Plasmin
- Generic Name
- Plasmin
- Brand Names
- Ryplazim
- Drug Type
- Biotech
- CAS Number
- 9001-91-6
- Unique Ingredient Identifier
- 1EF190B6M7
- Background
Plasminogen is a pro-enzyme (i.e. a zymogen) which is cleaved to form plasmin - also known as fibrinolysin - as part of the fibrinolytic pathway that breaks down fibrin blood clots. This pathway is activated when a clot is no longer needed or to prevent a clot from extending beyond the site of injury.
In June 2021, the FDA approved a plasma-derived plasminogen (Ryplazim, human plasminogen-tvmh) for the treatment of type 1 plasminogen deficiency (hypoplasminogenemia). It is the first and only FDA-approved treatment for this condition, which causes wood-like lesions to form on the mucous membranes of patients, providing an unmet medical need for patients with this rare congenital disease.
- Indication
Plasma-derived human plasminogen, marketed under the brand name Ryplazim, is indicated for the treatment of patients with plasminogen deficiency type 1 (hypoplasminogenemia).
- Associated Conditions
- Type I Plasminogen Deficiency
Alnylam Unveils Ambitious Pipeline and Platform Innovation at R&D Day
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• Alnylam presented promising data on emerging clinical programs for Huntington's disease, bleeding disorders, and type 2 diabetes, while outlining platform innovations to deliver RNAi therapeutics to all major tissue types by 2030.
Cerebrolysin Shows Promise in Improving Outcomes After Mechanical Thrombectomy for Acute Stroke
A groundbreaking study reveals that Cerebrolysin, when used as an adjuvant therapy following mechanical thrombectomy in acute ischemic stroke patients, significantly improved functional outcomes. The treatment led to a notable increase in favorable outcomes at 90 days and reduced the risk of hemorrhagic transformation, marking a potential advancement in post-stroke care.
Acoramidis Gains Global Momentum: FDA Approval, EU Recommendation, and Promising Clinical Data
• Acoramidis (Attruby), developed by Stanford Medicine and BridgeBio, receives FDA approval for transthyretin amyloid cardiomyopathy (ATTR-CM) treatment, marking a significant milestone.
• The European Medicines Agency's CHMP recommends acoramidis for EU marketing authorization based on positive Phase 3 ATTRibute-CM trial results.
• Clinical trials demonstrate acoramidis' efficacy in reducing cardiovascular-related hospitalizations and improving survival rates for ATTR-CM patients.
• Bayer and BridgeBio collaborate to commercialize acoramidis, with Bayer holding EU rights and plans for a launch in Europe in early 2025.
FDA Approves Kedrion's Facility for RYPLAZIM Production, Expanding Access for PLGD-1 Patients
• The FDA has approved Kedrion Biopharma's Bolognana facility in Italy for RYPLAZIM production, a crucial treatment for plasminogen deficiency type 1 (PLGD-1).
• This approval significantly expands RYPLAZIM's production capacity, ensuring more patients in the U.S. and worldwide can access this vital therapy.
• RYPLAZIM is the first and only FDA-approved treatment for PLGD-1, addressing the root cause of the disease and improving patients' quality of life.
• The expanded capacity addresses the growing demand for RYPLAZIM, ensuring that more diagnosed patients can receive the treatment they need.
Muvalaplin Shows Promise in Lowering Lipoprotein(a) Levels in High-Risk Patients
• A phase 2 trial evaluated muvalaplin, an oral small molecule lipoprotein(a) inhibitor, in patients with elevated lipoprotein(a) and high cardiovascular risk.
• Muvalaplin demonstrated significant, dose-dependent reductions in lipoprotein(a) levels, with up to 85.8% reduction at the 240 mg/d dosage, using an intact lipoprotein(a) assay.
• The study reported that muvalaplin was generally well-tolerated, with no significant safety concerns observed across the different dosage groups.
• These findings suggest muvalaplin could be a potential therapeutic option for individuals with elevated lipoprotein(a), though further studies are needed to assess its impact on cardiovascular events.
The TREATT Trial: Evaluating Tranexamic Acid in Haematological Malignancies
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Drug Development Updates
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