Rare Disease Report

FDA Grants Rare Pediatric Disease Designation to Plasminogen Replacement Therapy

AUGUST 29, 2017
Mathew Shanley
The U.S. Food and Drug Administration (FDA) has granted a Rare Pediatric Disease Designation to Prometic’s Ryplazimä. The plasminogen replacement therapy has already been granted Orphan Drug and Fast Track Designation.

Ryplazimä is intended for the treatment of patients with congenital plasminogen deficiency.

Congenital plasminogen deficiency is an autosomal recessive condition, in which a patient lacks plasminogen, a naturally occurring protein that is synthesized by the liver and involved in the degradation of plasma proteins. Signs and symptoms vary from patient to patient, but include thick, woody – or ligneous – conjunctivitis and gingivitis, as well as fibrous lesions; all of which requires surgical excision and are likely to recur.

Additionally, hydrocephalus has been reported in 12% of all patients with congenital plasminogen deficiency.

"We are pleased that Ryplazim has received this pediatric designation from the FDA. In addition to Ryplazim's orphan drug status, this designation represents another validation of the approach we have taken regarding our plasminogen's clinical program," said Mr. Pierre Laurin, President and Chief Executive Officer of Prometic in a press release.

"The pediatric designation also provides an important incentive for Prometic to continue developing therapies addressing unmet medical needs for children with rare diseases. We look forward to continue to work closely with the FDA to bringing this innovative therapy to patients suffering from plasminogen deficiency."

There is currently no approved treatment for plasminogen deficiency, and treatment is limited to excision of the fibrous tissue that frequently recurs.
If approved, Prometic’s plasminogen replacement therapy will be eligible to receive a rare pediatric disease priority review voucher, which can be worth upward of several million dollars. Earlier this year, Sarepta sold a Priority Review Voucher for $125 million.

Plasimongen deficiency is extremely rare, and at present, an estimated 1 in every 1-2 million people have been diagnosed with the condition.

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