Drugs

Pediatric Menkes Disease Drug Approved

FDA has approved a Sentynl Therapeutics NDA for Zycubo (copper histidinate) injection as the first treatment for Menkes disease in pediatric patients. Menkes disease is a neurodegenerative disorder caused by a genetic defect that impairs a child’s ability to absorb copper. “The disease is characterized by seizures, failure to gain weight and grow, developmental delays, and intellectual disability, FDA says. “It leads to abnormalities of the vascular system, bladder, bowel, bones, muscles, and nervous system. Children with classical Menkes (90% of those with the disease) begin to develop symptoms in infancy and typically do not live past three years.

Subcutaneous injectable Zycubo is a copper replacement therapy that delivers copper in a form that bypasses the genetic defect in intestinal absorption, allowing the body to better use the mineral. FDA says it evaluated data from two open-label, single-arm clinical trials in pediatric patients treated for up to three years. “Children who began treatment within four weeks of birth had a 78% reduction in the risk of death compared with untreated patients,” it says. “Nearly half of early-treated patients survived beyond six years, and some survived more than 12 years. No patients in the untreated control group survived beyond six years. Children who started treatment later than four weeks after birth also experienced a substantial survival benefit.”

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

Read more

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