Drugs

Applied Therapeutics Seeks Pre-NDA Meeting for Govorestat

Applied Therapeutics plans to request a pre-NDA meeting with FDA on its govorestat (AT-007), a central nervous system penetrant aldose reductase inhibitor for treating patients with galactosemia, after reporting data from a recent Phase 3 study. Govorestat demonstrated “consistent long-term clinical outcomes benefit across a range of functional measures in the ACTION-Galactosemia Kids trial, confirming prior biomarker data,” the company says. Additionally, it says data showed that the drug “improved activities of daily living, behavior, cognition, fine motor skills, adaptive skills and tremor vs. placebo.”

The study was designed to evaluate the impact of govorestat treatment vs. placebo on clinical outcomes over time in 47 children ages two to 17 years. The primary endpoint, a composite score on the Global Statistical Test, was not met, Applied Therapeutics says, adding that systematic improvement over time was demonstrated and pre-specified sensitivity analyses were met. Govorestat provided a statistically significant benefit on tremor at 18 months (p=0.0428), as measured by the Archimedes Spiral Drawing Test, and adaptive skills as assessed by the BASC-3 Adaptive Skills Index (p=0.0265), it says.

Galactosemia is described by the company as a rare genetic metabolic disease resulting in an inability to metabolize the simple sugar galactose. “When not metabolized properly, galactose is converted to the toxic metabolite, galactitol, which causes neurological complications, including deficiencies in speech, cognition, behavior, and motor skills, and also results in juvenile cataracts and ovarian insufficiency (in women),” it says.

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