FDA Grants Kedrion's Bolognana Plant Approval for Production of PLGD-1 Treatment

Image Credit: Adobe Stock Images/ArakiIllustrations.com

The FDA has approved Kedrion Biopharma Inc.’s manufacturing facility in Bolognana, Italy, for the production of Ryplazim (plasminogen, human-tvmh).¹ This plasma-derived human plasminogen is currently indicated for the treatment of patients with plasminogen deficiency type 1 (PLGD-1), also known as hypoplasminogenemia.

PLGD-1 is a rare chronic disease that mainly appears in the form of “abnormal extra-vascular fibrin-rich, ligneous” lesions on various mucosal surfaces throughout the body. Most notably, lesions occur in the eye (known as ligneous conjunctivitis); however, their appearance in the respiratory, gastrointestinal, and genitourinary tracts—along with the central nervous system, ears, mouth, and skin—is not uncommon. They can potentially lead to serious consequences, including obstruction of airways; infertility; and hydrocephalus, along with vision and hearing loss.

"The FDA's approval of our Bolognana facility is a pivotal moment for our company, and I want to thank all employees worldwide who contributed to this achievement," said Bob Rossilli, CCO, global business and US general manager. "This expanded capacity for Ryplazim means we can provide treatment for more patients with PLGD-1. Offering a product like RYPLAZIM to patients in need is truly a privilege."

This development signifies greater patient access to this PLGD-1 medication, not only in the United States, but globally as well. Ryplazim is considered the first and only FDA-approved therapy specifically for the PLGD-1 indication after receiving approval in June 2021; Kedrion officially announced plans to commercialize and distribute the product later that year.² The active ingredient in Ryplazim is plasminogen, which is purified from human plasma.³ Treatment with Ryplazim helps to increase the plasma level of plasminogen, helping to limit or remove the lesions.

In a single-arm, open-label (unblinded) clinical trial that enrolled 15 adult and pediatric patients with PLGD-1, patients were administered Ryplazim every two to four days for 48 weeks. Overall, the medication’s effectiveness was apparent by at least a 50% improvement of their lesions in all 11 patients who had lesions at baseline, while there were no new lesions in any of the 15 patients through these 48 weeks.3 The most common side effects that patients reported were abdominal pain, back pain, bloating, bleeding, constipation, dry mouth, dizziness, fatigue, limb pain, nausea, headache, and joint pain.3

"With increased awareness of PLGD-1, the expanded capacity of Ryplazim ensures we are better equipped to meet the growing demand, noted Rebecca Bialas MD, co-founder and chair of The Plasminogen Deficiency Foundation. “This means more patients who receive the correct diagnosis can be confident they will get the treatment they so desperately deserve and need. This is an exciting time for the PLGD-1 community, marking a significant event in our collective journey towards better health."

References

1. FDA Approves Kedrion's Bolognana Facility for Production of First and Only Treatment for PLGD-1, Ryplazim. PR Newswire. November 19, 2024. Accessed November 22, 2024. https://www.prnewswire.com/news-releases/fda-approves-kedrions-bolognana-facility-for-production-of-first-and-only-treatment-for-plgd-1-ryplazim-302313070.html

2. Kedrion Biopharma to Commercialize Ryplazim in US. Kedrion Biopharma. November 19, 2024. Accessed November 22, 2024. https://www.kedrion.us/kedrion-biopharma-to-commercialize-ryplazim-in-u-s/

3. FDA Approves First Treatment for Patients with Plasminogen Deficiency, a Rare Genetic Disorder. US Food & Administration. June 4, 2021. Accessed November 22, 2024. https://www.fda.gov/news-events/press-announcements/fda-approves-first-treatment-patients-plasminogen-deficiency-rare-genetic-disorder