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FDA Approves Kedrion's Bolognana Facility for RYPLAZIM Production, Expanding Access to PLGD-1 Treatment

• The FDA has approved Kedrion Biopharma's Bolognana facility for producing RYPLAZIM, the first and only treatment for plasminogen deficiency type 1 (PLGD-1). • This approval significantly expands RYPLAZIM's production capacity, ensuring more patients globally can access this crucial therapy for the rare disease. • RYPLAZIM addresses the root cause of PLGD-1, offering patients the potential for a near-normal life by targeting fibrin-rich lesions. • The expanded production aims to meet the growing demand for RYPLAZIM, providing confidence to diagnosed patients that they will receive needed treatment.

The U.S. Food and Drug Administration (FDA) has approved Kedrion Biopharma's manufacturing facility in Bolognana, Italy, for the production of RYPLAZIM (human plasminogen). This approval marks a significant step in expanding access to the only available treatment for plasminogen deficiency type 1 (PLGD-1), a rare and chronic disease characterized by the development of fibrin-rich lesions on mucosal surfaces throughout the body.

Addressing a Critical Unmet Need in PLGD-1

PLGD-1 manifests through the formation of abnormal extra-vascular, ligneous lesions, most commonly observed in the eye (ligneous conjunctivitis). However, these lesions can also appear in the ears, mouth, central nervous system, skin, and respiratory, gastrointestinal, and genitourinary tracts. These lesions can lead to severe complications, including vision and hearing loss, airway obstruction, infertility, and hydrocephalus, highlighting the urgent need for effective treatment options.

RYPLAZIM: A Targeted Therapy for PLGD-1

RYPLAZIM is a plasma-derived human plasminogen product designed to address the underlying cause of PLGD-1. As the first and only FDA-approved therapy specifically for this condition, RYPLAZIM represents a major advancement in patient care, offering the possibility of a near-normal life for those affected.

Increased Production Capacity to Meet Growing Demand

"The FDA's approval of our Bolognana facility is a pivotal moment for our company," 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."
The approval of the Bolognana facility enables Kedrion to significantly increase RYPLAZIM production, ensuring that this crucial treatment can reach more PLGD-1 patients in the United States and worldwide. This expansion is particularly important given the increased awareness of PLGD-1 and the growing demand for RYPLAZIM.

Impact on the PLGD-1 Community

Rebecca Bialas MD, Co-founder and Chair of The Plasminogen Deficiency Foundation, stated, "With increased awareness of PLGD-1, the expanded capacity of RYPLAZIM ensures we are better equipped to meet the growing demand. 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."
This accomplishment underscores Kedrion's commitment to scientific excellence and patient-focused innovation, marking a crucial step in increasing RYPLAZIM's capacity and improving the lives of individuals and families affected by PLGD-1.
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FDA Approves Kedrion's Facility for RYPLAZIM Production, Expanding Access to PLGD-1 Treatment

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Reference News

[1]
FDA Approves Kedrion's Bolognana Facility for Production of First and Only Treatment for ...
newswise.com · Nov 21, 2024

FDA approves Kedrion Biopharma's Bolognana, Italy facility to produce RYPLAZIM®, a plasma-derived human plasminogen for ...

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