Drugs

FDA Orphan Status for Netherton Syndrome Drug

FDA has granted Quoin Pharmaceuticals an orphan drug designation for QRX003 and its use in treating Netherton syndrome, a rare and severe genetic skin disorder. Quoin is conducting two pivotal, late-stage clinical trials evaluating QRX003 lotion (4%) in patients with Netherton syndrome. Enrollment in both studies is expected to conclude in the first quarter of 2026, with top-line results anticipated in the second half, according to the company. It plans to submit an NDA in late 2026.

In patients with Netherton syndrome, a mutated gene leads to the absence of a protein called LEKTI. LEKTI normally inhibits certain enzymes, or serine proteases, which regulate skin shedding, the company says. Without LEKTI, these enzymes become overactive, causing severe and excessive skin shedding and creating a compromised skin barrier, it says.

QRX003 is a topical lotion that contains a broad-spectrum serine protease inhibitor. When applied to the skin, its active ingredient performs the function of the missing LEKTI protein by down-regulating the overactive enzymes, Quoin says, adding that this restores a more normal skin-shedding process and helps to form a stronger and more effective skin barrier.

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