FDA Grants Orphan Drug Designation to Kedrion's QIVIGY® for Stiff Person Syndrome, an Ultra-Rare Neurological Disorder
Designation enhances Kedrion's development program for QIVIGY® and represents an important milestone for patients with Stiff Person Syndrome
FORT LEE, N.J., Sept. 22, 2026 /PRNewswire/ -- Kedrion Biopharma announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to Immune globulin intravenous, human 10% solution (tradename QIVIGY®) for the treatment of Stiff Person Syndrome (SPS), a rare and often disabling neurological disorder estimated to affect approximately two per 100,000 people.
Stiff Person Syndrome is an ultra-rare, immune-mediated disorder marked by progressive muscle rigidity and painful, unpredictable muscle spasms. If left untreated, SPS can significantly affect a person's ability to perform routine tasks and movement. The condition is two to three times more common in women and diagnosis is often delayed by years due to its rarity and common onset in midlife stages, from ages 30 to 50. To date, there are no approved products in the U.S. to treat this condition. Despite the availability of symptomatic treatments, significant unmet patient needs remain.
"Orphan Drug Designation is an important regulatory and clinical milestone as we explore the potential of QIVIGY to address the critical unmet need for people living with Stiff Person Syndrome," said Nisha Jain, M.D., Vice President, Global Clinical Development and Strategy at Kedrion Biopharma. "This designation underscores Kedrion's scientific commitment to advancing plasma-derived therapies for rare and ultra-rare disorders, both through targeted drug development and by expanding the therapeutic reach of established therapies for underserved patient communities."
The FDA grants Orphan Drug Designation to therapies intended to address rare diseases or conditions affecting fewer than 200,000 people in the United States at the time of designation. Under the FDA's Orphan Drug Act, orphan drug status provides incentives, including tax credits, grants, and waiver of certain administrative fees for clinical trials, and seven years of market exclusivity following drug approval.
QIVIGY is currently approved in the U.S. for the treatment of adults with primary humoral immunodeficiency. QIVIGY's safety and efficacy for SPS are being assessed in a clinical trial (NCT 07552987).
"People living with Stiff Person Syndrome continue to face significant diagnostic delays, progressive functional impairment, and limited strategies for symptom management," said Dr. Scott Newsome, Professor of Neurology and Director of the Johns Hopkins Stiff Person Syndrome Center of Excellence, John Hopkins University School of Medicine, Baltimore MD. "Evaluating QIVIGY in structured clinical trials provides an opportunity to assess whether targeted intravenous immunoglobulin therapy can modify disease progression and meaningfully improve functional outcomes."
The continued development of QIVIGY for SPS reflects Kedrion's broader commitment to addressing unmet needs across rare and ultra-rare disease communities and exploring new ways its therapies may support patients with limited treatment options.
"People living with Stiff Person Syndrome have long faced a severe lack of effective therapies for this disease," said Tara Zier, Founder and CEO of The Stiff Person Syndrome Research Foundation. "Seeing QIVIGY being studied specifically for SPS brings excitement to a community that has spent years waiting for dedicated treatment options."
About QIVIGY
QIVIGY® (immune globulin intravenous, human-kthm) 10% solution is indicated for the treatment of adults with primary humoral immunodeficiency (PI). QIVIGY carries a Boxed Warning regarding thrombosis, renal dysfunction and acute renal failure. Please see the full Prescribing Information. QIVIGY is not approved for the treatment of Stiff Person Syndrome; the safety and effectiveness of QIVIGY for this use have not been established.
About Kedrion Biopharma
Kedrion Biopharma collects and fractionates blood plasma to produce and distribute plasma‑derived therapies for rare, ultra‑rare, and debilitating conditions, including coagulation and neurological disorders, immunodeficiencies, and Rh sensitization. We employ approximately 5,400 people worldwide and operate plasma collection centers in the United States and the Czech Republic. With a portfolio of 39 products distributed in over 100 countries, Kedrion is the world's fifth‑largest player in plasma‑derived products.
We are committed to creating a world where science and care know no bounds, working in partnership with the medical‑scientific community, institutions, patient organizations, and research bodies to foster innovation and improve care. Every connection we make impacts someone, somewhere.
IMPORTANT SAFETY INFORMATION
QIVIGY (immune globulin intravenous, human-kthm) is a 10% immune globulin (Ig) liquid indicated for the treatment of adults with primary humoral immunodeficiency. QIVIGY may cause:
Blood clots (thrombosis) may occur in people taking Ig intravenous (IGIV) products, including QIVIGY.
Renal dysfunction, acute renal failure, osmotic nephrosis may occur with immune globulin intravenous (IGIV) products in predisposed patients. Such events require immediate medical intervention, if not recognized or managed appropriately, may result in persistent or significant disability or incapacity or lead to fatal outcome.
For patients at risk of thrombosis, renal dysfunction or failure, administer QIVIGY at the minimum dose available and the minimum infusion rate practicable. Ensure adequate hydration in patients before administration. Monitor for signs and symptoms of thrombosis and assess blood viscosity in patients at risk for hyperviscosity.
Do NOT use QIVIGY if:
You have had an anaphylactic or severe systemic reaction to the administration of human immune globulin and in IgA deficient patients with antibodies against IgA and history of hypersensitivity. WARNINGS AND PRECAUTIONS
Severe hypersensitive reactions, including anaphylaxis, may occur. In case of hypersensitive, discontinue QIVIGY infusion and manage as appropriate.
Hyperproteinemia, hyperviscosity, and hyponatremia may occur in patients receiving IGIV treatment, including QIVIGY.
Aseptic meningitis syndrome may occur in patients receiving IGIV treatment, especially with high doses or rapid infusion.
Hemolysis can develop subsequent to IGIV treatment. Monitor patients for hemolysis.
Transfusion-related acute lung injury: Monitor patients for pulmonary adverse reactions.
Transmissible infectious agents: QIVIGY is made from human plasma and may carry a risk of transmitting infectious agents, e.g., viruses, the variant Creutzfeldt-Jakob disease (vCJD) agent, and, theoretically, the Creutzfeldt-Jakob disease (CJD) agent.
Interference with laboratory tests: After infusion of Ig, transitory rise of passively transferred antibodies may yield positive serological results, with potential for misleading interpretation.
ADVERSE REACTIONS
The most common adverse reactions occurring in > 5% of patients treated were headache, fatigue, infusion-related reaction, Coombs direct test positive, nausea, sinusitis, dizziness, and diarrhea.
To report SUSPECTED ADVERSE REACTIONS, contact Kedrion Biopharma Inc. at 1-855-3KDRION
(1-855-353-7466) or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.
Please see full Prescribing Information for complete prescribing details, including Boxed Warning.
Forward Looking Statements:
This release includes "forward-looking statements," within the meaning of the securities laws and certain other jurisdictions, based on current expectations and projections about future events. All statements other than statements of historical facts included in this release, including, without limitation, statements regarding our future financial position, risks and uncertainties related to our business, strategy, capital expenditures and our plans and objectives for future operations, may be deemed to be forward-looking statements. These forward-looking statements are subject to a number of risks and uncertainties. Words such as "believe," "expect," "anticipate," "may," "assume," "plan," "intend," "will," "should," "estimate," "risk," "target," "objectives," "outlook," "probably," "project," "seek" and similar expressions or the negatives of these expressions are intended to identify forward-looking statements. Although we believe that the expectations reflected in our forward-looking statements are reasonable, we can give no assurance that such expectations will prove to be correct. Any forward-looking statement speaks only as of the date on which it is made, and we undertake no obligation to publicly update or revise any forward-looking statements, whether as a result of new information, future events or otherwise.
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SOURCE Kedrion Biopharma
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