New drug for stiff person syndrome granted designation to speed up development: ‘Thrilled for the community’

Neurologist Dr. Scott Newsome, director of the Johns Hopkins Stiff Person Syndrome Center, explains the popular singer's diagnosis. Patients with 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) a Regenerative Medicine Advanced Therapy (RMAT), the company announced on Monday.

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 such disease or condition," according to the FDA’s website. STIFF PERSON SYNDROME PATIENTS SHARE WHAT IT’S LIKE TO LIVE WITH THE RARE DISEASE One of the benefits of RMAT is that Kyverna will be able to work closely with the FDA to help support accelerated development, review and approval, according to the company. The FDA’s decision was based on positive results from clinical trials with patients, a company press release stated.

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 for every million — including singer Celine Dion .

The disease can have a devastating impact, causing muscle rigidity.