Drugs

IND Cleared for Ultragenyx mRNA Therapy

FDA has cleared an Ultragenyx Pharmaceutical IND for UX053, an investigational mRNA therapy being evaluated for treating Glycogen Storage Disease Type III (GSDIII). Enrollment in a planned Phase 1/2 study is expected to begin in the second half of this year. The two-part clinical trial will see Part 1 as an open label, 12-patient study who receive a single ascending dose of UX053, according to the company. Part 2 is a randomized, double-blind, placebo-controlled multi-ascending study of five doses in 16 patients across four cohorts randomized 3:1 to UX053 or placebo. In addition to safety, tolerability, and pharmacokinetics, study endpoints include clinician- and patient-reported outcomes, muscle strength, and biomarkers of liver, cardiac, and muscle health, Ultragenyx says.

GSDIII is caused by a glycogen debranching enzyme (AGL) deficiency that leads to glycogen accumulation in the liver and muscle, which can cause hepatomegaly, hypoglycemia, hyperlipidemia, some progressive liver cirrhosis, and skeletal and cardiac muscle disease, the company says. There are no approved treatment options and the current standard of care is a strict diet, including frequent doses of cornstarch, to reduce the risk of hypoglycemia.

“UX053 is an investigational mRNA-based biologic therapy encoding full-length, glycogen debranching enzyme encapsulated in a lipid nanoparticle (LNP) designed to provide the deficient protein in GSDIII,” Ultragenyx 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