Drugs

PTC Therapeutics’ Phenylketonuria Drug Approved

FDA has approved a PTC Therapeutics NDA for Sephience (sepiapterin), indicated for treating adult and pediatric patients with phenylketonuria (PKU), a rare inherited metabolic disorder. “The approval includes broad labeling for the treatment of hyperphenylalaninemia (HPA) in adult and pediatric patients one month of age and older with sepiapterin-responsive PKU,” the company says.

Approval was based on data from the Phase 3 APHENITY trial, which demonstrated both significant efficacy and safety, the company says, adding that the findings were further supported by long-term extension data showing sustained benefits over time.

Phenylketonuria is caused by a deficiency in phenylalanine hydroxylase (PAH), an enzyme required to break down the amino acid phenylalanine, PTC Therapeutics says. Without proper treatment, phenylalanine can build up to harmful levels, leading to intellectual disability and other complications. Sephience is said to work by providing sepiapterin, a precursor to the essential PAH cofactor BH4, helping to restore the body’s ability to metabolize phenylalanine.

Gupta Raises Concerns About Overton’s Independence, Ideology

FDA commissioner nominee Heidi Overton’s responses on vaccines, contraception and abortion medication during her 9/24 Senate confirmation hearing raise concerns that ideology could influence her decision-making if she takes over the agency, MSNBC senior medical analyst Dr. Vin Gupta said Friday. Gupta was particularly critical of Overton’

Overton Confirmation Remains Uncertain After Senate Hearing

FDA commissioner nominee Heidi Overton emerged from her Senate confirmation hearing Thursday (see earlier story) without resolving questions about whether she has enough Republican support to advance — and with a rapidly narrowing window for confirmation before the midterm elections. The Senate Health, Education, Labor and Pensions Committee hearing provided few

FDA Classifies Boston Scientific Imager II Catheter Recall as Class I

FDA has classified Boston Scientific's recall of its Imager II Angiographic Catheters as Class I because a manufacturing problem could cause catheter tips to degrade and detach, potentially resulting in life-threatening complications. The agency announced the Class I classification 9/25, updating an Early Alert it issued

FDA Approves Gazyva for Childhood-Onset Idiopathic Nephrotic Syndrome

FDA has approved Roche’s Gazyva (obinutuzumab) for reducing the risk of relapse in adults and children aged 2 and older with frequently relapsing or steroid-dependent, childhood-onset idiopathic nephrotic syndrome who are in remission. The expanded approval covers the use of the CD20-directed antibody beyond its existing

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Gupta Raises Concerns About Overton’s Independence, Ideology

Overton Confirmation Remains Uncertain After Senate Hearing

FDA Classifies Boston Scientific Imager II Catheter Recall as Class I

FDA Approves Gazyva for Childhood-Onset Idiopathic Nephrotic Syndrome