Plasminogen (medication)
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| Trade names | Ryplazim |
| Other names | plasminogen, human-tvmh |
| License data |
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| Routes of administration | Intravenous |
| ATC code |
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| Legal status | |
| Legal status | |
Plasminogen, sold under the brand name Ryplazim, is a biologic medication for the treatment of hypoplasminogenemia (plasminogen deficiency type 1).[1][2][3] It is purified from human plasma[1][2] and is administered intravenously.[1][4]
The most common side effects include abdominal pain, bloating, nausea, bleeding, limb pain, fatigue, constipation, dry mouth, headache, dizziness, joint pain, and back pain.[1][2]
Individuals with hypoplasminogenemia lack a protein called plasminogen, which is responsible for the ability of the body to break down fibrin clots.[2] Plasminogen deficiency leads to an accumulation of fibrin, causing the development of growths (lesions) that can impair normal tissue and organ function and may lead to blindness when these lesions affect the eyes.[2]
Plasminogen, human-tvmh was approved for medical use in the United States in June 2021.[2] It is the first therapy for hypoplasminogenemia approved by the U.S. Food and Drug Administration (FDA).[2]
Medical uses[edit]
Plasminogen, human-tvmh is indicated for the treatment of people with plasminogen deficiency type 1, also referred to as hypoplasminogenemia, a disorder that can impair normal tissue and organ function and may lead to blindness.[1][2]
History[edit]
The effectiveness and safety of plasminogen is primarily based on one single-arm, open-label (unblinded) clinical trial enrolling 15 adult and pediatric participants with plasminogen deficiency type 1.[2] All participants received plasminogen administered every two to four days for 48 weeks.[2] The effectiveness of plasminogen was demonstrated by at least 50% improvement of their lesions in all 11 participants who had lesions at baseline, and absence of recurrent or new lesions in any of the 15 participants through the 48 weeks of treatment.[2]
The U.S. Food and Drug Administration (FDA) granted the application for plasminogen orphan drug designation,[5] fast track designation, priority review, and a rare pediatric disease priority review voucher.[2] The FDA granted approval of Ryplazim to ProMetic Biotherapeutics Inc.[2]
References[edit]
- ^ a b c d e f "Ryplazim- plasminogen injection, powder, lyophilized, for solution". DailyMed. Retrieved 3 July 2021.
- ^ a b c d e f g h i j k l m n "FDA Approves First Treatment for Patients with Plasminogen Deficiency, a Rare Genetic Disorder". U.S. Food and Drug Administration (FDA) (Press release). 4 June 2021. Retrieved 5 June 2021.
This article incorporates text from this source, which is in the public domain.
- ^ "Liminal BioSciences Announces FDA Approval for its Biologics License Application for Ryplazim (plasminogen, human-tvmh)" (Press release). Liminal BioSciences Inc. 4 June 2021. Retrieved 5 June 2021 – via PR Newswire.
- ^ Kuehn BM (August 2021). "First Treatment for Plasminogen Deficiency Is Approved". JAMA. 326 (5): 379. doi:10.1001/jama.2021.12278. PMID 34342632. S2CID 236883889.
- ^ "Plasminogen Orphan Drug Designations and Approvals". U.S. Food and Drug Administration (FDA). 5 March 2013. Retrieved 5 June 2021.
This article incorporates public domain material from the United States Department of Health and Human Services
External links[edit]
- "Plasminogen". Drug Information Portal. U.S. National Library of Medicine.
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