FDA Grants Orphan Drug Designation to Qivigy for People With Stiff Person Syndrome
KEY TAKEAWAYS
- The FDA granted Orphan Drug Designation to Qivigy for the treatment of people with stiff person syndrome, a rare immune-mediated neurologic disorder.
- Qivigy was previously approved for adults with primary humoral immunodeficiency.
- The safety and effectiveness of Qivigy treatment for people with stiff person syndrome are being evaluated in an ongoing clinical trial.
The Food and Drug Administration (FDA) granted Orphan Drug Designation to Qivigy (immune globulin intravenous, human-kthm; Kedrion Biopharma, Fort Lee, NJ) for the treatment of people with stiff person syndrome (SPS), according to a statement from Kedrion Biopharma.
SPS is a rare immune-mediated neurologic disorder characterized by progressive muscle rigidity and painful spasms. The condition can substantially impair mobility and activities of daily living, and no products are currently approved in the United States specifically for those with SPS.
Qivigy is a 10% intravenous immune globulin formulation currently approved for the treatment of adults with primary humoral immunodeficiency. Its safety and effectiveness in people with SPS have not been established, and the therapy is being evaluated in an ongoing clinical trial (NCT07552987).
Regulatory and Clinical Details
- FDA Orphan Drug Designation is intended for therapies being developed for rare conditions affecting fewer than 200,000 people in the United States.
- The designation can provide development incentives, including certain tax credits, fee waivers, grants, and potential market exclusivity after approval.
- Qivigy’s prescribing information contains a Boxed Warning for thrombosis, renal dysfunction, and acute renal failure.
- Other important risks associated with intravenous immune globulin therapy include hypersensitivity, aseptic meningitis, hemolysis, and transfusion-related acute lung injury.
Practical Implications in the Clinic
For neurologists caring for people with SPS, the designation marks a regulatory step in the formal study of an intravenous immune globulin product for this population.
Source
Kedrion Biopharma. FDA grants Orphan Drug Designation to Kedrion’s Qivigy for stiff person syndrome, an ultra-rare neurological disorder. PR Newswire. Published September 22, 2026. Accessed September 25, 2026. https://www.prnewswire.com/news-releases/fda-grants-orphan-drug-designation-to-kedrions-qivigy-for-stiff-person-syndrome-an-ultra-rare-neurological-disorder-302885458.html