FDA Grants Plakous Rare Pediatric Disease Designation for NEC Therapy

Rare Daily Staff

The U.S. Food and Drug Administration granted Plakous Therapeutics Rare Pediatric Disease designation for its human placental extract, Protego-PD, for the prevention of necrotizing enterocolitis, a rare intestinal condition affected early preterm infants.

Necrotizing enterocolitis (NEC) is caused by inflammation and lack of development of the intestine and is observed in very low birth weight babies, those babies born weighing less than three pounds. More than 90 percent of NEC cases occur in very low birth weight babies.  Managing NEC consumes 20 percent of the $5 billion in annual NICU expenditures plus an estimated $4 billion in hospital costs for subsequent treatments. Currently, NEC has no approved treatments or diagnostic tests.

Protego-P, is an orally delivered acellular biotherapeutic developed from post-delivery placentas. It provides growth factor concentrations that prevent inflammation and cartilage breakdown.

The FDA grants Rare Pediatric Disease designation for serious or life-threatening diseases with manifestations in individuals aged from birth to 18 years, including access to the FDA’s expedited review and approval process. The designation makes Protego-P eligible for a Rare Pediatric Disease Priority Review voucher upon approval of the therapy by the FDA. There are no approved therapies for the condition.

The vouchers can be used to reduce the time of an FDA new drug approval review to six months from ten months. The vouchers are potentially lucrative because they are transferable. Most recently, Sarepta sold its priority review voucher to Vifor Pharma $111 million in February 2020.

The FDA also granted Orphan Drug designation to Plakous for Potego-PD.

“With no currently approved treatment for NEC, receiving these designations are a significant step forward to stop this devastating disease from affecting fragile, premature babies,” said Robert Boyce, CEO of Plakous Therapeutics. “We are focused on expanding our successful preclinical data and achieving the regulatory milestones to develop the first FDA-approved treatment for the prevention of NEC.”

Photo: Robert Boyce, CEO of Plakous Therapeutics

Editor’s note: This story was updated to correct the most recent sales of a priority review voucher.