SYBX - Synlogic's metabolic disorder therapy gets EMA backing for orphan drug tag

2023-03-28 09:13:18 ET

• The European Medicines Agency (EMA) issued a positive opinion on Synlogic's ( NASDAQ: SYBX ) application seeking orphan designation for SYNB1934 to treat phenylketonuria (PKU).
• PKU is a rare inherited metabolic disorder which causes an amino acid called phenylalanine to build up in the body. People with PKU cannot properly break down the extra phenylalanine.
• "This designation also comes at a pivotal time as we prepare to begin our Phase 3 trial for PKU in the first half of this year," said Synlogic President and CEO Aoife Brennan.
• The orphan drug status in the EU is aimed at therapies for treating or preventing diseases which affect fewer than five in 10,000 people in the EU. The designation provides incentives, including 10 years of market exclusivity for the drug, if approved.
• SYNB1934 previously received rare pediatric disease designation from the U.S. Food and Drug Administration (FDA).

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