PTC Therapeutics receives FDA approval for PKU treatment drug

PTC Therapeutics Inc. (NASDAQ: PTCT) announced that the U.S. Food and Drug Administration has approved Sephience (sepiapterin) for treating phenylketonuria in children and adults. The approval covers patients 1 month of age and older with sepiapterin-responsive PKU.

The FDA approval is based on evidence from the Phase 3 APHENITY trial and its long-term extension study. Sephience is indicated for treating hyperphenylalaninemia in adult and pediatric patients with PKU and must be used alongside a phenylalanine-restricted diet.

"The broad labeling reflects the potential of Sephience to meet the significant unmet need of PKU patients," said Matthew B. Klein, chief executive officer of PTC Therapeutics.

The European Commission recently granted marketing authorization for Sephience. Review of approval applications continues in Japan and Brazil.

PKU is a rare inherited metabolic disease affecting approximately 58,000 people globally. The condition is caused by a defect in the gene that creates the enzyme needed to break down phenylalanine. Without proper management, phenylalanine can build up to harmful levels, causing intellectual disability, seizures, and developmental delays.

Sephience is a natural precursor of the enzymatic co-factor BH4, which is critical for phenylalanine hydroxylase. The drug works to reduce blood phenylalanine levels.

Common adverse reactions with Sephience include diarrhea, headache, abdominal pain, hypophenylalaninemia, feces discoloration, and oropharyngeal pain. The drug may increase bleeding risk and can cause hypophenylalaninemia in some pediatric patients.

PTC Therapeutics will host a conference call on July 28 at 5:00 p.m. ET to discuss the approval.

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