New PKU Pill Gets FDA Orphan Drug Designation

Mathew Shanley

The U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to SYNB1618, a preclinical-stage drug candidate developed by Synlogic to treat patients with phenylketonuria (PKU).

PKU is a genetic disorder in which patients have a reduced ability to metabolize the amino acid phenylalanine (Phe).. An untreated accumulation of Phe can result in cognitive issues and intellectual disability, among other severe health-related issues. SYNB1618 is an orally-administered medication being designed to provide alternative metabolic pathways to degrade Phe in the gut.

The capability to ingest a pill that would allow for the digestion of Phe could radically improve patients’ lives. Currenty, those suffering from the condition are required to stick to a strict dietary protein restriction. There are currently an estimated 16,500 people living with the disorder in the U.S., as per the National PKU Alliance.

“We believe our Synthetic Biotic medicines could transform the treatment of PKU,” said Aoife Brennan, M.B., B.Ch., Synlogic’s chief medical officer in a press release. “Consequently, we were pleased to receive the FDA’s orphan drug designation which validates our approach and represents an important step toward achieving our goal of bringing novel treatments to the patients and families affected by this challenging disease.”

Synlogic has stated its intention to file an investigational new drug application (IND) with the FDA for SYNB1618 for the potential treatment of PKU in early 2018. The IND program is how a pharmaceutical company attains authorization to send an experimental drug across state lines – usually to clinical investigators – before a marketing application for the drug has been approved.

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Image courtesy WikiMedia Commons.
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