Synlogic Receives Rare Pediatric Disease Designation from FDA for SYNB1934 for Phenylketonuria (PKU)

The U.S. Food and Drug Administration granted Rare Pediatric Disease designation to Synlogic’s SYNB1934 for the potential treatment of phenylketonuria.

Photo: Dave Hava, head of Research and Development at Synlogic

Phenylketonuria (PKU) is a rare, inherited metabolic disorder caused by a deficiency of the enzyme phenylalanine hydroxylase (PAH). This enzyme is required for the breakdown of phenylalanine (Phe), an amino acid found in all protein-containing foods. When PAH is deficient or defective, Phe accumulates to abnormally high levels in the blood, and if left untreated, these toxic levels of Phe in the blood can result in progressive and severe neurological impairment and neuropsychological complications.

SYNB1934 is an orally administered, non-systemically absorbed drug candidate being studied as a potential treatment for phenylketonuria (PKU) that is designed to reduce levels of Phe in people with PKU using precision genetic engineering of the well-characterized probiotic E. coli Nissle. Treatment options for PKU are currently limited due to efficacy and safety, and many of those who are treated need additional Phe-lowering.

“This designation for SYNB1934 demonstrates the urgent need for new PKU treatment options for patients, especially children,” said Dave Hava, head of Research and Development at Synlogic. “This is also extraordinary news as our program heads towards initiation of Synpheny-3, the pivotal phase 3 study for SYNB1934 in PKU in the first half of the year.”

The FDA grants Rare Pediatric Disease designation for serious and life-threatening diseases that primarily affect children ages 18 years or younger and fewer than 200,000 people in the United States. The Rare Pediatric Disease Priority Review Voucher program is intended to address the challenges that drug companies face when developing treatments for these unique patient populations. Under this program, a sponsor who receives an approval for a drug or biologic for a “rare pediatric disease” may be eligible for a voucher that can be redeemed to receive priority review of a subsequent marketing application for a different product or sold to another sponsor for priority review of their marketing application.

Recently, Bluebird Bio sold two priority review vouchers: one to Argenx for $102 million and one to Bristol Myers Squibb for $95 million.

Author: Rare Daily Staff