FDA Granted Orphan Drug Status to Qivigy
The drug candidate is being tested for treating the rare autoimmune neurological condition stiff person syndrome.
Updated on Sept. 25, 2026 in Autism
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The Food and Drug Administration has awarded Orphan Drug designation to Qivigy, an immune globulin intravenous 10% solution, for the treatment of stiff person syndrome. This rare autoimmune disorder is characterized by progressive muscle rigidity and painful muscle spasms.
Why it matters
Orphan Drug designation provides incentives for developing treatments for rare conditions that might otherwise lack commercial interest. Clinical evidence suggests that immune globulin therapies can help reduce muscle stiffness and improve mobility for patients with the syndrome.
The Phase 3 trial involves 38 adult participants receiving a 10% solution administered every 4 weeks over a 24-week period. The study measures efficacy through the Timed 25-Foot Walk test.
The players
Food and Drug Administration
The federal agency responsible for protecting public health by ensuring the safety and efficacy of human drugs and biological products.
Qivigy
An immune globulin intravenous 10% solution currently indicated for primary humoral immunodeficiency in adults.
The details
Qivigy, which is already approved for primary humoral immunodeficiency, is now being evaluated for its potential to alleviate the debilitating symptoms of stiff person syndrome. The treatment is administered via intravenous infusion over a period of 2 to 5 days per cycle.
Timeline
The FDA granted Orphan Drug designation on September 22, 2026.
Health Landscape
The designation of Qivigy follows the regulatory pathways established by the Orphan Drug Act to encourage investment in treatments for rare neurological disorders. This move highlights the ongoing scientific efforts to repurpose existing immunotherapies for complex autoimmune conditions.
Patients suffering from the rare autoimmune condition may gain access to a new therapeutic option if the ongoing clinical trials successfully demonstrate efficacy. This designation does not immediately change current treatment availability but represents a step toward potential regulatory approval.
The takeaway
The FDA's decision marks a critical milestone for a therapy that could eventually offer relief to those with limited treatment options for stiff person syndrome. Families and patients should consult with specialists regarding clinical trial participation as data continues to emerge.
Further reading
Learn more about the latest research and regulatory updates on Autism.
More information
View the official Kedrion news release for additional details.
Source note: This article includes information reported by MPR.
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