Fort Lee, NJ (June 28, 2022) – Kedrion Biopharma, an international biopharmaceutical company specializing in the manufacture and distribution of plasma-derived therapeutic products used in treating rare and serious diseases, was presented with an Industry Innovation Award from the National Organization for Rare Disorders (NORD) for RYPLAZIM®, its treatment for Plasminogen Deficiency Type 1 (PLGD-1). RYPLAZIM, the first FDA-approved treatment for this ultra-rare disorder, was initially developed by Kedrion’s recently acquired subsidiary Prometic Bioproduction in Laval, Canada.
Individuals with PLGD-1 have a deficiency of plasminogen. This protein enables the body to break down fibrin clots, which are involved in the final stages of blood clotting.1 This results in the accumulation of fibrin and leads to the development of lesions on mucus membranes throughout the body.2
The award was presented Sunday at NORD’s Rare Impact Awards at the Rock & Roll Hall of Fame in Cleveland, Ohio. Rebecca Bialas, President of the Plasminogen Deficiency Foundation, presented the award to Giorgio Masetti-Zannini, VP, US Regional Commercial Director of Kedrion Biopharma, who accepted the award on behalf of Kedrion Biopharma. “We thank NORD for this honor and for all the important work they do to support those who suffer from rare diseases and conditions, said Masetti-Zannini. I’d also like to give special recognition to our colleagues at Prometic in Laval, Canada, whose creativity, hard work, and persistence ultimately resulted in the approval by the FDA of RYPLAZIM. Special recognition also goes to all patients, parents, caregivers, and in particular, Dr. Amy Shapiro and the Indiana Hemophilia Treatment Center for their drive to find a solution for their loved ones and patients.
Masetti-Zannini added: “Our mission at Kedrion is to provide patients with rare disease treatments that bring them hope. RYPLAZIM is especially important as it is the first treatment available to people suffering for years from PLGD-1 with no relief. We are proud to deliver this breakthrough treatment to them.”
Rebecca Bialas, the mother of a child with PLGD-1 who participated in the clinical trial for RYPLAZIM, said, “I’m so thankful the folks at Kedrion have taken up this cause and are doing all the wonderful things they’re doing. I can’t put into words how thankful I am that this is happening. This treatment has given us hope that we can move forward with a future for our children and other patients with this disorder.”
Bialas and her husband Ryan founded the Plasminogen Deficiency Foundation, whose mission is to advance the care and treatment of patients with plasminogen deficiency.
NORD holds the Rare Impact Awards annually to recognize individuals and organizations making extraordinary strides on behalf of the millions of Americans impacted by rare diseases.
- Plasminogen replacement therapy for the treatment of children and adults with congenital plasminogen deficiency. Shapiro et al. BLOOD. March 22, 2018. Accessed 6/21/22.
2. An international registry of patients with plasminogen deficiency, (HISTORY). Shapiro et al. Jan 30, 2020. https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7049368/. Accessed 10/12/22
About Kedrion Biopharma
Kedrion Biopharma is a leading international biopharmaceutical company that specializes in the development, production, and distribution of plasma-derived therapeutic products for use in treating serious diseases, disorders, and conditions such as immune system deficiencies and coagulation disorders. Kedrion has special expertise in the treatment of rare diseases and the manufacture of hyper-immune products such as anti-hepatitis B, anti-tetanus, and RhoGAM® for prophylaxis against Rh sensitization and subsequent hemolytic disease of the fetus and newborn.
The company operates through a fully integrated business model from the collection of plasma in its centers in North America, to fractionation and production in its manufacturing facilities located in Italy, Hungary, and North America.
Headquartered in Castelvecchio Pascoli (Italy), Kedrion has over 2,700 employees and a commercial presence in approximately 100 countries worldwide. Kedrion places a high value on the welfare of those who benefit from its products, as well as on the people and the communities it serves. Additional information about Kedrion Biopharma can be found at kedrion.com and kedrion.us.
|Kedrion Biopharma: Sandy Sklareski Communications Manager, North America, and LATAM [email protected]|
INDICATIONS AND USAGE
RYPLAZIM® (plasminogen, human-time) is a plasma-derived human plasminogen indicated for the treatment of patients with plasminogen deficiency type 1 (hypoplasminogenemia).
IMPORTANT SAFETY INFORMATION
RYPLAZIM is contraindicated in patients with known hypersensitivity to plasminogen or other components of RYPLAZIM.
WARNINGS AND PRECAUTIONS:
- Bleeding: RYPLAZIM administration may lead to bleeding at active mucosal disease-related lesion sites or worsen active bleeding not related to disease lesions. Discontinue RYPLAZIM if serious bleeding occurs. Monitor patients during and for 4 hours after infusion when administering RYPLAZIM to patients with bleeding diatheses and patients taking anticoagulants, antiplatelet drugs, or other agents which may interfere with normal coagulation.
- Tissue Sloughing: Respiratory distress due to tissue sloughing may occur in patients with mucosal lesions in the tracheobronchial tree following RYPLAZIM administration. Please monitor appropriately.
- Transmission of Infectious Agents: RYPLAZIM is made from human plasma and therefore carries a risk of transmitting infectious agents, e.g., viruses, the variant Creutzfeldt-Jakob disease (vCJD) agent, and theoretically, the Creutzfeldt-Jakob Disease (CJD) agent.
- Hypersensitivity Reactions: Hypersensitivity reactions, including anaphylaxis, may occur with RYPLAZIM. If symptoms occur, discontinue RYPLAZIM and administer appropriate treatment.
- Neutralizing Antibodies: Neutralizing antibodies (inhibitors) may develop, although they were not observed in clinical trials. If clinical efficacy is not maintained (e.g., development of new or recurrent lesions), determine plasminogen activity trough levels in plasma.
- Laboratory Abnormalities: Patients receiving RYPLAZIM may have elevated blood levels of
D-dimer. D-dimer levels will lack interpretability in patients being screened for venous thromboembolism (VTE).
The most frequent (incidence ≥ 10 percent) adverse reactions in clinical trials were abdominal pain, bloating, nausea, fatigue, extremity pain, hemorrhage, constipation, dry mouth, headache, dizziness, arthralgia, and back pain.
To report SUSPECTED ADVERSE REACTIONS, contact KEDRION at 1-855-427-6378 or the FDA at 1-800-FDA-1088 or www.fda.gov/medwatch
Please see RYPLAZIM’s full Prescribing Information for complete prescribing details. You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch, or call 1-800-FDA-1088.