The Food and Drug Administration (FDA) has approved Ryplazim® (plasminogen, human-tmvh) for the treatment of patients with plasminogen deficiency type 1, also referred to as hypoplasminogenemia.

Hypoplasminogenemia is a rare genetic disorder caused by a lack of plasminogen that results in the accumulation of fibrin. A deficiency in plasminogen leads to the development of lesions on mucous membranes of the body and may lead to blindness if the lesions occur in the eye. Ryplazim contains plasminogen, purified from human plasma, which is designed to help increase the plasma levels of plasminogen.

The approval was based on data from a single-arm, open-label phase 2/3 trial (ClinicalTrials.gov Identifier: NCT02690714) that assessed the efficacy and safety of Ryplazim in 15 adult and pediatric patients with hypoplasminogenemia. All patients received Ryplazim 6.6mg/kg as an intravenous infusion every 2 to 4 days for 48 weeks. The primary endpoint was overall clinical success at 48 weeks, defined as 50% of patients with visible or other measurable lesions achieving at least 50% improvement in lesion number/size, or functionality impact from baseline.

Findings showed at least 50% improvement in the number or size of lesions in 11 patients who had lesions at baseline, and an absence of recurrent or new lesions in all 15 patients through 48 weeks. The most common adverse reactions reported were abdominal pain, bloating, nausea, fatigue, extremity pain, hemorrhage, constipation, dry mouth, headache, dizziness, arthralgia, and back pain.


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Ryplazim may prolong or worsen active bleeding; monitoring is recommended for patients with bleeding diatheses and for those on medications that may interfere with normal coagulation. In patients with mucosal lesions in the tracheobronchial tree, treatment with Ryplazim may cause respiratory distress due to tissue sloughing.

“Until now, there were no FDA-approved treatment options for patients with plasminogen deficiency type 1,” said Peter Marks, MD, PhD, director of FDA’s Center for Biologics Evaluation and Research. “Today’s approval helps address an unmet medical need for individuals affected by this rare genetic disease.”

References

  1. FDA approves first treatment for patients with plasminogen deficiency, a rare genetic disorder. [press release]. Silver Spring, MD: US Food and Drug Administration; June 4, 2021. 
  2. Liminal BioSciences announces FDA approval for its Biologics License Application for Ryplazim® (plasminogen, human-tvmh). [press release]. Laval, Québec and Cambridge, England; Liminal BioSciences Inc.; June 4, 2021.