Drugs

Regeneron Files sBLA for Evkeeza in Young Children

FDA has accepted for priority review a Regeneron Pharmaceuticals supplemental BLA for Evkeeza (evinacumab-dgnb) as an adjunct to other lipid-lowering therapies to treat children aged 5 to 11 years with homozygous familial hypercholesterolemia (HoFH). The agency has set a user fee review action date of 3/30/2023.

HoFH is a rare inherited condition that affects about 1,300 patients in the U.S. It occurs when two copies of the FH-causing genes are inherited, one from each parent, which leads to dangerously high levels of low-density lipoprotein-cholesterol (LDL-C or bad cholesterol), according to the company.

The submission is based on data from a trial evaluating Evkeeza’s efficacy in 14 children. Regeneron says the trial met its primary endpoint, showing children who added Evkeeza to other lipid-lowering therapies reduced their bad cholesterol by 48% at week 24.

Evkeeza is described as the first angiopoietin-like 3 targeted therapy approved by FDA as an adjunct therapy for patients aged 12 years and older with HoFH.

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