Fort Lee, New Jersey, U.S., September 22, 2026
Kedrion Biopharma has announced a significant regulatory milestone for its rare-disease development portfolio after the U.S. Food and Drug Administration (FDA) granted Orphan Drug Designation (ODD) to QIVIGY® (Immune Globulin Intravenous Human 10% solution) for the potential treatment of Stiff Person Syndrome (SPS). The designation supports Kedrion’s ongoing clinical development efforts in an ultra-rare neurological disorder characterized by progressive muscle rigidity and painful, unpredictable muscle spasms. The company said the milestone reinforces its commitment to developing plasma-derived therapies for rare and ultra-rare diseases where substantial unmet medical needs remain.
FDA Designation Advances Rare Disease Program
The FDA’s Orphan Drug Designation applies to QIVIGY® for Stiff Person Syndrome, a rare immune-mediated neurological condition that can significantly affect mobility and quality of life. People living with SPS may experience progressive muscle stiffness and severe spasms, creating substantial challenges in everyday activities. While symptomatic treatment options are available, Kedrion said significant unmet medical needs remain for patients with the disorder. The new designation provides an important regulatory milestone as the company investigates whether QIVIGY® can provide a therapeutic option for this underserved patient population.
Kedrion’s development strategy reflects its broader focus on immunoglobulin therapies and rare diseases. The company specializes in plasma-derived treatments and has been expanding its research and development activities across rare and serious conditions. QIVIGY® is an immune globulin product that is already part of Kedrion’s commercial portfolio, while its use in Stiff Person Syndrome remains investigational. The company is currently evaluating the safety and efficacy of QIVIGY® in a clinical study for the new indication.
QIVIGY® Enters Clinical Evaluation for SPS
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The FDA designation comes as Kedrion advances a clinical program evaluating intravenous immune globulin 10% in adults with Stiff Person Syndrome. A Phase 3 study sponsored by Kedrion is registered to investigate the therapy in adults with SPS, with the study designed to evaluate its potential treatment effects in this rare neurological population. The clinical development program is particularly significant because SPS is an ultra-rare disorder, meaning clinical research can face challenges associated with identifying and enrolling sufficiently large patient populations. The development of therapies for rare diseases therefore requires focused clinical research and specialized regulatory pathways. Kedrion’s latest milestone demonstrates how established plasma-derived therapies can also be investigated for additional indications where significant patient needs remain.
The company said the ODD represents both a regulatory and clinical development milestone for its SPS program. Nisha Jain, Vice President of Global Clinical Development and Strategy at Kedrion, highlighted the designation as part of the company’s efforts to explore new treatment possibilities for people living with Stiff Person Syndrome and other ultra-rare conditions.
Expanding Kedrion’s Rare Disease Pipeline
The SPS program adds another potential indication to Kedrion’s expanding rare-disease research and development pipeline. The company has previously pursued additional indications for its plasma-derived therapies, including programs targeting other rare and immune-mediated disorders. Its broader portfolio includes therapies and development programs across immunodeficiency, hematology and rare diseases. For the Stiff Person Syndrome program, however, FDA Orphan Drug Designation does not represent marketing approval. Safety and efficacy of QIVIGY® for SPS are still being evaluated in clinical research, and the company must generate clinical evidence before the therapy could potentially receive approval for this indication.
The designation nevertheless represents an important step in the development pathway for a potential therapy targeting a rare neurological disease with significant unmet medical needs. As Kedrion continues its clinical evaluation of QIVIGY®, future study results will help determine the potential role of the therapy in Stiff Person Syndrome and contribute to the broader development of treatment options for patients affected by this debilitating condition.
Source: Kedrion Biopharma press release
