dm+d

Unassigned

New Medicines

RyplazimPlasminogen type 1 deficiency

Information

Ryplazim
New formulation
Kedrion Biopharma
Kedrion Biopharma

Development and Regulatory status

None
Phase III Clinical Trials
Approved (Licensed)
Yes
Yes
Oct 21US launch planned for 2022. Kedrion is ramping up production capacity to meet patient demand and anticipates Ryplazim will initially be available in limited quantities [9].
Jun 21Liminal sells Ryplazim, and its subsidiary Prometic, to Kedrion [8].
Jun 21Approved in US [7].
Sep 20Liminal announce resubmission of BLA to FDA [6].
Jun 20Liminal hopes for re-submission of BLA in the third quarter of 2020 [5].
Nov 19Liminal expects to complete the necessary manufacturing and related activities to allow for submission to the FDA of an amendment to the BLA in the first half of 2020 [4].
Apr 19ProMetic 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 18FDA issues a Complete Response Letter requesting further clinical data and changes to the product´s manufacturing assays and in-process controls [3].
Oct 17US 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 17Granted Rare Pediatric Disease Designation by the US FDA for the treatment of congenital plasminogen deficiency [2].
Jul 17ProMetic 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 15Granted orphan drug status in EU. Also has orphan drug status in US [2].

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