Plasminogen

Unassigned

New Medicines

Ryplazim · Plasminogen type 1 deficiency

Information

Ryplazim
New formulation
ProMetic Life Sciences
ProMetic Life Sciences

Development and Regulatory status

None
Phase III Clinical Trials
Not approved
Yes
Yes

Apr 19: ProMetic will submit the manufacturing data requested by the FDA, along with the requested clinical data as an amendment to the BLA in the first half of 2020 [3].


Dec 18: FDA issues a Complete Response Letter requesting further clinical data and changes to the product´s manufacturing assays and in-process controls [3].


Oct 17: US FDA accepts the BLA for IV formulation of plasminogen and grants priority review status, with a PDUFA action date of April 14, 2018 [2].


Aug 17: Granted Rare Pediatric Disease Designation by the US FDA for the treatment of congenital plasminogen deficiency [2].


Jul 17: ProMetic is in talks with the EMA with regards to the clinical information that is required to secure regulatory approval of plasminogen in Europe [2].


Aug 15: Granted orphan drug status in EU. Also has orphan drug status in US [2].

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Category

Plasminogen is a plasma‐derived drug which increases the plasminogen levels in the body. Plasminogen on activation is converted to plasmin which dissolves the fibrinogen fibers that entangle the blood cells in a blood clot.
Severe hypoplasminogenemia (HPG) or type 1 plasminogen (plg) deficiency is a systemic disease characterised by markedly impaired extracellular fibrinolysis leading to the formation of ligneous (fibrin-rich) pseudomembranes on mucosae during wound healing. Prevalence of severe HPG is in the range of 1.6 per 1,000,000 [1]
Plasminogen type 1 deficiency
Intravenous infusion

Trial or other data

Nov 16: PII/III study meets its primary endpoint of targeted increase in plasma concentration of plasminogen and secondary endpoint of completed healing of lesions was achieved, with significant clinical benefit and 100% response rate in patients with long-standing lesions and other primary endpoints, including a targeted increase in the blood plasma concentration level of plasminogen as a surrogate target. The trial enrolled patients in the US and Norway to evaluate intravenous plasminogen in patients with type 1 plasminogen deficiency (NCT02690714). The US FDA accepted the company´s proposed PII/III programme with a surrogate endpoint for approval using the accelerated approval pathway [2].

Evidence based evaluations