Ryplazim · Plasminogen type 1 deficiency
Development and Regulatory status
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 .
Dec 18: FDA issues a Complete Response Letter requesting further clinical data and changes to the product´s manufacturing assays and in-process controls .
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 .
Aug 17: Granted Rare Pediatric Disease Designation by the US FDA for the treatment of congenital plasminogen deficiency .
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 .
Aug 15: Granted orphan drug status in EU. Also has orphan drug status in US ..
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 .