Drugs

PTC Therapeutics NDA for Treating Phenylketonuria

FDA has accepted for review a PTC Therapeutics NDA for sepiapterin, which the company says is )indicated for treating pediatric and adult patients with phenylketonuria (PKU), including all age groups and the full spectrum of disease subtypes. The agency has a user fee review action target date of 7/29/2025.

The submission is based on data from the Phase 3 APHENITY trial, recently published in The Lancet, which the company says provides evidence of the durability of effect of sepiapterin and the ability of patients to liberalize their diet and still maintain control of phenylalanine (Phe) levels. “The most recent results from the Phe tolerance assessment demonstrate that approximately 60% of subjects achieve protein intake above the age-adjusted recommended daily allowance for an unaffected individual while still maintaining Phe levels < 360 μmol/L,” it adds.

PKU is described as a rare, inherited metabolic disease affecting the brain. “It is caused by a defect in the gene that helps create the enzyme needed to break down phenylalanine,” PTC Therapeutics says. “If left untreated or poorly managed, phenylalanine — an essential amino acid found in all proteins and most foods — can build up to harmful levels in the body. This causes severe and irreversible disabilities, such as permanent intellectual disability, seizures, delayed development, memory loss, and behavioral and emotional problems.”

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