Drug Administration (FDA) as a regenerative medicine advanced therapy (RMAT), the company announced Monday.
A drug is eligible for RMAT designation if it is “intended to treat, modify, reverse or cure a serious disease or life-threatening disease or condition” and if “preliminary clinical evidence indicates that the drug has the potential to address unmet medical needs for that disease or condition,” according to the FDA website.
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One benefit of RMAT is that Kyverna will be able to work closely with the FDA to help support expedited development, review and approval, according to the company.
The FDA’s decision is based on positive results from clinical trials in patients, the company said in a press release.
Carrie Robinette, left, was diagnosed with stiff person syndrome in 2023. The FDA named KYV-101 a regenerative medicine advanced therapy (RMAT) on Monday. (Carrie Robinette; iStock)
A rare neurological disorder, stiff person syndrome affects only one or two people in a million, including singer Celine Dion.
The disease can have a devastating impact, causing muscle stiffness, pain, spasms and loss of mobility.
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Kyverna Therapeutics, based in Emeryville, Californiahas developed a novel CAR-T cell therapy, KYV-101, with the goal of “resetting” the immune system of patients with autoimmune diseases, according to the company.
“As a physician dedicated to optimizing diagnosis and treatment for patients with autoimmune diseases neurological disorders“I am grateful to be able to witness and contribute to the advancement of treatments and outcomes for patients with stiff person syndrome through collaborative research efforts with key players in the field and the support of FDA oversight,” Amanda Piquet, MD, director of the autoimmune neurology program at CU Anschutz Medical Campus in Aurora, Colo., told Fox News Digital via email.
Celine Dion shared her Stiff Person Syndrome diagnosis with the world in December 2022. (Edward Berthelot/GC Images/Getty Images)
Piquet was not involved in the development of KYV-101, but served on one of the medical advisory boards.
Peter Maag, PhD, CEO of Kyverna, also commented on the announcement.
“This RMAT designation means that science agrees that accelerating this treatment could save lives.”
“Kyverna is focused on supporting patients and their unmet clinical needs by creating patient-centric clinical trials to enable access to the latest medical advancements in the CAR-T space for autoimmune diseases,” Maag told Fox News Digital in a written statement.
“We are proud to be able to study the potential of safe and effective methods long term treatment “This can lead to significant and lasting remission for patients with autoimmune diseases like PSS.”
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Patients also reacted to the news with a sense of excitement.
“As soon as I heard what the Kyverna treatment did for the SPS patient in Germany, my dream was to have it quickly approved by the FDA so that we could all benefit,” Carrie Robinette, 45, of San Diego, California, told Fox News Digital.
A drug is eligible for RMAT designation if it is “intended to treat, modify, reverse, or cure a serious or life-threatening disease or condition” and if “preliminary clinical evidence indicates that the drug has the potential to address unmet medical needs for that disease or condition,” according to the FDA website. (REUTERS/Andrew Kelly/File Photo)
Robinette, a Navy Wife and his mother, were diagnosed with stiff person syndrome in spring 2023.
“This RMAT designation means that science agrees that accelerating this treatment could save lives,” she added. “I am thrilled for the entire autoimmune disease community.”
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During clinical trials, 50 patients with oncological and autoimmune diseases were treated with KYV-101 at more than 15 sites in Europe and the United States, according to the company.
Kyverna will now continue to collect data from Phase 2 trials of the drug for stiff person syndrome, multiple sclerosis and myasthenia gravis.
Carrie Robinette, 45, of San Diego, Calif., left, and Corwyn Wilkey, 44, of Anchorage, Alaska, right, both live with stiff person syndrome. (Carrie Robinette; Corwyn Wilkey)
Phase 1 and 2 trials are also underway for systemic sclerosis and lupus.
“We are eager to begin generating data from our sponsored trial “to advance knowledge about a potential immunological reset of the patient’s immune system,” Maag said in the company’s statement.
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Piquet added: “Stiff Person Syndrome has devastating and life-changing effects on patients suffering from this rare autoimmune disease… I look forward to the data that will emerge from the KYSA-8 trial, as this trial could radically change the treatment landscape for PSS.”
Fox News Digital has reached out to the FDA for additional comment.
