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Ryplazim Biologics License Application Accepted by the FDA

OCTOBER 13, 2017
Mathew Shanley
Yesterday, Rare Disease Report published an interview with Pierre Laurin, the President and CEO of Prometic, developers of the plasminogen replacement therapy Ryplazim.

This morning, the U.S. Food and Drug Administration (FDA) announced its acceptance of the drug’s Biologics License Application (BLA), granted it a priority review status and set a Prescription Drug User Fee Act (PDUFA) action date for April 14, 2018. The drug had previously been granted Fast Track, Orphan Drug and Rare Pediatric Disease designations by the FDA.

Congenital plasminogen deficiency is an autosomal recessive condition in which the lack of plasminogen – a protein synthesized by the liver and essential to the degradation of plasma proteins – can lead to a variety of problems, like conjunctivitis and gingivitis, that impede a patient’s vision.

Patients are also at risk for fibrous lesions that require surgery, are likely to recur after removal, and can cause permanent damage to the eyes.

Ryplazim is an endogenous protein, extracted by an FDA-approved process from plasma and processed, using a proprietary procedure, to form a stable form of plasminogen that can be stored in a vial.

"The acceptance of our BLA filing and setting of the PDUFA date by the FDA is a significant milestone for Prometic", said Laurin, President and Chief Executive Officer of Prometic in a press release. "The FDA has recognized the seriousness and unmet need of available therapeutic solutions for plasminogen deficiency, granting orphan, fast-track and rare pediatric disease designations. We will continue to work closely with the FDA in the coming months with the goal of making our plasminogen replacement therapy Ryplazim™ available to patients as soon as possible.”

In July, Prometic announced positive long-term clinical data of Ryplazim, in which the drug was shown to prevent recurrence of lesions at 48 weeks and maintained the same safety and tolerability profiles without any adverse events (AEs).

“The pediatric designation gives us a chance to be eligible for a Priority Review Voucher,” Laurin said in the interview with RDR. “We can use this voucher for a future drug in our pipeline and/or monetize it. These vouchers have been sold anywhere from $70 million to over $300 million in the recent past, so we are really proud to have received this pediatric designation. The program by the FDA is a great incentive for companies to focus on pediatric indications that are often overlooked.”

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