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Investing.com -- PTC (NASDAQ:PTC) Therapeutics (NASDAQ:PTCT) stock rose 9% after the company announced that the U.S. Food and Drug Administration (FDA) has approved its drug SEPHIENCE (sepiapterin) for the treatment of phenylketonuria (PKU).
The approval includes broad labeling for the treatment of hyperphenylalaninemia (HPA) in adult and pediatric patients 1 month of age and older with sepiapterin-responsive PKU. This comprehensive labeling allows the company to target all disease subtypes across a wide age range.
"We are excited to have reached this important milestone for those affected by PKU," said Matthew B. Klein, M.D., Chief Executive Officer of PTC Therapeutics. "The broad labeling reflects the potential of Sephience to meet the significant unmet need of PKU patients."
The FDA approval is based on efficacy and safety data from the Phase 3 APHENITY trial and durability of treatment effect demonstrated in the APHENITY long-term extension study.
Catherine Warren, Executive Director of the National PKU Alliance, called the approval "an exciting milestone for the PKU community" that "brings renewed hope" for individuals with the condition.
SEPHIENCE recently received marketing authorization from the European Commission, with approval applications under review in several other countries including Japan and Brazil.
The company plans to leverage its experience in launching rare disease therapies to bring the treatment to U.S. patients quickly, with customer-facing teams already prepared for the rollout.
PTC Therapeutics will host a conference call on Monday, July 28th at 5:00 pm ET to discuss the approval.
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